medical research questions

77 interesting medical research topics for 2024

Last updated

25 November 2023

Reviewed by

Brittany Ferri, PhD, OTR/L

Medical research is the gateway to improved patient care and expanding our available treatment options. However, finding a relevant and compelling research topic can be challenging.

Use this article as a jumping-off point to select an interesting medical research topic for your next paper or clinical study.

• How to choose a medical research topic

When choosing a research topic , it’s essential to consider a couple of things. What topics interest you? What unanswered questions do you want to address? 

During the decision-making and brainstorming process, here are a few helpful tips to help you pick the right medical research topic:

Focus on a particular field of study

The best medical research is specific to a particular area. Generalized studies are often too broad to produce meaningful results, so we advise picking a specific niche early in the process. 

Maybe a certain topic interests you, or your industry knowledge reveals areas of need.

Look into commonly researched topics

Once you’ve chosen your research field, do some preliminary research. What have other academics done in their papers and projects? 

From this list, you can focus on specific topics that interest you without accidentally creating a copycat project. This groundwork will also help you uncover any literature gaps—those may be beneficial areas for research.

Get curious and ask questions

Now you can get curious. Ask questions that start with why, how, or what. These questions are the starting point of your project design and will act as your guiding light throughout the process. 

For example: 

What impact does pollution have on children’s lung function in inner-city neighborhoods? 

Why is pollution-based asthma on the rise? 

How can we address pollution-induced asthma in young children? 

• 77 medical research topics worth exploring in 2023

Need some research inspiration for your upcoming paper or clinical study? We’ve compiled a list of 77 topical and in-demand medical research ideas. Let’s take a look. 

• Exciting new medical research topics

If you want to study cutting-edge topics, here are some exciting options:

COVID-19 and long COVID symptoms

Since 2020, COVID-19 has been a hot-button topic in medicine, along with the long-term symptoms in those with a history of COVID-19. 

Examples of COVID-19-related research topics worth exploring include:

The long-term impact of COVID-19 on cardiac and respiratory health

COVID-19 vaccination rates

The evolution of COVID-19 symptoms over time

New variants and strains of the COVID-19 virus

Changes in social behavior and public health regulations amid COVID-19

Vaccinations

Finding ways to cure or reduce the disease burden of chronic infectious diseases is a crucial research area. Vaccination is a powerful option and a great topic to research. 

Examples of vaccination-related research topics include:

mRNA vaccines for viral infections

Biomaterial vaccination capabilities

Vaccination rates based on location, ethnicity, or age

Public opinion about vaccination safety 

Artificial tissues fabrication

With the need for donor organs increasing, finding ways to fabricate artificial bioactive tissues (and possibly organs) is a popular research area. 

Examples of artificial tissue-related research topics you can study include:

The viability of artificially printed tissues

Tissue substrate and building block material studies

The ethics and efficacy of artificial tissue creation

• Medical research topics for medical students

For many medical students, research is a big driver for entering healthcare. If you’re a medical student looking for a research topic, here are some great ideas to work from:

Sleep disorders

Poor sleep quality is a growing problem, and it can significantly impact a person’s overall health. 

Examples of sleep disorder-related research topics include:

How stress affects sleep quality

The prevalence and impact of insomnia on patients with mental health conditions

Possible triggers for sleep disorder development

The impact of poor sleep quality on psychological and physical health

How melatonin supplements impact sleep quality

Alzheimer’s and dementia 

Cognitive conditions like dementia and Alzheimer’s disease are on the rise worldwide. They currently have no cure. As a result, research about these topics is in high demand. 

Examples of dementia-related research topics you could explore include:

The prevalence of Alzheimer’s disease in a chosen population

Early onset symptoms of dementia

Possible triggers or causes of cognitive decline with age

Treatment options for dementia-like conditions

The mental and physical burden of caregiving for patients with dementia

• Lifestyle habits and public health

Modern lifestyles have profoundly impacted the average person’s daily habits, and plenty of interesting topics explore its effects. 

Examples of lifestyle and public health-related research topics include:

The nutritional intake of college students

The impact of chronic work stress on overall health

The rise of upper back and neck pain from laptop use

Prevalence and cause of repetitive strain injuries (RSI)

• Controversial medical research paper topics

Medical research is a hotbed of controversial topics, content, and areas of study. 

If you want to explore a more niche (and attention-grabbing) concept, here are some controversial medical research topics worth looking into:

The benefits and risks of medical cannabis

Depending on where you live, the legalization and use of cannabis for medical conditions is controversial for the general public and healthcare providers.

Examples of medical cannabis-related research topics that might grab your attention include:

The legalization process of medical cannabis

The impact of cannabis use on developmental milestones in youth users

Cannabis and mental health diagnoses

CBD’s impact on chronic pain

Prevalence of cannabis use in young people

The impact of maternal cannabis use on fetal development 

Understanding how THC impacts cognitive function

Human genetics

The Human Genome Project identified, mapped, and sequenced all human DNA genes. Its completion in 2003 opened up a world of exciting and controversial studies in human genetics.

Examples of human genetics-related research topics worth delving into include:

Medical genetics and the incidence of genetic-based health disorders

Behavioral genetics differences between identical twins

Genetic risk factors for neurodegenerative disorders

Machine learning technologies for genetic research

Sexual health studies

Human sexuality and sexual health are important (yet often stigmatized) medical topics that need new research and analysis.

As a diverse field ranging from sexual orientation studies to sexual pathophysiology, examples of sexual health-related research topics include:

The incidence of sexually transmitted infections within a chosen population

Mental health conditions within the LGBTQIA+ community

The impact of untreated sexually transmitted infections

Access to safe sex resources (condoms, dental dams, etc.) in rural areas

• Health and wellness research topics

Human wellness and health are trendy topics in modern medicine as more people are interested in finding natural ways to live healthier lifestyles. 

If this field of study interests you, here are some big topics in the wellness space:

Gluten sensitivity

Gluten allergies and intolerances have risen over the past few decades. If you’re interested in exploring this topic, your options range in severity from mild gastrointestinal symptoms to full-blown anaphylaxis. 

Some examples of gluten sensitivity-related research topics include:

The pathophysiology and incidence of Celiac disease

Early onset symptoms of gluten intolerance

The prevalence of gluten allergies within a set population

Gluten allergies and the incidence of other gastrointestinal health conditions

Pollution and lung health

Living in large urban cities means regular exposure to high levels of pollutants. 

As more people become interested in protecting their lung health, examples of impactful lung health and pollution-related research topics include:

The extent of pollution in densely packed urban areas

The prevalence of pollution-based asthma in a set population

Lung capacity and function in young people

The benefits and risks of steroid therapy for asthma

Pollution risks based on geographical location

Plant-based diets

Plant-based diets like vegan and paleo diets are emerging trends in healthcare due to their limited supporting research. 

If you’re interested in learning more about the potential benefits or risks of holistic, diet-based medicine, examples of plant-based diet research topics to explore include:

Vegan and plant-based diets as part of disease management

Potential risks and benefits of specific plant-based diets

Plant-based diets and their impact on body mass index

The effect of diet and lifestyle on chronic disease management

Health supplements

Supplements are a multi-billion dollar industry. Many health-conscious people take supplements, including vitamins, minerals, herbal medicine, and more. 

Examples of health supplement-related research topics worth investigating include:

Omega-3 fish oil safety and efficacy for cardiac patients

The benefits and risks of regular vitamin D supplementation

Health supplementation regulation and product quality

The impact of social influencer marketing on consumer supplement practices

Analyzing added ingredients in protein powders

• Healthcare research topics

Working within the healthcare industry means you have insider knowledge and opportunity. Maybe you’d like to research the overall system, administration, and inherent biases that disrupt access to quality care. 

While these topics are essential to explore, it is important to note that these studies usually require approval and oversight from an Institutional Review Board (IRB). This ensures the study is ethical and does not harm any subjects. 

For this reason, the IRB sets protocols that require additional planning, so consider this when mapping out your study’s timeline. 

Here are some examples of trending healthcare research areas worth pursuing:

The pros and cons of electronic health records

The rise of electronic healthcare charting and records has forever changed how medical professionals and patients interact with their health data. 

Examples of electronic health record-related research topics include:

The number of medication errors reported during a software switch

Nurse sentiment analysis of electronic charting practices

Ethical and legal studies into encrypting and storing personal health data

Inequities within healthcare access

Many barriers inhibit people from accessing the quality medical care they need. These issues result in health disparities and injustices. 

Examples of research topics about health inequities include:

The impact of social determinants of health in a set population

Early and late-stage cancer stage diagnosis in urban vs. rural populations

Affordability of life-saving medications

Health insurance limitations and their impact on overall health

Diagnostic and treatment rates across ethnicities

People who belong to an ethnic minority are more likely to experience barriers and restrictions when trying to receive quality medical care. This is due to systemic healthcare racism and bias. 

As a result, diagnostic and treatment rates in minority populations are a hot-button field of research. Examples of ethnicity-based research topics include:

Cancer biopsy rates in BIPOC women

The prevalence of diabetes in Indigenous communities

Access inequalities in women’s health preventative screenings

The prevalence of undiagnosed hypertension in Black populations

• Pharmaceutical research topics

Large pharmaceutical companies are incredibly interested in investing in research to learn more about potential cures and treatments for diseases. 

If you’re interested in building a career in pharmaceutical research, here are a few examples of in-demand research topics:

Cancer treatment options

Clinical research is in high demand as pharmaceutical companies explore novel cancer treatment options outside of chemotherapy and radiation. 

Examples of cancer treatment-related research topics include:

Stem cell therapy for cancer

Oncogenic gene dysregulation and its impact on disease

Cancer-causing viral agents and their risks

Treatment efficacy based on early vs. late-stage cancer diagnosis

Cancer vaccines and targeted therapies

Immunotherapy for cancer

Pain medication alternatives

Historically, opioid medications were the primary treatment for short- and long-term pain. But, with the opioid epidemic getting worse, the need for alternative pain medications has never been more urgent. 

Examples of pain medication-related research topics include:

Opioid withdrawal symptoms and risks

Early signs of pain medication misuse

Anti-inflammatory medications for pain control

• Identify trends in your medical research with Dovetail

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As your go-to resource for speedy and accurate data analysis , we are proud to partner with healthcare researchers to innovate and improve the future of healthcare.

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Explore the Best Medical and Health Research Topics Ideas

Table of contents

• 1 How to Choose Medical Research Paper Topics
• 2 New Medical Research Paper Topics
• 3 Medical Research Topics for College Students
• 4 Controversial Medical Topics for Research Paper
• 5 Health Research Topics
• 6 Medicine Research Topics
• 7 Healthcare Research Topics
• 8 Public Health Research Topics
• 9 Mental Health Research Paper Topics
• 10 Anatomy Research Topics
• 11 Biomedical Research Topics
• 12 Bioethics Research Topics
• 13 Cancer Research Topics
• 14 Clinical Research Topics
• 15 Critical Care Research Topics
• 16 Pediatric Research Topics
• 17 Dental Research Topics Ideas
• 18 Dermatology Research Topics
• 19 Primary Care Research Topics
• 20 Pharmaceutical Research Topics
• 21 Medical Anthropology Research Topics
• 22 Paramedic Research Paper Topics
• 23 Surgery Research Topics
• 24 Radiology Research Paper Topics
• 25 Anatomy and Physiology Research Paper Topics
• 26 Healthcare Management Research Paper Topics
• 27 Medical Ethics Research Paper Topics
• 28 Conclusion

In such a complex and broad field as medicine, writing an original and compelling research paper is a daunting task. From investigating public care concerns to cancer treatment studies, each student decides where his interests lie. Our goal is to help students find new angles to study and focus on relevant topics. With our resources, you can write an engaging and rigorous paper.

How to Choose Medical Research Paper Topics

Choosing good research paper topics is often more challenging than the writing process itself. You need to select a captivating subject matter that will grab the reader’s attention, showcase your knowledge of a specific field, help you progress in your studies, and perhaps even inspire future research.

To accomplish that, you need to start with brainstorming, followed by thorough research. Here are some great tips to follow:

• Pick an interesting topic – The key is to pick something that you find interesting, and yet make sure it’s not too general or too narrow. It should allow you to delve deep into the subject matter and show that you’re a professional who is ready to take on a challenge when it comes to your chosen field of medicine.
• Narrow down your focus – Once you have a list of potential topics, sift through recent medical research papers to get up-to-date with the latest trends, developments, and issues in medicine and healthcare. Check out textbooks, news articles, and other relevant sources for more information related to your potential topics. If a particular condition or disease interests you (perhaps something that drew you to a career in medicine), there’s your cue for narrowing down your topic.
• Pinpoint the “why,” “how,” and “what” – Whether you are looking into nutrition research paper topics , controversial medical topics, nursing research topics, or anything in-between, ask yourself why each of them is important. How could they contribute to the available medical studies, if any? What new information could they bring to improve the future of medicine? Asking these questions will help you pick the right medical research paper topic that suits you and helps you move forward and reach your aspirations.

To help you on that quest, we’ve compiled a list of topics that you could use or that might inspire you to come up with something unique. Let’s dive in.

New Medical Research Paper Topics

Are you interested in the newest and most interesting developments in medicine? We put hours of effort into identifying the current trends in health research so we could provide you with these examples of topics. Whether you hire a research paper writing service for students or write a paper by yourself, you need an appealing topic to focus on.

• Epidemics versus pandemics
• Child health care
• Medical humanitarian missions in the developing world
• Homoeopathic medicines – the placebo effect
• Virus infections – causes and treatment
• Is medical research on animals ethical
• Vaccination – dangers versus benefits
• Artificial tissues and organs
• Rare genetic diseases
• Brain injuries

Medical Research Topics for College Students

You don’t know where to start with your medical research paper? There are so many things you could write about that the greatest challenge is to narrow them down. This is why we decided to help.

• Antibiotics treatments
• Chronic diseases
• Palliative treatment
• Battling Alzheimer’s disease
• How modern lifestyle affects public health
• Professional diseases
• Sleep disorders
• Changes in physical and mental health due to aging
• Eating disorders
• Terminal diseases

Controversial Medical Topics for Research Paper

In healthcare, new discoveries can change people’s lives in the blink of an eye. This is also the reason why there are so many controversial topics in medicine, which involve anything from religion to ethics or social responsibility. Read on to discover our top controversial research topics.

• Implementing food standards
• Gluten allergy
• Assisted suicide for terminal patients
• Testing vaccines on animals – ethical concerns
• Moral responsibilities regarding cloning
• Marijuana legalization for medical purposes
• Abortion – medical approaches
• Vegan diets – benefits and dangers
• Increased life expectancy: a burden on the healthcare system?
• Circumcision effects

Health Research Topics

Students conducting health research struggle with finding good ideas related to their medical interests. If you want to write interesting college papers, you can select a good topic for our list.

• How environmental changes affect human health
• Deafness: communication disorders
• Household air pollution
• Diabetes – a public danger
• Coronaviruses
• Oral health assessment
• Tobacco and alcohol control
• Diseases caused by lack of physical exercise
• How urban pollution affects respiratory diseases
• Healthy diets

Medicine Research Topics

Regardless of the requirements in your research assignment, you can write about something that is both engaging and useful in your future career. Choose a topic from below.

• Causes for the increasing cancer cases
• Insulin resistance
• How terrorism affects mental health
• AIDS/HIV – latest developments
• Treating pregnant women versus non-pregnant women
• Latest innovations in medical instruments
• Genetic engineering
• Successful treatment of mental diseases
• Is autism a disease
• Natural coma versus artificial coma

Healthcare Research Topics

Healthcare research includes political and social aspects, besides medical. For college students who want to explore how medicine is affected by society’s values or principles, we provide examples of topics for papers. Select yours from the list below.

• Government investment in healthcare services in the EU versus the USA
• Inequalities in healthcare assistance and services
• Electronic health records systems – pros and cons
• Can asylums treat mental issues
• Health care for prison inmates
• Equipment for improving treatment of AIDS
• Correlation between economic development and health care services across countries
• Impact of smoking on organs
• Heart attacks – causes and effects
• Breast cancer – recent developments

Public Health Research Topics

For current examples of public health topics, browse our list. We provide only original, researchable examples for which you can easily find supporting data and evidence.

• Public versus private hospitals
• Health care professionals – management principles
• Surgery failures – who is responsible
• What legal responsibilities has the hospital administration
• Patient service quality in public versus private hospitals
• What benefits national health care systems have
• Estimated costs of cancer treatments
• Public health in developing countries
• Banning tobacco ads – importance for public health
• Government solutions to the anti-vaccine’s movement

Mental Health Research Paper Topics

Mental health is one of the most complex areas of medicine, where things are never as clear as with other medical issues. This increases the research potential of the field with plenty of topics left for debate.

• Causes of anxiety disorders
• Bulimia versus anorexia
• Childhood trauma
• Mental health public policies
• Postpartum Depression
• Posttraumatic Stress Disorder
• Seasonal Affective Disorder
• Schizophrenia

Anatomy Research Topics

Anatomy covers everything about the human body and how it works. If you find that intriguing and want to pay for medical research paper, start by selecting a topic.

• Chemotherapy: how it affects the body
• Thyroid glands – functions in the body
• Human endocrine system
• Heart diseases
• How does the human muscular system develop
• Lymphatic system – importance
• Investigating genetic diseases
• Digestive system

Biomedical Research Topics

Biology and medicine often work together. For the newest changes in the biomedical field, check our topics.

• Alzheimer’s disease – paths for treatment
• Vaccines and drug development in the treatment of Ebola
• Antibiotic resistance
• Biological effects caused by aging
• Air pollution effects on health
• Infectious disease past versus present
• Regenerative medicine
• Biomedical diagnostics
• Biomedical technology

Bioethics Research Topics

A controversial area of medicine, bioethics is where you get the chance to add personal input to a research topic and come up with new insights. You could consider these subjects.

• Organ donation
• Alternative or complementary medicine
• Assisted suicide or the right to die
• Artificial insemination or surrogacy
• Chemical and biological warfare
• Contraception
• Environmental bioethics
• In Vitro fertilization

Cancer Research Topics

Are you writing a paper related to cancer causes, diagnosis, treatment or effects? Look below for a hot topic that it’s easy to research and important for medical advance.

• Ability of immune system cells to fight cancer
• Computational oncology
• Metastasis affected by drug resistance
• Stem cells – applications for cancer treatment
• Tumor microenvironment
• Obesity and age in cancer occurrence
• Early cancer detection – benefits
• Artificial intelligence predicting cancer
• Hematologic malignancies
• Pathogen-related cancers

Clinical Research Topics

Learn more about clinical medicine by conducting more in-depth research. We prepared for you a list of relevant issues to touch upon.

• Ethical concerns regarding research on human subjects
• Subject recruitment
• Budget preparation
• Human subject protection
• Clinical trials – financial support
• Clinical practices for health professionals
• Using vulnerable populations in clinical research
• Quality assurance in clinical research
• Academic clinical trials versus clinical trials units
• Data collection and management

Critical Care Research Topics

Critical care is a key area in medical studies. Explore these topics in your research paper to gain more valuable knowledge in this field. You can also get in contact with nursing research paper writers .

• Obesity and asthma – clinical manifestations
• Chronic obstructive pulmonary disease
• Rhythm analysis for cardiac arrest
• Traumatic brain injury – fluid resuscitation
• Hydrocortisone for multiple trauma patients
• Care and nutrition for critically ill adults
• Diagnosis of hypersensitivity pneumonitis
• Coma and sedation scales
• Artificial airways suctioning
• Arterial puncture and arterial line

Pediatric Research Topics

Any topic that refers to health care for children, pregnant women, mothers, and adolescents goes under pediatric care.

• Attention deficit hyperactivity disorder (ADHD)
• Congenital heart disease in newborns
• Adolescent medicine
• Neonatal medicine
• Rare diseases in children and teenagers
• Obesity and weight fluctuations
• Behavioral sleep problems in children
• Children with anemia

Dental Research Topics Ideas

Choose a topic on oral health or dental care from this list of the most interesting topics in the field.

• How smoking affects oral health
• Children’s risk for dental caries
• Dental anxiety
• Types of dental materials – new advances
• Bad breath bacteria
• How diabetes affects oral health
• Oral cancer
• Dental pain – types, causes
• Dental implants
• Oral health-related quality of life

Dermatology Research Topics

Find the best research topic for your dermatology paper among our examples.

• Atopic dermatitis
• Contact dermatitis
• Epidemiology behind uncommon skin disorders
• Cutaneous aging
• Risk factors of melanoma skin cancer
• Acne versus rosacea
• Genetic testing for skin conditions
• Effects of cosmetic agents on skin health
• Improving skin barrier with pharmaceutical agents
• Skin manifestations of autoimmune disorders

Primary Care Research Topics

Write a primary care paper that can demonstrate your research skills and interest in powerful scientific findings.

• Primary care for vulnerable/uninsured populations
• Interpersonal continuity in care treatment
• How primary care contributes to health systems
• Primary care delivery models
• Developments in family medicine
• Occupational/environmental health
• Pharmacotherapy approaches
• Formal allergy testing
• Oral contraception side effects
• Dietary or behavioral interventions for obesity management

Pharmaceutical Research Topics

Pharma students who need paper topics can use one from our list. We include all things related to pharmacy life.

• Drugs that can treat cancer
• Drug excretion
• Elimination rate constant
• Inflammatory stress drug treatment
• Aspirin poising
• Ibuprofen – dangers versus benefits
• Toxicodynamics
• Opioid use disorder
• Pharmacotherapy for schizophrenia
• Ketamine in depression treatment

Medical Anthropology Research Topics

Medical anthropology unites different areas of human knowledge. Find powerful ideas for a paper below.

• Cultural contexts regarding reproductive health
• Women sexuality
• Anthropological aspects of health care
• Contributions of social sciences to public health
• Euthanasia and medical ethics across cultures
• Health-related behavior in adults across cultures
• Transcultural nursing
• Forensic psychiatry
• Symptoms of Celiac Disease – a disease with no symptoms
• Nursing ethics

Paramedic Research Paper Topics

Topics for paramedic research must be based on evidence, data, statistics, or practical experience. Just like ours.

• Trends and statistics in EMS
• Disaster medicine
• Mass casualties
• Pandemics and epidemics
• Infection control
• Basic versus advanced life support
• Scene safety in EMS
• Shock management
• Motor vehicle accidents

Surgery Research Topics

Discover all the intricacies of surgeries that save lives by writing about our topics.

• Medical malpractice and legal issues
• Methicillin-resistant Staphylococcus aureus
• Pain management
• Perioperative nursing
• Wound management
• Colorectal cancer surgery
• Breast cancer surgery
• Minimally invasive surgeries
• Vascular disease

Radiology Research Paper Topics

Find a radiology topic related to your academic interests to write a successful paper.

• Using MRI to diagnose hepatic focal lesions
• Multidetector computer tomography
• Ultrasound elastography in breast cancer
• Assessing traumatic spinal cord injuries with MRI diffusion tensor imaging
• Sonographic imaging to detect male infertility
• Role of tomography in diagnosing cancer
• Brain tumor surgery with magnetic resonance imaging
• Bacterial meningitis imaging

Anatomy and Physiology Research Paper Topics

Any ideas for a medical research paper? We have included the most important topics for an anatomy and physiology paper.

• What role has the endocrine system
• Staphylococcus aureus
• Environmental factors that affect development of human muscular system
• What role has the lymphatic system
• An investigation of genetic diseases
• Explaining the aging process
• The digestive tract
• Effects of stress on cells and muscles
• Evolution of the human nervous system
• What role has the cardiovascular system

Healthcare Management Research Paper Topics

There are numerous topics you could write about when it comes to healthcare management. There’s a wide range of options to pick, from infrastructure, staff, and financial management to HR and patient management. Here are some of the top healthcare management research paper options.

• Medical talent acquisition and retention
• Best methods for enhancing preventative care measures
• The role of telemedicine in reinventing healthcare management
• Patient care and the ability to pay for healthcare
• Mid-level healthcare providers in the emergency department
• The opioid crisis: policies and programs
• Urgent care and walk-in clinics
• Hospital emergency management plan during an epidemic
• Hospital records management and patient privacy
• Financial crises: challenges and opportunities

Medical Ethics Research Paper Topics

Medical ethics is a field that opens the door to numerous compelling topics for research papers. Here are some of the most appealing ones you could tackle.

• Clinical research on humans
• Vaccines and immunization
• Religious beliefs in healthcare
• Euthanasia and physician-assisted suicide
• Ethical issues across cultures
• Amniocentesis or prenatal birth defect testing
• Medical malpractice and going back to work
• Racial and ethnic preferences and perceptions in organ donations
• Racial and ethnic disparities in healthcare
• Ethical concerns of AI in healthcare

If you need further assistance with your medical research paper, PapersOwl is here for you. Our expert writers can provide you with top-notch research and help you write an impressive paper. Contact us anytime, pick your writer, tell them more about your topic, and get a unique, plagiarism-free research paper with impeccable grammar and formatting.

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Research Topics & Ideas: Healthcare

100+ Healthcare Research Topic Ideas To Fast-Track Your Project

Finding and choosing a strong research topic is the critical first step when it comes to crafting a high-quality dissertation, thesis or research project. If you’ve landed on this post, chances are you’re looking for a healthcare-related research topic , but aren’t sure where to start. Here, we’ll explore a variety of healthcare-related research ideas and topic thought-starters across a range of healthcare fields, including allopathic and alternative medicine, dentistry, physical therapy, optometry, pharmacology and public health.

NB – This is just the start…

The topic ideation and evaluation process has multiple steps . In this post, we’ll kickstart the process by sharing some research topic ideas within the healthcare domain. This is the starting point, but to develop a well-defined research topic, you’ll need to identify a clear and convincing research gap , along with a well-justified plan of action to fill that gap.

If you’re new to the oftentimes perplexing world of research, or if this is your first time undertaking a formal academic research project, be sure to check out our free dissertation mini-course. In it, we cover the process of writing a dissertation or thesis from start to end. Be sure to also sign up for our free webinar that explores how to find a high-quality research topic.

Overview: Healthcare Research Topics

• Allopathic medicine
• Alternative /complementary medicine
• Veterinary medicine
• Physical therapy/ rehab
• Optometry and ophthalmology
• Pharmacy and pharmacology
• Public health
• Examples of healthcare-related dissertations

Allopathic (Conventional) Medicine

• The effectiveness of telemedicine in remote elderly patient care
• The impact of stress on the immune system of cancer patients
• The effects of a plant-based diet on chronic diseases such as diabetes
• The use of AI in early cancer diagnosis and treatment
• The role of the gut microbiome in mental health conditions such as depression and anxiety
• The efficacy of mindfulness meditation in reducing chronic pain: A systematic review
• The benefits and drawbacks of electronic health records in a developing country
• The effects of environmental pollution on breast milk quality
• The use of personalized medicine in treating genetic disorders
• The impact of social determinants of health on chronic diseases in Asia
• The role of high-intensity interval training in improving cardiovascular health
• The efficacy of using probiotics for gut health in pregnant women
• The impact of poor sleep on the treatment of chronic illnesses
• The role of inflammation in the development of chronic diseases such as lupus
• The effectiveness of physiotherapy in pain control post-surgery

Topics & Ideas: Alternative Medicine

• The benefits of herbal medicine in treating young asthma patients
• The use of acupuncture in treating infertility in women over 40 years of age
• The effectiveness of homoeopathy in treating mental health disorders: A systematic review
• The role of aromatherapy in reducing stress and anxiety post-surgery
• The impact of mindfulness meditation on reducing high blood pressure
• The use of chiropractic therapy in treating back pain of pregnant women
• The efficacy of traditional Chinese medicine such as Shun-Qi-Tong-Xie (SQTX) in treating digestive disorders in China
• The impact of yoga on physical and mental health in adolescents
• The benefits of hydrotherapy in treating musculoskeletal disorders such as tendinitis
• The role of Reiki in promoting healing and relaxation post birth
• The effectiveness of naturopathy in treating skin conditions such as eczema
• The use of deep tissue massage therapy in reducing chronic pain in amputees
• The impact of tai chi on the treatment of anxiety and depression
• The benefits of reflexology in treating stress, anxiety and chronic fatigue
• The role of acupuncture in the prophylactic management of headaches and migraines

Topics & Ideas: Dentistry

• The impact of sugar consumption on the oral health of infants
• The use of digital dentistry in improving patient care: A systematic review
• The efficacy of orthodontic treatments in correcting bite problems in adults
• The role of dental hygiene in preventing gum disease in patients with dental bridges
• The impact of smoking on oral health and tobacco cessation support from UK dentists
• The benefits of dental implants in restoring missing teeth in adolescents
• The use of lasers in dental procedures such as root canals
• The efficacy of root canal treatment using high-frequency electric pulses in saving infected teeth
• The role of fluoride in promoting remineralization and slowing down demineralization
• The impact of stress-induced reflux on oral health
• The benefits of dental crowns in restoring damaged teeth in elderly patients
• The use of sedation dentistry in managing dental anxiety in children
• The efficacy of teeth whitening treatments in improving dental aesthetics in patients with braces
• The role of orthodontic appliances in improving well-being
• The impact of periodontal disease on overall health and chronic illnesses

Tops & Ideas: Veterinary Medicine

• The impact of nutrition on broiler chicken production
• The role of vaccines in disease prevention in horses
• The importance of parasite control in animal health in piggeries
• The impact of animal behaviour on welfare in the dairy industry
• The effects of environmental pollution on the health of cattle
• The role of veterinary technology such as MRI in animal care
• The importance of pain management in post-surgery health outcomes
• The impact of genetics on animal health and disease in layer chickens
• The effectiveness of alternative therapies in veterinary medicine: A systematic review
• The role of veterinary medicine in public health: A case study of the COVID-19 pandemic
• The impact of climate change on animal health and infectious diseases in animals
• The importance of animal welfare in veterinary medicine and sustainable agriculture
• The effects of the human-animal bond on canine health
• The role of veterinary medicine in conservation efforts: A case study of Rhinoceros poaching in Africa
• The impact of veterinary research of new vaccines on animal health

Topics & Ideas: Physical Therapy/Rehab

• The efficacy of aquatic therapy in improving joint mobility and strength in polio patients
• The impact of telerehabilitation on patient outcomes in Germany
• The effect of kinesiotaping on reducing knee pain and improving function in individuals with chronic pain
• A comparison of manual therapy and yoga exercise therapy in the management of low back pain
• The use of wearable technology in physical rehabilitation and the impact on patient adherence to a rehabilitation plan
• The impact of mindfulness-based interventions in physical therapy in adolescents
• The effects of resistance training on individuals with Parkinson’s disease
• The role of hydrotherapy in the management of fibromyalgia
• The impact of cognitive-behavioural therapy in physical rehabilitation for individuals with chronic pain
• The use of virtual reality in physical rehabilitation of sports injuries
• The effects of electrical stimulation on muscle function and strength in athletes
• The role of physical therapy in the management of stroke recovery: A systematic review
• The impact of pilates on mental health in individuals with depression
• The use of thermal modalities in physical therapy and its effectiveness in reducing pain and inflammation
• The effect of strength training on balance and gait in elderly patients

Topics & Ideas: Optometry & Opthalmology

• The impact of screen time on the vision and ocular health of children under the age of 5
• The effects of blue light exposure from digital devices on ocular health
• The role of dietary interventions, such as the intake of whole grains, in the management of age-related macular degeneration
• The use of telemedicine in optometry and ophthalmology in the UK
• The impact of myopia control interventions on African American children’s vision
• The use of contact lenses in the management of dry eye syndrome: different treatment options
• The effects of visual rehabilitation in individuals with traumatic brain injury
• The role of low vision rehabilitation in individuals with age-related vision loss: challenges and solutions
• The impact of environmental air pollution on ocular health
• The effectiveness of orthokeratology in myopia control compared to contact lenses
• The role of dietary supplements, such as omega-3 fatty acids, in ocular health
• The effects of ultraviolet radiation exposure from tanning beds on ocular health
• The impact of computer vision syndrome on long-term visual function
• The use of novel diagnostic tools in optometry and ophthalmology in developing countries
• The effects of virtual reality on visual perception and ocular health: an examination of dry eye syndrome and neurologic symptoms

Topics & Ideas: Pharmacy & Pharmacology

• The impact of medication adherence on patient outcomes in cystic fibrosis
• The use of personalized medicine in the management of chronic diseases such as Alzheimer’s disease
• The effects of pharmacogenomics on drug response and toxicity in cancer patients
• The role of pharmacists in the management of chronic pain in primary care
• The impact of drug-drug interactions on patient mental health outcomes
• The use of telepharmacy in healthcare: Present status and future potential
• The effects of herbal and dietary supplements on drug efficacy and toxicity
• The role of pharmacists in the management of type 1 diabetes
• The impact of medication errors on patient outcomes and satisfaction
• The use of technology in medication management in the USA
• The effects of smoking on drug metabolism and pharmacokinetics: A case study of clozapine
• Leveraging the role of pharmacists in preventing and managing opioid use disorder
• The impact of the opioid epidemic on public health in a developing country
• The use of biosimilars in the management of the skin condition psoriasis
• The effects of the Affordable Care Act on medication utilization and patient outcomes in African Americans

Topics & Ideas: Public Health

• The impact of the built environment and urbanisation on physical activity and obesity
• The effects of food insecurity on health outcomes in Zimbabwe
• The role of community-based participatory research in addressing health disparities
• The impact of social determinants of health, such as racism, on population health
• The effects of heat waves on public health
• The role of telehealth in addressing healthcare access and equity in South America
• The impact of gun violence on public health in South Africa
• The effects of chlorofluorocarbons air pollution on respiratory health
• The role of public health interventions in reducing health disparities in the USA
• The impact of the United States Affordable Care Act on access to healthcare and health outcomes
• The effects of water insecurity on health outcomes in the Middle East
• The role of community health workers in addressing healthcare access and equity in low-income countries
• The impact of mass incarceration on public health and behavioural health of a community
• The effects of floods on public health and healthcare systems
• The role of social media in public health communication and behaviour change in adolescents

Examples: Healthcare Dissertation & Theses

While the ideas we’ve presented above are a decent starting point for finding a healthcare-related research topic, they are fairly generic and non-specific. So, it helps to look at actual dissertations and theses to see how this all comes together.

Below, we’ve included a selection of research projects from various healthcare-related degree programs to help refine your thinking. These are actual dissertations and theses, written as part of Master’s and PhD-level programs, so they can provide some useful insight as to what a research topic looks like in practice.

• Improving Follow-Up Care for Homeless Populations in North County San Diego (Sanchez, 2021)
• On the Incentives of Medicare’s Hospital Reimbursement and an Examination of Exchangeability (Elzinga, 2016)
• Managing the healthcare crisis: the career narratives of nurses (Krueger, 2021)
• Methods for preventing central line-associated bloodstream infection in pediatric haematology-oncology patients: A systematic literature review (Balkan, 2020)
• Farms in Healthcare: Enhancing Knowledge, Sharing, and Collaboration (Garramone, 2019)
• When machine learning meets healthcare: towards knowledge incorporation in multimodal healthcare analytics (Yuan, 2020)
• Integrated behavioural healthcare: The future of rural mental health (Fox, 2019)
• Healthcare service use patterns among autistic adults: A systematic review with narrative synthesis (Gilmore, 2021)
• Mindfulness-Based Interventions: Combatting Burnout and Compassionate Fatigue among Mental Health Caregivers (Lundquist, 2022)
• Transgender and gender-diverse people’s perceptions of gender-inclusive healthcare access and associated hope for the future (Wille, 2021)
• Efficient Neural Network Synthesis and Its Application in Smart Healthcare (Hassantabar, 2022)
• The Experience of Female Veterans and Health-Seeking Behaviors (Switzer, 2022)
• Machine learning applications towards risk prediction and cost forecasting in healthcare (Singh, 2022)
• Does Variation in the Nursing Home Inspection Process Explain Disparity in Regulatory Outcomes? (Fox, 2020)

Looking at these titles, you can probably pick up that the research topics here are quite specific and narrowly-focused , compared to the generic ones presented earlier. This is an important thing to keep in mind as you develop your own research topic. That is to say, to create a top-notch research topic, you must be precise and target a specific context with specific variables of interest . In other words, you need to identify a clear, well-justified research gap.

Need more help?

If you’re still feeling a bit unsure about how to find a research topic for your healthcare dissertation or thesis, check out Topic Kickstarter service below.

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15 Comments

I need topics that will match the Msc program am running in healthcare research please

Hello Mabel,

I can help you with a good topic, kindly provide your email let’s have a good discussion on this.

Can you provide some research topics and ideas on Immunology?

Thank you to create new knowledge on research problem verse research topic

Help on problem statement on teen pregnancy

This post might be useful: https://gradcoach.com/research-problem-statement/

can you provide me with a research topic on healthcare related topics to a qqi level 5 student

Please can someone help me with research topics in public health ?

Hello I have requirement of Health related latest research issue/topics for my social media speeches. If possible pls share health issues , diagnosis, treatment.

I would like a topic thought around first-line support for Gender-Based Violence for survivors or one related to prevention of Gender-Based Violence

Please can I be helped with a master’s research topic in either chemical pathology or hematology or immunology? thanks

Can u please provide me with a research topic on occupational health and safety at the health sector

Good day kindly help provide me with Ph.D. Public health topics on Reproductive and Maternal Health, interventional studies on Health Education

may you assist me with a good easy healthcare administration study topic

May you assist me in finding a research topic on nutrition,physical activity and obesity. On the impact on children

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Examples of Research Questions

Phd in nursing science program, examples of broad clinical research questions include:.

• Does the administration of pain medication at time of surgical incision reduce the need for pain medication twenty-four hours after surgery?
• What maternal factors are associated with obesity in toddlers?
• What elements of a peer support intervention prevent suicide in high school females?
• What is the most accurate and comprehensive way to determine men’s experience of physical assault?
• Is yoga as effective as traditional physical therapy in reducing lymphedema in patients who have had head and neck cancer treatment?
• In the third stage of labor, what is the effect of cord cutting within the first three minutes on placenta separation?
• Do teenagers with Type 1 diabetes who receive phone tweet reminders maintain lower blood sugars than those who do not?
• Do the elderly diagnosed with dementia experience pain?
•  How can siblings’ risk of depression be predicted after the death of a child?
•  How can cachexia be prevented in cancer patients receiving aggressive protocols involving radiation and chemotherapy?

Examples of some general health services research questions are:

• Does the organization of renal transplant nurse coordinators’ responsibilities influence live donor rates?
• What activities of nurse managers are associated with nurse turnover?  30 day readmission rates?
• What effect does the Nurse Faculty Loan program have on the nurse researcher workforce?  What effect would a 20% decrease in funds have?
• How do psychiatric hospital unit designs influence the incidence of patients’ aggression?
• What are Native American patient preferences regarding the timing, location and costs for weight management counseling and how will meeting these preferences influence participation?
•  What predicts registered nurse retention in the US Army?
• How, if at all, are the timing and location of suicide prevention appointments linked to veterans‘ suicide rates?
• What predicts the sustainability of quality improvement programs in operating rooms?
• Do integrated computerized nursing records across points of care improve patient outcomes?
• How many nurse practitioners will the US need in 2020?

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• 1. Assemble Your Team

2. Develop a Research Question

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• 8. Write the Review
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A well-developed and answerable question is the foundation for any systematic review. This process involves:

• Systematic review questions typically follow a PICO-format (patient or population, intervention, comparison, and outcome)
• Using the PICO framework can help team members clarify and refine the scope of their question. For example, if the population is breast cancer patients, is it all breast cancer patients or just a segment of them? 
• When formulating your research question, you should also consider how it could be answered. If it is not possible to answer your question (the research would be unethical, for example), you'll need to reconsider what you're asking
• Typically, systematic review protocols include a list of studies that will be included in the review. These studies, known as exemplars, guide the search development but also serve as proof of concept that your question is answerable. If you are unable to find studies to include, you may need to reconsider your question

Other Question Frameworks

PICO is a helpful framework for clinical research questions, but may not be the best for other types of research questions. Did you know there are at least  25 other question frameworks  besides variations of PICO?  Frameworks like PEO, SPIDER, SPICE, and ECLIPS can help you formulate a focused research question. The table and example below were created by the  Medical University of South Carolina (MUSC) Libraries .

The PEO question framework is useful for qualitative research topics. PEO questions identify three concepts: population, exposure, and outcome. Research question : What are the daily living experiences of mothers with postnatal depression?

The SPIDER question framework is useful for qualitative or mixed methods research topics focused on "samples" rather than populations. SPIDER questions identify five concepts: sample, phenomenon of interest, design, evaluation, and research type.

Research question : What are the experiences of young parents in attendance at antenatal education classes?

The SPICE question framework is useful for qualitative research topics evaluating the outcomes of a service, project, or intervention. SPICE questions identify five concepts: setting, perspective, intervention/exposure/interest, comparison, and evaluation.

Research question : For teenagers in South Carolina, what is the effect of provision of Quit Kits to support smoking cessation on number of successful attempts to give up smoking compared to no support ("cold turkey")?

The ECLIPSE framework is useful for qualitative research topics investigating the outcomes of a policy or service. ECLIPSE questions identify six concepts: expectation, client group, location, impact, professionals, and service.

Research question:  How can I increase access to wireless internet for hospital patients?

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Health Humanities: Primary and Secondary Sources: Research Question

• Research Question
• Primary Sources
• Secondary Sources
• Wheel of Sources

How to Develop your Research Question?

What is a research question? A research question is a question around which you center your research. It should be:

• clear : it provides enough specifics that one’s audience can easily understand its purpose without needing additional explanation.
• focused : it is narrow enough that it can be answered thoroughly in the space the writing task allows.
• concise : it is expressed in the fewest possible words.
• complex : it is not answerable with a simple “yes” or “no,” but rather requires synthesis and analysis of ideas and sources prior to the composition of an answer.
• arguable : its potential answers are open to debate rather than accepted facts.

You should ask a question about an issue that you are genuinely curious and/or passionate about.

The question you ask should be developed for the discipline you are studying. A question appropriate for Biology, for instance, is different from an appropriate one in Political Science or Sociology. If you are developing your question for a course other than the first-year composition, you may want to discuss your ideas for a research question with your professor.

Why is a Research Question Essential?

Why is a research question essential to the research process? Research questions help writers focus their research by providing a path through the research and writing process. The specificity of a well-developed research question helps writers avoid the “all-about” paper and work toward supporting a specific, arguable thesis.

Steps to developing a research question:

• Choose an interesting general topic.  Most professional researchers focus on topics they are genuinely interested in studying. Writers should choose a broad topic about which they genuinely would like to know more. An example of a general topic might be “Slavery in the American South” or “Films of the 1930s.”
• Do some preliminary research on your general topic.  Do a few quick searches in current periodicals and journals on your topic to see what’s already been done and to help you narrow your focus. What issues are scholars and researchers discussing, when it comes to your topic? What questions occur to you as you read these articles?
• Consider your audience.  For most college papers, your audience will be academic, but always keep your audience in mind when narrowing your topic and developing your question. Would that particular audience be interested in the question you are developing?
• Start asking questions.  Taking into consideration all of the above, start asking yourself open-ended “how” and “why” questions about your general topic. For example, “Why were slave narratives effective tools in working toward the abolishment of slavery?” or “How did the films of the 1930s reflect or respond to the conditions of the Great Depression?”
• Is your research question clear?  With so much research available on any given topic, research questions must be as clear as possible in order to be effective in helping the writer direct his or her research.
• Is your research question focused?  Research questions must be specific enough to be well covered in the space available.
• Is your research question complex?  Research questions should not be answerable with a simple “yes” or “no” or by easily-found facts.  They should, instead, require both research and analysis on the part of the writer. They often begin with “How” or “Why.”
• Begin your research .  After you’ve come up with a question, think about the possible paths your research could take. What sources should you consult as you seek answers to your question? What research process will ensure that you find a variety of perspectives and responses to your question?

Sample Research Questions

Unclear:  How should social networking sites address the harm they cause? Clear:  What action should social networking sites like Instagram and Facebook take to protect users’ personal information and privacy? The unclear version of this question doesn’t specify which social networking sites or suggest what kind of harm the sites might be causing. It also assumes that this “harm” is proven and/or accepted. The clearer version specifies sites (Instagram and Facebook), the type of potential harm (privacy issues), and who may be experiencing that harm (users). A strong research question should never leave room for ambiguity or interpretation. Unfocused:   What is the effect on the environment from global warming? Focused:   What is the most significant effect of glacial melting on the lives of penguins in Antarctica?

The unfocused research question is so broad that it couldn’t be adequately answered in a book-length piece, let alone a standard college-level paper. The focused version narrows down to a specific effect of global warming (glacial melting), a specific place (Antarctica), and a specific animal that is affected (penguins). It also requires the writer to take a stance on which effect has the greatest impact on the affected animal. When in doubt, make a research question as narrow and focused as possible.

Too simple:   How are doctors addressing diabetes in the U.S.? Appropriately Complex:    What main environmental, behavioral, and genetic factors predict whether Americans will develop diabetes, and how can these commonalities be used to aid the medical community in the prevention of the disease?

The simple version of this question can be looked up online and answered in a few factual sentences; it leaves no room for analysis. The more complex version is written in two parts; it is thought-provoking and requires both significant investigation and evaluation from the writer. As a general rule of thumb, if a quick Google search can answer a research question, it’s likely not very effective.

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• Last Updated: Apr 28, 2023 3:46 PM
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• Open access
• Published: 26 November 2018

The 150 most important questions in cancer research and clinical oncology series: questions 94–101

Edited by Cancer Communications

Cancer Communications

Cancer Communications volume  38 , Article number:  69 ( 2018 ) Cite this article

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Since the beginning of 2017, Cancer Communications (former title: Chinese Journal of Cancer ) has published a series of important questions regarding cancer research and clinical oncology, to provide an enhanced stimulus for cancer research, and to accelerate collaborations between institutions and investigators. In this edition, the following 8 valuable questions are presented. Question 94. The origin of tumors: time for a new paradigm? Question 95. How can we accelerate the identification of biomarkers for the early detection of pancreatic ductal adenocarcinoma? Question 96. Can we improve the treatment outcomes of metastatic pancreatic ductal adenocarcinoma through precision medicine guided by a combination of the genetic and proteomic information of the tumor? Question 97. What are the parameters that determine a competent immune system that gives a complete response to cancers after immune induction? Question 98. Is high local concentration of metformin essential for its anti-cancer activity? Question 99. How can we monitor the emergence of cancer cells anywhere in the body through plasma testing? Question 100. Can phytochemicals be more specific and efficient at targeting P-glycoproteins to overcome multi-drug resistance in cancer cells? Question 101. Is cell migration a selectable trait in the natural evolution of carcinoma?

Until now, the battle against cancer is still ongoing, but there are also ongoing discoveries being made. Milestones in cancer research and treatments are being achieved every year; at a quicker pace, as compared to decades ago. Likewise, some cancers that were considered incurable are now partly curable, lives that could not be saved are now being saved, and for those with yet little options, they are now having best-supporting care. With an objective to promote worldwide cancer research and even accelerate inter-countries collaborations, since the beginning of 2017, Cancer Communications (former title: Chinese Journal of Cancer ) has launched a program of publishing 150 most important questions in cancer research and clinical oncology [ 1 ]. We are providing a platform for researchers to freely voice-out their novel ideas, and propositions to enhance the communications on how and where our focus should be placed [ 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 , 11 , 12 , 13 ]. In this edition, 8 valuable and inspiring questions, Question 94–101, from highly distinguished professionals from different parts of the world are presented. If you have any novel proposition(s) and Question(s), please feel free to contact Ms. Ji Ruan via email: [email protected].

Question 94: The origin of tumors: time for a new paradigm?

Background and implications.

“There is no worse blind man than the one who doesn’t want to see. There is no worse deaf man than the one who doesn’t want to hear. And there is no worse madman than the one who doesn’t want to understand.” —Ancient Proverb

In the past half-century, cancer biologists have focused on a dogma in which cancer was viewed as a proliferative disease due to mechanisms that activate genes (oncogenes) to promote cell proliferation or inactivate genes (tumor suppressor genes) to suppress tumor growth. In retrospect, these concepts were established based on functional selections, by using tissue culture (largely mouse NIH 3T3 cells) for the selection of transformed foci at the time when we knew virtually nothing about the human genome [ 14 ]. However, it is very difficult to use these genes individually or in combinations to transform primary human cells. Further, the simplified view of uncontrolled proliferation cannot explain the tumor as being a malignant organ or a teratoma, as observed by pathologists over centuries. Recently, the cancer genomic atlas project has revealed a wide variety of genetic alterations ranging from no mutation to multiple chromosomal deletions or fragmentations, which make the identification of cancer driver mutations very challenging in a background of such a massive genomic rearrangement. Paradoxically, this increase the evidences demonstrating that the oncogenic mutations are commonly found in many normal tissues, further challenging the dogma that genetic alteration is the primary driver of this disease.

Logically, the birth of a tumor should undergo an embryonic-like development at the beginning, similar to that of a human. However, the nature of such somatic-derived early embryo has been elusive. Recently, we provided evidence to show that polyploid giant cancer cells (PGCCs), which have been previously considered non-dividing, are actually capable of self-renewal, generating viable daughter cells via amitotic budding, splitting and burst, and capable of acquisition of embryonic-like stemness [ 15 , 16 , 17 ]. The mode of PGCC division is remarkably similar to that of blastomere, a first step in human embryogenesis following fertilization. The blastomere nucleus continuously divides 4–5 times without cytoplasmic division to generate 16–32 cells and then to form compaction/morulae before developing into a blastocyst [ 18 ]. Based on these data and similarity to the earliest stage of human embryogenesis, I propose a new theory that tumor initiation can be achieved via a dualistic origin, similar to the first step of human embryogenesis via the formation of blastomere-like cells, i.e. the activation of blastomere or blastomere-like cells which leads to the dedifferentiation of germ cells or somatic cells, respectively, which is then followed by the differentiation to generate their respective stem cells, and the differentiation arrest at a specific developmental hierarchy leading to tumor initiation [ 19 ]. The somatic-derived blastomere-like cancer stem cell follows its own mode of cell growth and division and is named as the giant cell cycle. This cycle includes four distinct but overlapping phases: the initiation, self-renewal, termination, and stability phases. The giant cell cycle can be tracked in vitro and in vivo due to their salient giant cell morphology (Fig.  1 ).

One mononucleated polyploid giant cancer cell (PGCC) in the background of regular size diploid cancer cells. The PGCC can be seen to be at least 100 times larger than that of regular cancer cells

This new theory challenges the traditional paradigm that cancer is a proliferative disease, and proposes that the initiation of cancer requires blastomere-like division that is similar to that of humans before achieving stable proliferation at specific developmental hierarchy in at least half of all human cancers. This question calls for all investigators in the cancer research community to investigate the role of PGCCs in the initiation, progression, resistance, and metastasis of cancer and to look for novel agents to block the different stages of the giant cell cycle.

The histopathology (phenotype) of cancers has been there all the time. It is just the theory of cancer origin proposed by scientists that changes from time to time. After all, trillions of dollars have been invested in fighting this disease by basing on its genetic origin in the past half-century, yet, little insight has been gained [ 14 ]. Here are two quotes from Einstein: “Insanity: doing the same thing over and over again expecting different results”, and “We cannot solve our problems with the same thinking we used when created them”.

In short, it is time to change our mindset and to start pursuing PGCCs, which we can observe under the microscope. But with very little understanding about these cells, it is time for a shift in paradigm.

Jinsong Liu.

Affiliation

Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030-4095, USA.

Email address

[email protected]

Question 95: How can we accelerate the identification of biomarkers for the early detection of pancreatic ductal adenocarcinoma?

Pancreatic ductal adenocarcinoma (PDAC) is one of the most lethal cancers in the world with a dismal 5-year overall survival rate of less than 5%; which has not been significantly improved since the past decades. Although surgical resection is the only option for curative treatment of PDAC, only 15%–20% of patients with PDAC have the chance to undergo curative resection, leaving the rest with only palliative options in hope for increasing their quality of life; since they were already at unresectable and non-curative stages at their first diagnosis.

The lack of specific symptoms in the early-stage of PDAC is responsible for rendering an early diagnosis difficult. Therefore, more sensitive and specific screening methodologies for its early detection is urgently needed to improve its diagnosis, starting early treatments, and ameliorating prognoses. The diagnosis so far relies on imaging modalities such as abdominal ultrasound, computed tomography (CT), magnetic resonance imaging (MRI), endoscopic ultrasound (EUS), endoscopic retrograde cholangiopancreatography (ERCP), and positron emission tomography (PET). One may propose to screen for pancreatic cancer in high-risk populations, which is highly recommended, however screening intervention for all the people is not a wise choice; when considering the relatively low prevalence of PDAC, and the difficulty for diagnosing it in its early stage [ 20 ].

Therefore, alternative diagnostic tools for early detection of PDAC are highly expected. Among the biomarkers currently used in clinical practice, carbohydrate antigen 19–9 (CA19–9) is among the most useful one for supporting the diagnosis of PDAC, but it is neither sufficiently sensitive nor specific for its early detection. Yachida et al. reported in 2010 that the initiating mutation in the pancreas occurs approximately two decades before the PDAC to start growing in distant organs [ 21 ], which indicates a broad time of the window of opportunity for the early detection of PDAC. With the advancement in next-generation sequencing technology, the number of reported studies regarding novel potential molecular biomarkers in bodily fluids including the blood, feces, urine, saliva, and pancreatic juice for early detection of PDAC has been increasing. Such biomarkers may be susceptible to detect mutations at the genetic or epigenetic level, identifying important non-coding RNA (especially microRNA and long non-coding RNA), providing insights regarding the metabolic profiles, estimating the tumor level in liquid biopsies (circulating free DNA, circulating tumor cells and exosomes), and so on.

Another approach to identifying biomarkers for the early detection of pancreatic cancer is using animal models. In spontaneous animal models of pancreatic cancer, such as Kras-mutated mouse models, it is expected that by high throughput analyses of the genetic/epigenetic/proteomic alterations, some novel biomarkers might be able to be identified. For instance, Sharma et al. reported in 2017 that the detection of phosphatidylserine-positive exosomes enabled the diagnosis of early-stage malignancies in LSL-Kras G12D , Cdkn2a lox/lox : p48 Cre and LSL-Kras G12d/+ , LSL-Trp R172H/+ , and P48 Cre mice [ 22 ].

These analyses in clinical samples or animal models hold the clues for the early detection of PDAC, however, further studies are required to validate their diagnostic performance. What’s most important, will be the lining-up of these identified prospective biomarkers, to validate their sensitivities and specificities. This will determine their potential for widespread clinical applicability, and hopefully, accelerate the early diagnosis of PDAC.

Mikiya Takao 1,2 , Hirotaka Matsuo 2 , Junji Yamamoto 1 , and Nariyoshi Shinomiya 2 .

1 Department of Surgery, National Defense Medical College, 3-2 Namiki, Tokorozawa, Saitama 359-8513, Japan; 2 Department of Integrative Physiology and Bio-Nano Medicine, National Defense Medical College, 3-2 Namiki, Tokorozawa, Saitama 359-8513, Japan.

E-mail address

[email protected]; [email protected]; [email protected]; [email protected]

Question 96: Can we improve the treatment outcomes of metastatic pancreatic ductal adenocarcinoma through precision medicine guided by a combination of the genetic and proteomic information of the tumor?

Pancreatic ductal adenocarcinoma (PDAC) is one of the most malignant cancers, and nearly half of the patients had metastatic PDAC when they are initially diagnosed. When they are accompanied by metastatic tumors, unlike most solid cancer, PDAC cannot be cured with primary surgical resection alone [ 23 , 24 ]. Also, since PDAC has poor responses to conventional therapies, improvements in adjunctive treatment approach including chemo- and immuno-therapy are earnestly required. From this standpoint, recent results regarding the differences in the molecular evolution of pancreatic cancer subtypes provide a new insight into its therapeutic development [ 25 ], which may lead to the improvement of the prognosis of not only metastatic PDAC but also of locally advanced or recurrent PDAC.

In fact, new chemotherapeutic regimens such as the combination of gemcitabine with nab-paclitaxel and FOLFIRINOX have been reported to show improved prognosis despite a lack of examples of past successes in the treatment of patients with metastatic PDAC who had undergone R0 resection [ 26 ]. While many mutations including KRAS , CDKN2A , TP53, and SMAD4 are associated with pancreatic carcinogenesis, no effective molecular targeted drug has been introduced in the clinical setting so far. A recent report of a phase I/II study on refametinib, a MEK inhibitor, indicated that KRAS mutation status might affect the overall response rate, disease control rate, progression-free survival, and overall survival of PDAC in combination with gemcitabine [ 27 ].

While immunotherapy is expected to bring a great improvement in cancer treatment, until now, immune checkpoint inhibitors have achieved limited clinical benefit for patients with PDAC. This might be because PDAC creates a uniquely immunosuppressive tumor microenvironment, where tumor-associated immunosuppressive cells and accompanying desmoplastic stroma prevent the tumor cells from T cell infiltration. Recently reported studies have indicated that immunotherapy might be effective when combined with focal adhesion kinase (FAK) inhibitor [ 28 ] or IL-6 inhibitor [ 29 ], but more studies are required to validate their use in clinical practice.

As such, we believe that if the dynamic monitoring of drug sensitivity/resistance in the individual patients is coupled with precision treatment based on individualized genetics/epigenetics/proteomics alterations in the patients’ tumor, this could improve the treatment outcomes of PDAC.

Mikiya Takao 1,2 , Hirotaka Matsuo 2 , Junji Yamamoto 1 , and Nariyoshi Shinomiya 2.

Question 97: What are the parameters that determine a competent immune system that gives a complete response to cancers after immune induction?

Recently, cancer immunotherapy has shown great clinical benefit in multiple types of cancers [ 30 , 31 , 32 ]. It has provided new approaches for cancer treatment. However, it has been observed that only a fraction of patients respond to immunotherapy.

Much effort has been made to identify markers for immunotherapeutic response. Tumor mutation burden (TMB), mismatch repair (MMR) deficiency, PD-L1 expression, and tumor infiltration lymphocyte (TIL) have been found to be associated with an increased response rate in checkpoint blockade therapies. Unfortunately, a precise prediction is still challenging in this field. Moreover, when to stop the treatment of immunotherapy is an urgent question that remains to be elucidated.

In other words, there is no available approach to determine if a patient has generated a good immune response against the cancer after immunotherapy treatments. All of these indicate the complexity and challenges that reside for implementing novel man-induced cancer-effective immune response therapeutics. A variety of immune cells play collaborative roles at different stages to recognize antigens and eventually to generate an effective anti-cancer immune response. Given the high complexity of the immune system, a rational evaluation approach is needed to cover the whole process. Moreover, we need to perfect vaccine immunization and/or in vitro activation of T cells to augment the function of the immune system; particularly the formation of immune memory.

Edison Liu 1 , Penghui Zhou 2 , Jiang Li 2 .

1 The Jackson Laboratory, Bar Harbor, ME 04609, USA; 2 Sun Yat-sen University Cancer Center, Guangzhou, Guangdong 510060, P. R. China.

[email protected]; [email protected]; [email protected]

Question 98: Is high local concentration of metformin essential for its anti-cancer activity?

Metformin was approved as a first line of anti-diabetic drug since decades. Interestingly, the fact that clinical epidemiological studies have shown that metformin can reduce the risk of a variety of cancers stimulates considerable recognition to explore its anticancer activity.

Although the in vitro and in vivo experimental results have demonstrated that metformin can have some potential anti-tumor effects, more than 100 clinical trials did not achieve such desirable results [ 33 ]. We and others believe that the main problem resides in the prescribing doses used. For cancer treatment, a much higher dose may be needed for observing any anti-tumor activities, as compared to the doses prescribed for diabetics [ 34 , 35 , 36 ].

Further, if the traditional local/oral administration approach is favored, the prescribed metformin may not be at the required dose-concentration once it reaches the blood to have the effective anti-cancer activities. We, therefore, propose that intravesical instillation of metformin into the bladder lumen could be a promising way to treat for bladder cancer, at least. We have already obtained encouraging results both in vitro and in vivo experiments, including in an orthotopical bladder cancer model [ 36 , 37 ]. Now, we are waiting to observe its prospective clinical outcome.

Mei Peng 1 , Xiaoping Yang 2 .

1 Department of Pharmacy, Xiangya Hospital, Central South University. Changsha, Hunan 410083, P. R. China; 2 Key Laboratory of Study and Discovery of Small Targeted Molecules of Hunan Province, Department of Pharmacy, School of Medicine, Hunan Normal University, Changsha, Hunan 410013, P. R. China.

[email protected]; [email protected]

Question 99: How can we monitor the emergence of cancer cells anywhere in the body through plasma testing?

The early detection of cancer is still a relentless worldwide challenge. The sensitivity and specificity of traditional blood tumor markers and imaging technologies are still to be greatly improved. Hence, novel approaches for the early detection of cancer are urgently needed.

The emergence of liquid biopsy technologies opens a new driveway for solving such issues. According to the definition of the National Cancer Institute of the United States, a liquid biopsy is a test done on a sample of blood to look for tumorigenic cancer cells or pieces of tumor cells’ DNA that are circulating in the blood [ 38 ]. This definition implies two main types of the current liquid biopsy: one that detects circulating tumor cells and the other that detects non-cellular material in the blood, including tumor DNA, RNA, and exosomes.

Circulating tumor cells (CTCs) are referred to as tumor cells that have been shed from the primary tumor location and have found their way to the peripheral blood. CTCs were first described in 1869 by an Australian pathologist, Thomas Ashworth, in a patient with metastatic cancer [ 39 ]. The importance of CTCs in modern cancer research began in the mid-1990s with the demonstration that CTCs exist early in the course of the disease.

It is estimated that there are about 1–10 CTCs per mL in whole blood of patients with metastatic cancer, even fewer in patients with early-stage cancer [ 40 ]. For comparison, 1 mL of blood contains a few million white blood cells and a billion erythrocytes. The identification of CTCs, being in such low frequency, requires some special tumoral markers (e.g., EpCAM and cytokeratins) to capture and isolate them. Unfortunately, the common markers for recognizing the majority of CTCs are not effective enough for clinical application [ 41 ]. Although accumulated evidences have shown that the presence of CTCs is a strong negative prognostic factor in the patients with metastatic breast, lung and colorectal cancers, detecting CTCs might not be an ideal branch to hold on for the hope of early cancer detection [ 42 , 43 , 44 , 45 ].

Circulating tumor DNA (ctDNA) is tumor-derived fragmented DNA in the circulatory system, which is mainly derived from the tumor cell death through necrosis and/or apoptosis [ 46 ]. Given its origin, ctDNA inherently carries cancer-specific genetic and epigenetic aberrations, which can be used as a surrogate source of tumor DNA for cancer diagnosis and prognostic prediction. Ideally, as a noninvasive tumor early screening tool, a liquid biopsy test should be able to detect many types of cancers and provide the information of tumor origin for further specific clinical management. In fact, the somatic mutations of ctDNA in different types of tumor are highly variable, even in the different individuals with the same type of tumor [ 47 ]. Additionally, most tumors do not possess driver mutations, with some notable exceptions, which make the somatic mutations of ctDNA not suitable for early detection of the tumor.

Increased methylation of the promoter regions of tumor suppressor genes is an early event in many types of tumor, suggesting that altered ctDNA methylation patterns could be one of the first detectable neoplastic changes associated with tumorigenesis [ 48 ]. ctDNA methylation profiling provides several advantages over somatic mutation analysis for cancer detection including higher clinical sensitivity and dynamic range, multiple detectable methylation target regions, and multiple altered CpG sites within each targeted genomic region. Further, each methylation marker is present in both cancer tissue and ctDNA, whereas only a fraction of mutations present in cancer tissue could be detected in ctDNA.

In 2017, there were two inspiring studies that revealed the values of using ctDNA methylation analysis for cancer early diagnosis [ 49 , 50 ]. After partitioning the human genome into blocks of tightly coupled CpG methylation sites, namely methylation haplotype blocks (MHBs), Guo and colleagues performed tissue-specific methylation analyses at the MHBs level to accurately determine the tissue origin of the cancer using ctDNA from their enrolled patients [ 49 ]. In another study, Xu and colleagues identified a hepatocellular carcinoma (HCC) enriched methylation marker panel by comparing the HCC tissue and blood leukocytes from normal individuals and showed that methylation profiles of HCC tumor DNA and matched plasma ctDNA were highly correlated. In this study, after quantitative measurement of the methylation level of candidate markers in ctDNA from a large cohort of 1098 HCC patients and 835 normal controls, ten methylation markers were selected to construct a diagnostic prediction model. The proposed model demonstrated a high diagnostic specificity and sensitivity, and was highly correlated with tumor burden, treatment response, and tumor stage [ 50 ].

With the rapid development of highly sensitive detection methods, especially the technologies of massively parallel sequencing or next-generation sequencing (NGS)-based assays and digital PCR (dPCR), we strongly believe that the identification of a broader “pan-cancer” methylation panel applied for ctDNA analyses, probably in combination with detections of somatic mutation and tumor-derived exosomes, would allow more effective screening for common cancers in the near future.

Edison Liu 1 , Hui-Yan Luo 2 .

[email protected]; [email protected]

Question 100: Can phytochemicals be more specific and efficient at targeting P-glycoproteins to overcome multi-drug resistance in cancer cells?

Though several anticancer agents are approved to treat different types of cancers, their full potentials have been limited due to the occurrence of drug resistance. Resistance to anticancer drugs develops by a variety of mechanisms, one of which is increased drug efflux by transporters. The ATP-binding cassette (ABC) family drug efflux transporter P-glycoprotein (P-gp or multi-drug resistance protein 1 [MDRP1]) has been extensively studied and is known to play a major role in the development of multi-drug resistance (MDR) to chemotherapy [ 51 ]. In brief, overexpressed P-gp efflux out a wide variety of anticancer agents (e.g.: vinca alkaloids, doxorubicin, paclitaxel, etc.), leading to a lower concentration of these drugs inside cancer cells, thereby resulting in MDR. Over the past three decades, researchers have developed several synthetic P-gp inhibitors to block the efflux of anticancer drugs and have tested them in clinical trials, in combination with chemotherapeutic drugs. But none were found to be suitable enough in overcoming MDR and to be released for marketing, mainly due to the side effects associated with cross-reactivity towards other ABC transporters (BCRP and MRP-1) and the inhibition of CYP450 drug metabolizing enzymes [ 52 , 53 ].

On the other hand, a number of phytochemicals have been reported to have P-gp inhibitory activity. Moreover, detailed structure–activity studies on these phytochemicals have delineated the functional groups essential for P-gp inhibition [ 53 , 54 ]. Currently, one of the phytochemicals, tetrandrine (CBT-1 ® ; NSC-77037), is being used in a Phase I clinical trial ( http://www.ClinicalTrials.gov ; NCT03002805) in combination with doxorubicin for the treatment of metastatic sarcoma. Before developing phytochemicals or their derivatives as P-gp inhibitors, they need to be investigated thoroughly for their cross-reactivity towards other ABC transporters and CYP450 inhibition, in order to avoid toxicities similar to the older generation P-gp inhibitors that have failed in clinical trials.

Therefore, the selectivity for P-gp over other drug transporters and drug metabolizing enzymes should be considered as important criterias for the development of phytochemicals and their derivatives for overcoming MDR.

Mohane Selvaraj Coumar and Safiulla Basha Syed.

Centre for Bioinformatics, School of Life Sciences, Pondicherry University, Kalapet, Puducherry 605014, India.

[email protected]; [email protected]

Question 101: Is cell migration a selectable trait in the natural evolution of carcinoma?

The propensity of solid tumor malignancy to metastasize remains the main cause of cancer-related death, an extraordinary unmet clinical need, and an unanswered question in basic cancer research. While dissemination has been traditionally viewed as a late process in the progression of malignant tumors, amount of evidence indicates that it can occur early in the natural history of cancer, frequently when the primary lesion is still barely detectable.

A prerequisite for cancer dissemination is the acquisition of migratory/invasive properties. However, whether, and if so, how the migratory phenotype is selected for during the natural evolution of cancer and what advantage, if any, it may provide to the growing malignant cells remains an open issue. The answers to these questions are relevant not only for our understating of cancer biology but also for the strategies we adopt in an attempt of curbing this disease. Frequently, indeed, particularly in pharmaceutical settings, targeting migration has been considered much like trying “to shut the stable door after the horse has bolted” and no serious efforts in pursuing this aim has been done.

We argue, instead, that migration might be an intrinsic cancer trait that much like proliferation or increased survival confers to the growing tumor masses with striking selective advantages. The most compelling evidence in support for this contention stems from studies using mathematical modeling of cancer evolution. Surprisingly, these works highlighted the notion that cell migration is an intrinsic, selectable property of malignant cells, so intimately intertwined with more obvious evolutionarily-driven cancer traits to directly impact not only on the potential of malignant cells to disseminate but also on their growth dynamics, and ultimately provide a selective evolutionary advantage. Whether in real life this holds true remains to be assessed, nevertheless, work of this kind defines a framework where the acquisition of migration can be understood in a term of not just as a way to spread, but also to trigger the emergence of malignant clones with favorable genetic or epigenetic traits.

Alternatively, migratory phenotypes might emerge as a response to unfavorable conditions, including the mechanically challenging environment which tumors, and particularly epithelial-derived carcinoma, invariably experience. Becoming motile, however, may not per se being fixed as phenotypic advantageous traits unless it is accompanied or is causing the emergence of specific traits, including drug resistance, self-renewal, and survival. This might be the case, for example, during the process of epithelial-to-mesenchymal transition (EMT), which is emerging as an overarching mechanism for dissemination. EMT, indeed, may transiently equip individual cancer cells not only with migratory/invasive capacity but also with increased resistance to drug treatment, stemness potential at the expanse of fast proliferation.

Thus, within this framework targeting pro-migratory genes, proteins and processes may become a therapeutically valid alternative or a complementary strategy not only to control carcinoma dissemination but also its progression and development.

Giorgio Scita.

IFOM, The FIRC Institute of Molecular Oncology, Via Adamello 16, 20139 Milan, Italy; Department of Oncology and Hemato-Oncology (DIPO), School of Medicine, University of Milan, Via Festa del Perdono 7, 20122, Italy.

[email protected]

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Evidence-based medicine resource guide.

• Defining EBM

Types of Clinical Questions

Formulating a well built clinical question, type of clinical question and study design.

• Point-of-Care Tools
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Resources and Types of Clinical Question

Background questions are best answered by medical textbooks, point-of-care tools such as DynaMed Plus and Essential Evidence Plus, and narrative reviews.

Foreground questions are best answered by consulting medical databases such as MEDLINE (via PubMed or Ovid), Embase, Cochrane Database of Systematic Reviews and ACP Journal Club.

DML's Clinical Quick Reference page is a great place to locate EBM resources. Each resource has been labeled background and/or foreground, for you!

Clinical questions may be categorized as either background or foreground. Why is this important?

Determining the type of question will help you to select the best resource to consult for your answer.

Background questions ask for general knowledge about an illness, disease, condition, process or thing. These types of questions typically ask who, what, where, when, how & why about things like a disorder, test, or treatment, etc.

For example

• How overweight is a woman to be considered slightly obese?
• What are the clinical manifestations of menopause?
• What causes migraines?

Foreground questions ask for specific knowledge to inform clinical decisions. These questions typically concern a specific patient or particular population. Foreground questions tend to be more specific and complex compared to background questions. Quite often, foreground questions investigate comparisons, such as two drugs, two treatments, two diagnostic tests, etc. Foreground questions may be further categorized into one of 4 major types: treatment/therapy, diagnosis, prognosis, or etiology/harm.

• Is Crixivan effective when compared with placebo in slowing the rate of functional impairment in a 45 year old male patient with Lou Gehrig's Disease?
• In pediatric patients with Allergic Rhinitis, are Intranasal steroids more effective than antihistamines in the management of Allergic Rhinitis symptoms?

According to the Centre for Evidence Based Medicine (CEBM) , "one of the fundamental skills required for practising EBM is the asking of well-built clinical questions. To benefit patients and clinicians, such questions need to be both directly relevant to patients' problems and phrased in ways that direct your search to relevant and precise answers."

A well-built clinical foreground question should have all four components. The PICO model is a helpful tool that assists you in organizing and focusing your foreground question into a searchable query. Dividing into the PICO elements helps identify search terms/concepts to use in your search of the literature.

P = Patient, Problem, Population (How would you describe a group of patients similar to you? What are the most important characteristics of the patient?)

I = Intervention, Prognostic Factor, Exposure (What main intervention are you considering? What do you want to do with this patient?)

C = Comparison (What are you hoping to compare with the intervention: another treatment, drug, placebo, a different diagnostic test, etc.? It's important to include this element and to be as specific as possible.)

O = Outcome (What are you trying to accomplish, measure, improve or affect? Outcomes may be disease-oriented or patient-oriented.)

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Two additional important elements of the well-built clinical question to consider are the type of foreground question and the type of study (methodology) . This information can be helpful in focusing the question and determining the most appropriate type of evidence.

Foreground questions can be further divided into questions that relate to therapy, diagnosis, prognosis, etiology/harm

• Therapy: Questions of treatment in order to achieve some outcome. May include drugs, surgical intervention, change in diet, counseling, etc.
• Diagnosis: Questions of identification of a disorder in a patient presenting with specific symptoms.
• Prognosis: Questions of progression of a disease or likelihood of a disease occurring.
• Etiology/Harm: Questions of negative impact from an intervention or other exposure.

Meta-analysis: A statistical technique that summarizes the results of several studies in a single weighted estimate, in which more weight is given to results of studies with more events and sometimes to studies of higher quality.

Systematic Review: a review in which specified and appropriate methods have been used to identify, appraise, and summarize studies addressing a defined question. (It can, but need not, involve meta-analysis). In Clinical Evidence, the term systematic review refers to a systematic review of RCTs unless specified otherwise.

Randomized Controlled Trial: a trial in which participants are randomly assigned to two or more groups: at least one (the experimental group) receiving an intervention that is being tested and another (the comparison or control group) receiving an alternative treatment or placebo. This design allows assessment of the relative effects of interventions.

Controlled Clinical Trial: a trial in which participants are assigned to two or more different treatment groups. In Clinical Evidence, we use the term to refer to controlled trials in which treatment is assigned by a method other than random allocation. When the method of allocation is by random selection, the study is referred to as a randomized controlled trial (RCT). Non-randomized controlled trials are more likely to suffer from bias than RCTs.

Cohort Study: a non-experimental study design that follows a group of people (a cohort), and then looks at how events differ among people within the group. A study that examines a cohort, which differs in respect to exposure to some suspected risk factor (e.g. smoking), is useful for trying to ascertain whether exposure is likely to cause specified events (e.g. lung cancer). Prospective cohort studies (which track participants forward in time) are more reliable than retrospective cohort studies.

Case control study: a study design that examines a group of people who have experienced an event (usually an adverse event) and a group of people who have not experienced the same event, and looks at how exposure to suspect (usually noxious) agents differed between the two groups. This type of study design is most useful for trying to ascertain the cause of rare events, such as rare cancers.

Case Series: analysis of series of people with the disease (there is no comparison group in case series).

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Medical Research Paper Topics

This page provides a comprehensive list of medical research paper topics divided into 20 categories, each with 10 unique subjects. The categories span across various subfields, including anatomy and physiology, diseases, epidemiology, health and fitness, health disparities, healthcare, kinesiology, mental health, pharmacology, and veterinary medicine. Additionally, the page offers expert advice on choosing the right topic and crafting an impactful medical research paper. It also introduces iResearchNet’s writing services, which are designed to assist students in creating high-quality, custom medical research papers.

200 Medical Research Paper Topics:

Medical research is a vast and diverse field, offering a plethora of topics for students and researchers to explore. The choice of topic can significantly influence the direction of your research and the impact of your findings. Therefore, it’s crucial to choose a topic that not only interests you but also aligns with your academic and career goals. To assist you in this endeavor, we have compiled a comprehensive list of medical research paper topics, divided into 20 categories, each with 10 unique topics.

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1. Anatomy and Physiology:

• The role of the lymphatic system in maintaining homeostasis.
• The impact of aging on muscle function and structure.
• The physiological effects of stress on the human body.
• The role of the endocrine system in metabolism.
• The impact of exercise on cardiovascular health.
• The role of the nervous system in sensation and perception.
• The physiological effects of sleep deprivation.
• The role of the digestive system in nutrient absorption.
• The impact of genetic factors on human physiology.
• The role of the immune system in disease prevention.

2. Diseases:

• The genetic and environmental factors contributing to the development of cancer.
• The impact of lifestyle factors on the prevalence of heart disease.
• The role of vaccination in the prevention of infectious diseases.
• The challenges in the management and treatment of diabetes.
• The impact of the human microbiome on disease development.
• The role of inflammation in the pathogenesis of autoimmune diseases.
• The challenges in the diagnosis and treatment of Alzheimer’s disease.
• The impact of antibiotic resistance on the treatment of bacterial infections.
• The role of genetic mutations in the development of rare diseases.
• The impact of viral evolution on the spread of infectious diseases.

3. Epidemiology:

• The impact of socioeconomic factors on the prevalence of infectious diseases.
• The role of vaccination in the prevention of disease outbreaks.
• The impact of climate change on the spread of vector-borne diseases.
• The role of epidemiological studies in the development of public health policies.
• The impact of population density on the spread of infectious diseases.
• The role of epidemiology in the identification of risk factors for chronic diseases.
• The impact of global travel on the spread of infectious diseases.
• The role of epidemiological data in the management of disease outbreaks.
• The impact of demographic factors on disease prevalence.
• The role of epidemiology in the study of health disparities.

4. Health and Fitness:

• The impact of physical activity on mental health.
• The role of diet in the prevention of chronic diseases.
• The impact of sedentary lifestyle on health.
• The role of physical fitness in the prevention of heart disease.
• The impact of obesity on health and lifespan.
• The role of exercise in the management of stress.
• The impact of nutrition on cognitive function.
• The role of physical activity in the management of chronic diseases.
• The impact of sleep on health and wellbeing.
• The role of lifestyle interventions in the prevention of diabetes.

5. Health Disparities:

• The impact of socioeconomic status on health outcomes.
• The role of healthcare access in health disparities.
• The impact of racial and ethnic disparities on health outcomes.
• The role of social determinants of health in health disparities.
• The impact of health literacy on health outcomes.
• The role of cultural competence in reducing health disparities.
• The impact of gender disparities on health outcomes.
• The role of health policy in addressing health disparities.
• The impact of environmental factors on health disparities.
• The role of education in reducing health disparities.

6. Healthcare:

• The impact of healthcare reform on patient outcomes.
• The role of telemedicine in improving healthcare access.
• The impact of health information technology on patient care.
• The role of healthcare quality in patient satisfaction.
• The impact of healthcare costs on health outcomes.
• The role of healthcare leadership in improving patient care.
• The impact of health insurance on healthcare access.
• The role of healthcare innovation in improving patient outcomes.
• The impact of healthcare policy on patient care.
• The role of healthcare management in improving healthcare quality.

7. Kinesiology:

• The impact of physical activity on muscle function.
• The role of biomechanics in injury prevention.
• The impact of exercise on cognitive function.
• The role of kinesiology in the prevention of chronic diseases.
• The impact of physical fitness on health outcomes.
• The role of kinesiology in the management of sports injuries.
• The impact of exercise physiology on athletic performance.
• The role of kinesiology in the promotion of physical activity.
• The impact of motor control on athletic performance.
• The role of kinesiology in the study of human movement.

8. Mental Health:

• The impact of stress on mental health.
• The role of psychotherapy in the treatment of mental disorders.
• The impact of mental illness on quality of life.
• The role of mental health promotion in the prevention of mental disorders.
• The impact of stigma on mental health outcomes.
• The role of mental health services in the treatment of mental disorders.
• The impact of mental health policy on mental health services.
• The role of mental health literacy in the prevention of mental disorders.
• The impact of mental health disparities on mental health outcomes.
• The role of mental health research in improving mental health care.

9. Pharmacology:

• The impact of drug interactions on patient safety.
• The role of pharmacokinetics in drug development.
• The impact of pharmacogenomics on personalized medicine.
• The role of pharmacology in the treatment of diseases.
• The impact of drug resistance on the treatment of infectious diseases.
• The role of pharmacology in the management of pain.
• The impact of drug abuse on public health.
• The role of pharmacology in the prevention of drug interactions.
• The impact of drug development on patient care.
• The role of pharmacology in the study of drug action.

10. Veterinary Medicine:

• The impact of zoonotic diseases on public health.
• The role of veterinary medicine in the prevention of animal diseases.
• The impact of veterinary medicine on food safety.
• The role of veterinary medicine in the treatment of animal diseases.
• The impact of veterinary public health on human health.
• The role of veterinary medicine in the prevention of zoonotic diseases.
• The impact of animal health on public health.
• The role of veterinary medicine in the promotion of animal welfare.
• The impact of veterinary medicine on animal behavior.
• The role of veterinary medicine in the study of animal physiology.

11. Neurology:

• The impact of neurodegenerative diseases on public health.
• The role of neurology in the treatment of neurological disorders.
• The impact of neurological research on patient care.
• The role of neurology in the study of the nervous system.
• The impact of neurological disorders on quality of life.
• The role of neurology in the diagnosis of neurological disorders.
• The impact of neurological diseases on mental health.
• The role of neurology in the prevention of neurological diseases.
• The impact of neurological disorders on cognitive function.
• The role of neurology in the study of brain function.

12. Oncology:

• The impact of cancer on public health.
• The role of oncology in the treatment of cancer.
• The impact of oncological research on patient care.
• The role of oncology in the study of cancer.
• The impact of cancer on quality of life.
• The role of oncology in the diagnosis of cancer.
• The impact of cancer on mental health.
• The role of oncology in the prevention of cancer.
• The impact of cancer on physical health.
• The role of oncology in the study of cancer biology.

13. Cardiology:

• The impact of heart disease on public health.
• The role of cardiology in the treatment of heart disease.
• The impact of cardiological research on patient care.
• The role of cardiology in the study of the cardiovascular system.
• The impact of heart disease on quality of life.
• The role of cardiology in the diagnosis of heart disease.
• The impact of heart disease on mental health.
• The role of cardiology in the prevention of heart disease.
• The impact of heart disease on physical health.
• The role of cardiology in the study of heart function.

14. Endocrinology:

• The impact of endocrine disorders on public health.
• The role of endocrinology in the treatment of endocrine disorders.
• The impact of endocrinological research on patient care.
• The role of endocrinology in the study of the endocrine system.
• The impact of endocrine disorders on quality of life.
• The role of endocrinology in the diagnosis of endocrine disorders.
• The impact of endocrine disorders on mental health.
• The role of endocrinology in the prevention of endocrine disorders.
• The impact of endocrine disorders on physical health.
• The role of endocrinology in the study of hormone function.

15. Gastroenterology:

• The impact of gastrointestinal disorders on public health.
• The role of gastroenterology in the treatment of gastrointestinal disorders.
• The impact of gastroenterological research on patient care.
• The role of gastroenterology in the study of the gastrointestinal system.
• The impact of gastrointestinal disorders on quality of life.
• The role of gastroenterology in the diagnosis of gastrointestinal disorders.
• The impact of gastrointestinal disorders on mental health.
• The role of gastroenterology in the prevention of gastrointestinal disorders.
• The impact of gastrointestinal disorders on physical health.
• The role of gastroenterology in the study of digestive function.

16. Dermatology:

• The impact of skin disorders on public health.
• The role of dermatology in the treatment of skin disorders.
• The impact of dermatological research on patient care.
• The role of dermatology in the study of the skin.
• The impact of skin disorders on quality of life.
• The role of dermatology in the diagnosis of skin disorders.
• The impact of skin disorders on mental health.
• The role of dermatology in the prevention of skin disorders.
• The impact of skin disorders on physical appearance.
• The role of dermatology in the study of skin health.

17. Pulmonology:

• The impact of respiratory disorders on public health.
• The role of pulmonology in the treatment of respiratory disorders.
• The impact of pulmonological research on patient care.
• The role of pulmonology in the study of the respiratory system.
• The impact of respiratory disorders on quality of life.
• The role of pulmonology in the diagnosis of respiratory disorders.
• The impact of respiratory disorders on mental health.
• The role of pulmonology in the prevention of respiratory disorders.
• The impact of respiratory disorders on physical health.
• The role of pulmonology in the study of lung function.

18. Pediatrics:

• The impact of childhood diseases on public health.
• The role of pediatrics in the treatment of childhood diseases.
• The impact of pediatric research on patient care.
• The role of pediatrics in the study of childhood diseases.
• The impact of childhood diseases on quality of life.
• The role of pediatrics in the diagnosis of childhood diseases.
• The impact of childhood diseases on mental health.
• The role of pediatrics in the prevention of childhood diseases.
• The impact of childhood diseases on physical health.
• The role of pediatrics in the study of child health.

19. Geriatrics:

• The impact of aging on public health.
• The role of geriatrics in the treatment of age-related diseases.
• The impact of geriatric research on patient care.
• The role of geriatrics in the study of aging.
• The impact of aging on quality of life.
• The role of geriatrics in the diagnosis of age-related diseases.
• The impact of aging on mental health.
• The role of geriatrics in the prevention of age-related diseases.
• The impact of aging on physical health.
• The role of geriatrics in the study of the aging process.

20. Psychiatry:

• The impact of mental disorders on public health.
• The role of psychiatry in the treatment of mental disorders.
• The impact of psychiatric research on patient care.
• The role of psychiatry in the study of mental disorders.
• The impact of mental disorders on quality of life.
• The role of psychiatry in the diagnosis of mental disorders.
• The impact of mental disorders on social interactions.
• The role of psychiatry in the prevention of mental disorders.
• The impact of mental disorders on physical health.
• The role of psychiatry in the study of mental health.

The field of medical research is vast and diverse, offering a multitude of topics for exploration. The choice of a research topic is a critical step in the research process, and it can significantly influence the direction of your research and the impact of your findings. Therefore, it’s crucial to choose a topic that not only interests you but also aligns with your academic and career goals. This comprehensive list of medical research paper topics provides a starting point for your research journey. Remember, the best research topic is one that is original, manageable, and meaningful.

More Medical Research Paper Topics

• Anatomy and Physiology Research Paper Topics
• Biomedical Research Paper Topics
• Clinical Research Paper Topics
• Disease Research Paper Topics
• Epidemiology Research Paper Topics
• Exercise Physiology Research Paper Topics
• Health Disparities Research Paper Topics
• Healthcare Research Paper Topics
• Kinesiology Research Paper Topics
• Mental Health Research Paper Topics
• Pharmacology Research Paper Topics
• Phlebotomy Research Paper Topics
• Physical Fitness Research Paper Topics
• Radiology Research Paper Topics
• Veterinary Medicine Research Paper Topics

Introduction to Medical Research

Medical research is a broad and multifaceted field that encompasses a wide range of topics and disciplines. It is the backbone of modern healthcare and plays a crucial role in improving the quality of life for people around the world. Medical research is the driving force behind new discoveries, innovative treatments, and the development of policies that shape public health.

At its core, medical research seeks to understand the complexities of the human body, the diseases that afflict it, and the various external factors that influence health. It is a field that is constantly evolving, with new technologies and methodologies continually pushing the boundaries of what we know about health and disease.

One of the key aspects of medical research is its interdisciplinary nature. It draws upon a variety of fields, including biology, chemistry, physics, and even social sciences. This interdisciplinary approach allows for a more comprehensive understanding of health and disease, as it considers not only the biological aspects but also the social, environmental, and psychological factors that influence health.

For instance, research in anatomy and physiology delves into the structure and function of the human body, providing the foundation for understanding health and disease. Studies in diseases and epidemiology, on the other hand, focus on the patterns, causes, and effects of health and disease conditions in defined populations. Health and fitness research explores the impact of lifestyle choices on health, while health disparities research examines the inequalities in health outcomes and healthcare that exist among different population groups.

Research in healthcare and kinesiology often intersects, examining how healthcare services and physical activity can improve health outcomes. Mental health research is a rapidly growing field, addressing a wide range of mental health disorders and the impact they have on overall health. Pharmacology research is vital for the development of new drugs and therapies, while veterinary medicine research not only improves the health of animals but can also provide insights into human health through comparative medicine.

Choosing a topic for a medical research paper can be a daunting task given the breadth and depth of the field. However, it also offers a wealth of opportunities to delve into a topic that is not only interesting but can also contribute to the betterment of health and healthcare.

How to Choose Medical Research Paper Topics

Choosing a topic for your medical research paper is a critical step in your academic journey. The topic you select will guide your research direction, influence your motivation, and determine the depth of your understanding of the subject matter. Therefore, it’s essential to choose wisely and consider various factors before making a decision. Here are ten comprehensive tips to help you select the right medical research paper topic:

• Identify Your Interests: The first step in choosing a research topic is to consider your interests within the field of medicine. Are you fascinated by neurology, passionate about public health, or intrigued by the complexities of surgery? Identifying your interests can help you narrow down potential topics. When you choose a topic you’re passionate about, the research process becomes less of a task and more of an exploration. You’ll find yourself more engaged, and the enthusiasm will reflect in the quality of your research.
• Consider the Scope: The scope of your topic is an important factor to consider. A topic that’s too broad can make your research overwhelming, as you may have to sift through an enormous amount of information and struggle to cover all aspects within the constraints of your paper. On the other hand, a topic that’s too narrow might not provide enough material for a comprehensive study. Strive for a balance where your topic is specific enough to manage but broad enough to explore in depth.
• Check for Resources: Before finalizing a topic, ensure there are sufficient resources available for your research. These resources could include books, scholarly articles, reputable online sources, and expert interviews. Conduct a preliminary literature review to gauge the availability of sources. A lack of resources can lead to a weak paper, while an abundance of resources can provide multiple perspectives and strengthen your research.
• Relevance to Current Times: Choosing a topic that is relevant to current medical issues or ongoing research can make your paper more engaging and significant. It allows you to contribute to the existing body of knowledge and possibly influence future research or policy. Whether it’s a new treatment method, an emerging disease, or a controversial medical practice, current topics can spark interest and debate.
• Consult with Your Instructor: Your instructor or advisor can be a valuable resource when choosing a topic. They can provide guidance, help you refine a broad topic, expand a narrow one, and provide feedback on your initial ideas. They can also point you towards resources or research areas you might not have considered.
• Consider the Target Audience: Always keep your target audience in mind when choosing a research topic. The topic should be interesting and accessible to them. If you’re writing for a class, consider what you’ve already discussed and what themes your instructor has emphasized. If you’re writing for publication, consider the interests and level of expertise of the readers of the journal.
• Practicality: Consider the practical aspects of your research. If your research involves primary data collection, consider the feasibility of the methods you plan to use. Do you have access to the necessary equipment or population? Is your study ethical and approved by an ethics committee? Practical considerations can significantly influence your choice of topic.
• Originality: While it’s important to build upon existing research, strive to bring a unique perspective to your topic. This could involve studying a well-researched topic from a new angle, exploring a less-studied area of medical research, or proposing a new hypothesis. Originality can make your research stand out and contribute to the advancement of your field.
• Alignment with Course Objectives: Your research topic should align with the objectives of your course or curriculum. This ensures that your research paper will not only be interesting but also academically beneficial. It can demonstrate your understanding ofthe course material and your ability to apply it in a real-world context.
• Future Career Relevance: If you have a clear idea of your future career path, consider choosing a topic that could be beneficial in your professional life. This could mean researching a topic related to a field you’d like to specialize in, a current issue in your future profession, or a novel area of study that could give you a competitive edge in your career.

In addition to these tips, it’s also important to consider the timeline of your research. Some topics may require more time to research than others, especially if they involve complex experiments or hard-to-reach populations. Make sure you choose a topic that you can reasonably research and write about within your given timeframe.

Also, consider the potential impact of your research. While it’s not always possible to predict how influential a research paper will be, you can consider whether the topic has the potential to affect policy, influence clinical practice, or lead to new research questions. Choosing a topic with potential impact can be particularly important if you plan to pursue a career in academia or research.

Remember, choosing a research topic is not a decision to be taken lightly. It’s the foundation of your research paper and can significantly influence your enjoyment of the writing process, your grade, and even your future career. Take your time, consider your options, and choose a topic that you are passionate about, meets practical considerations, and has the potential to contribute to the field of medical science.

Finally, be flexible. Research is a dynamic process, and it’s okay to refine or even change your topic as you delve deeper into your research. The most important thing is to stay curious, open-minded, and dedicated to uncovering new knowledge. With the right topic, research can be a rewarding journey of discovery.

How to Write a Medical Research Paper

Writing a medical research paper is a comprehensive task that demands a profound understanding of the subject matter, a systematic approach, and a lucid writing style. This process can be quite daunting, especially for those who are new to it. However, with the right guidance and a well-structured plan, it can be a rewarding experience. Here are ten detailed tips to guide you through the process:

• Understand the Assignment Thoroughly: The first step in writing a medical research paper is to fully comprehend the assignment. What is the specific question you’re being asked to address? What is the scope of the research paper? What are the formatting requirements? Are there any specific sources you need to use? Understanding the assignment in its entirety will help you focus your research and ensure you meet all the requirements. It’s crucial to clarify any doubts with your professor or advisor at this stage to avoid any misunderstandings later on.
• Choose a Suitable Topic: If you have the freedom to choose your own topic, select one that genuinely interests you. Your passion for the subject will come through in your writing, making the process more enjoyable and less of a chore. Make sure the topic is relevant to your field of study and is something you can manage within the given timeframe and word limit. A well-chosen topic can make the difference between a paper that is a joy to research and write and one that is a burdensome chore.
• Conduct Thorough Research: The next step is to conduct comprehensive research. Use reputable sources such as academic journals, textbooks, and government health websites to gather information. Make sure to use the most recent data and research available, as the medical field is constantly evolving and it’s important to stay up-to-date. Keep track of your sources as you go along for easy referencing later. This will also save you a lot of time when you are compiling your bibliography.
• Create an Outline: An outline is a roadmap for your research paper. It should include an introduction, a body with several points or arguments, and a conclusion. Each point should be supported by evidence from your research. An outline will help structure your thoughts and ideas, making your writing process smoother and more organized. It also allows you to see the flow of your arguments and make sure that everything is in a logical order.
• Write a Strong Thesis Statement: Your thesis statement is the backbone of your research paper. It should clearly state the main idea or argument of your paper. It should be concise, specific, and arguable. The thesis statement will guide your writing and keep your argument focused. Spend some time crafting a strong thesis statement. It’s worth the effort as it will give direction to your research and writing.
• Start Writing: Once you have your outline and thesis statement, you can start writing. Begin with a draft. Don’t worry about making it perfect the first time around. Just get your ideas down on paper. You can revise and improve it later. Remember, writing is a process. It’s okay to have multiple drafts. The important thing is to start writing.
• Use Clear, Concise Language: Medical research papers should be written in a clear, concise, and formal style. Avoid jargon and complex sentences. Make sure your ideas are expressed clearly and logically. Remember, your goal is to communicate your research and ideas, not to impress with big words or complex sentences.
• Cite Your Sources: Always cite your sources. This gives credit to the original authors and allows readers to follow up on your research. Be sure to use the citation style required by your professor or the journal you are submitting to. Proper citation is not just about avoiding plagiarism. It also lends credibility to yourpaper and demonstrates the breadth of your research.
• Revise and Edit: Once you’ve finished your draft, take the time to revise and edit. This is where you refine your arguments, clarify your thoughts, and polish your language. Look for any inconsistencies, grammatical errors, or areas that could be clarified or improved. Consider having a peer or mentor review your paper. They can provide valuable feedback and catch errors you might have missed. Remember, good writing is rewriting. Don’t be afraid to make changes, and don’t be discouraged if your first draft isn’t perfect.
• Proofread: Finally, proofread your paper. Check for any spelling, grammar, or punctuation errors. Make sure all citations and references are correct. Proofreading is a crucial step in the writing process. Even the most well-researched and well-written paper can lose credibility if it’s full of errors. Consider using a proofreading tool or ask someone else to proofread your paper. A fresh pair of eyes can often catch mistakes you might have overlooked.

In addition to these tips, remember to take breaks during your writing process. Writing a medical research paper is a significant undertaking, and it’s important to avoid burnout. Take time to rest and refresh your mind. This will help you maintain your focus and energy levels.

Also, keep in mind that writing a research paper is not just about the end product. It’s also about the process. It’s an opportunity to learn more about a topic you’re interested in, to improve your research and writing skills, and to contribute to your field of study. Approach it with curiosity and enthusiasm, and you’ll find that writing a medical research paper can be a rewarding experience.

Writing a medical research paper is a systematic process that requires careful planning, thorough research, and meticulous writing. By following these tips, you can produce a paper that is informative, insightful, and contributes to your field of study. Remember, every writer faces challenges along the way, so don’t be discouraged if you encounter difficulties. With persistence and dedication, you can write a successful medical research paper.

iResearchNet’s Writing Services

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Literature Searching

In this guide.

• Introduction
• Steps for searching the literature in PubMed
• Step 1 - Formulate a search question
• Step 2- Identify primary concepts and gather synonyms
• Step 3 - Locate subject headings (MeSH)
• Step 4 - Combine concepts using Boolean operators
• Step 5 - Refine search terms and search in PubMed
• Step 6 - Apply limits

Formulating a Well-Defined, Answerable Research Question

It is important to develop a well-defined, answerable research question because it

• Defines the focus of your literature search
• Identifies the appropriate study design and methods
• Makes searching for evidence simpler and more effective
• Helps you identify relevant results and separate relevant results from irrelevant ones

Tips for developing a clinical research question:

• The question is directly relevant to the most important health issue for the patient
• The question is focused and when answered, will help the patient the most
• The question is phrased to facilitate a targeted literature search for precise answers

Adopted from CEBM:  what makes a good clinical question

Example of a vague question:

"Is mobile technology good at managing diabetes?"

• Mobile technology is very broad. Mobile apps, mobile phones, voice/text? 
• What does “good” mean? How will you determine if the technology is effective?
• What kind of diabetes? Type 1? Type 2? Both?
• Who are the “patients”? Adults? Adolescents? Women? Patients with specific diagnoses?
• Any time frame?

Example of a well-defined question:

"Are mobile health technology interventions more effective in managing patients with Type 1 or Type 2 diabetes than in-person care?"

PICO Framework

PICO or PICO(T) (patient/problem/population, intervention, comparison, outcome, time) is a well-known approach for framing a research question. It divides the research question into key components making it easy and searchable.

Example:  "Are mobile health technology interventions more effective in managing patients with Type 1 or Type 2 diabetes than in-person care?"

Other question formulation frameworks:

PIE  (Population, Intervention, Effect / Outcome)

SPIDER  (Sample, Phenomena of Interest, Design, Evaluation, Research type)

SPICE  (Setting, Perspective, Intervention, Comparison, Evaluation)

ECLIPSE (Expectation, Client group, Location, Impact, Professionals, Service)

• << Previous: Steps for searching the literature in PubMed
• Next: Step 2- Identify primary concepts and gather synonyms >>
• Last Updated: Jan 9, 2024 10:30 AM
• URL: https://laneguides.stanford.edu/LitSearch

Health (Nursing, Medicine, Allied Health)

• Find Articles/Databases
• Reference Resources
• Evidence Summaries & Clinical Guidelines
• Drug Information
• Health Data & Statistics
• Patient/Consumer Facing Materials
• Images and Streaming Video
• Grey Literature
• Mobile Apps & "Point of Care" Tools
• Tests & Measures This link opens in a new window
• Citing Sources
• Selecting Databases
• Framing Research Questions
• Crafting a Search
• Narrowing / Filtering a Search
• Expanding a Search
• Cited Reference Searching
• Saving Searches
• Term Glossary
• Critical Appraisal Resources
• What are Literature Reviews?
• Conducting & Reporting Systematic Reviews
• Finding Systematic Reviews
• Tutorials & Tools for Literature Reviews
• Finding Full Text

Defining the Question: Foreground & Background Questions

In order to most appropriately choose an information resource and craft a search strategy, it is necessary to consider what  kind  of question you are asking: a specific, narrow "foreground" question, or a broader background question that will help give context to your research?

Foreground Questions

A "foreground" question in health research is one that is relatively specific, and is usually best addressed by locating primary research evidence. 

Using a structured question framework can help you clearly define the concepts or variables that make up the specific research question. 

 Across most frameworks, you’ll often be considering:

• a who (who was studied - a population or sample)
• a what (what was done or examined - an intervention, an exposure, a policy, a program, a phenomenon)
• a how ([how] did the [what] affect the [who] - an outcome, an effect). 

PICO is the most common framework for developing a clinical research question, but multiple question frameworks exist.

PICO (Problem/Population, Intervention, Comparison, Outcome)

Appropriate for : clinical questions, often addressing the effect of an intervention/therapy/treatment

Example : For adolescents with type II diabetes (P) does the use of telehealth consultations (I) compared to in-person consultations  (C) improve blood sugar control  (O)?

Framing Different Types of Clinical Questions with PICO

Different types of clinical questions are suited to different syntaxes and phrasings, but all will clearly define the PICO elements.  The definitions and frames below may be helpful for organizing your question:

Intervention/Therapy

Questions addressing how a clinical issue, illness, or disability is treated.

"In__________________(P), how does__________________(I) compared to_________________(C) affect______________(O)?"

Questions that address the causes or origin of disease, the factors which produce or predispose toward a certain disease or disorder.

"Are_________________(P), who have_________________(I) compared with those without_________________(C) at_________________risk for/of_________________(O) over_________________(T)?" 

Questions addressing the act or process of identifying or determining the nature and cause of a disease or injury through evaluation.

In_________________(P) are/is_________________(I) compared with_________________(C) more accurate in diagnosing_________________(O)?

Prognosis/Prediction:

Questions addressing the prediction of the course of a disease.

In_________________(P), how does_________________(I) compared to_________________ (C) influence_________________(O)?

Questions addressing how one experiences a phenomenon or why we need to approach practice differently.

"How do_________________(P) with_________________(I) perceive_________________(O)?" 

Adapted from: Melnyk, B. M., & Fineout-Overholt, E. (2011). Evidence-based practice in nursing & healthcare: A guide to best practice. Philadelphia: Wolters Kluwer/Lippincott Williams & Wilkins.

Beyond PICO: Other Types of Question Frameworks

PICO is a useful framework for clinical research questions, but may not be appropriate for all kinds of reviews.  Also consider:

PEO (Population, Exposure, Outcome)

Appropriate for : describing association between particular exposures/risk factors and outcomes

Example : How do  preparation programs (E) influence the development of teaching competence  (O) among novice nurse educators  (P)?

SPIDER (Sample, Phenomenon of Interest, Design, Evaluation, Research Type)

Appropriate for : questions of experience or perspectives (questions that may be addressed by qualitative or mixed methods research)

Example : What are the experiences and perspectives (E) of  undergraduate nursing students  (S)  in clinical placements within prison healthcare settings (PI)?

SPICE (Setting, Perspective, Intervention/phenomenon of Interest, Comparison, Evaluation)

Appropriate for : evaluating the outcomes of a service, project, or intervention

Example : What are the impacts and best practices for workplace (S) transition support programs (I) for the retention (E) of newly-hired, new graduate nurses (P)?

PCC (Problem/population, Concept, Context)

Appropriate for : broader (scoping) questions

Example : How do nursing schools  (Context) teach, measure, and maintain nursing students ' (P)  technological literacy  (Concept))throughout their educational programs?

Background Questions

To craft a strong and reasonable foreground research question, it is important to have a firm understanding of the concepts of interest.  As such, it is often necessary to ask background questions, which ask for more general, foundational knowledge about a disorder, disease, patient population, policy issue, etc. 

For example, consider the PICO question outlined above:

"For adolescents with type II diabetes does the use of telehealth consultations compared to in-person consultations  improve blood sugar control ?

To best make sense of the literature that might address this PICO question, you would also need a deep understanding of background questions like:

• What are the unique barriers or challenges related to blood sugar management in adolescents with TII diabetes?
• What are the measures of effective blood sugar control?
• What kinds of interventions would fall under the umbrella of 'telehealth'?
• What are the qualitative differences in patient experience in telehealth versus in-person interactions with healthcare providers?
• << Previous: Selecting Databases
• Next: Crafting a Search >>
• Last Updated: May 22, 2024 1:35 PM
• URL: https://guides.nyu.edu/health

Transforming the understanding and treatment of mental illnesses.

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• Clinical Trials

Clinical Research Trials and You: Questions and Answers

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What is a clinical trial?

A clinical trial is a research study that involves people like you. Researchers conduct clinical trials to find new or better ways to prevent, detect, or treat health conditions. Often, researchers want to find out if a new test, treatment, or preventive measure is safe and effective. Tests can include ways to screen for, diagnose, or prevent a disease or condition. Treatments and preventive measures can include medications, surgeries, medical devices, and behavioral therapies.

Clinical trials are important because they serve as the foundation for most medical advances. Without clinical trials, many of the medical treatments and cures we have today wouldn’t exist.

Why should I volunteer for a clinical trial?

People volunteer for clinical trials for many reasons. Some want to advance science or help doctors and researchers learn more about disease and improve health care. Others, such as those with an illness, may join to try new or advanced treatments that aren’t widely available.

Whatever your reason for joining a clinical trial, researchers generally need two types of volunteers: those without specific illnesses or conditions and those with them.  

A healthy volunteer is someone in a clinical trial with no known related health problems. Researchers need healthy volunteers to establish a healthy or optimal reference point. They use data from healthy volunteers to test new treatments or interventions, not to provide direct benefit to participants.

A patient volunteer is someone in a clinical trial who has the condition being studied. Researchers need patient volunteers to learn if new tests, treatments, or preventive measures are safe and effective. Not all trial participants will receive experimental medications or treatments; sometimes, participants may receive a placebo. Researchers need to vary medications and treatments so they can compare results and learn from their differences.

While a study’s treatment or findings may help patients directly, sometimes participants will receive no direct benefit. However, in many cases, study results can still serve as building blocks that are used to help people later.

What would I experience during a clinical trial?

During a clinical trial, the study team will track your health. Participating in a clinical trial may take more time than standard treatment, and you may have more tests and treatments than you would if you weren’t in a clinical trial. The study team also may ask you to keep a log of symptoms or other health measures, fill out forms about how you feel, or complete other tasks. You may need to travel or reside away from home to take part in a study.

What are the risks and benefits of my participation in a clinical trial?

Clinical trials can provide many benefits to participants and society. However, before volunteering for a clinical trial, you should talk with your health care provider and the study team about the risks and benefits.

Potential Risks

When weighing the risks of volunteering, you should consider:

• The likelihood of any harm occurring
• How much harm could result from your participation in the study

Researchers try to limit patient discomfort during clinical trials. However, in some cases, volunteers have complications that require medical attention. In rare cases, volunteers have died when participating in clinical trials.

Potential Benefits

The benefits of volunteering can include:

• Treatment with study medications that may not be available elsewhere
• Care from health care professionals who are familiar with the most advanced treatments available
• The opportunity to learn more about an illness and how to manage it
• Playing an active role in your health care
• Helping others by contributing to medical research

Where can I find a mental health clinical trial?

The National Institute of Mental Health (NIMH) is the lead federal agency for research on mental disorders. While NIMH supports research around the world, it also conducts many clinical trials at the National Institutes of Health (NIH) campus in Bethesda, Maryland.

To learn more about NIMH studies conducted on the NIH campus, visit  NIMH's Join a Study webpage . These studies enroll volunteers from the local area and across the nation. In some cases, participants receive free study-related evaluations, treatment, and transportation to NIH.

To learn more about NIMH-funded clinical trials at universities, medical centers, and other institutions, visit  NIMH's clinical trials webpage .

What is the next step after I find a clinical trial?

To learn more about a specific clinical trial, contact the study coordinator. You can usually find this contact information in the trial’s description.

If you decide to join a clinical trial, let your health care provider know. They may want to talk to the study team to coordinate your care and ensure the trial is safe for you. Find tips to help prepare for and get the most out of your visit .

How do I know if I can join a clinical trial?

People of all ages, ethnicities, and racial backgrounds can volunteer for clinical trials. If you want to join a clinical trial, you must be eligible to participate in that specific trial. Your eligibility can usually be determined by phone or online screening.

All clinical trials have eligibility guidelines called inclusion and exclusion criteria. These criteria may include:

• The type and stage of an illness
• Treatment history
• Other medical conditions

Researchers use these guidelines to find suitable study participants, maximize participant safety, and ensure trial data are accurate.

What kinds of questions should I ask the study team before deciding if I want to take part in a clinical trial?

It can be helpful to write down any questions or concerns you have. When you speak with the study team, you may want to take notes or ask to record the conversation. Bringing a supportive friend or family member may also be helpful.

The following topics may give you some ideas for questions to ask:

• The study’s purpose and duration
• The possible risks and benefits
• Your participation and care
• Personal and cost concerns

For a list of specific questions, check out Questions to Ask About Volunteering for a Research Study  from the U.S. Department of Health and Human Services’ Office for Human Research Protections.

How is my safety protected if I choose to take part in a clinical trial?

Strict rules and laws help protect participants in research studies, and the study team must follow these rules to conduct research. Below are some measures that can help ensure your safety.  

Ethical Guidelines

Ethical guidelines protect volunteers and ensure a study’s scientific integrity. Regulators created these guidelines primarily in response to past research errors and misconduct. Federal policies and regulations require that researchers conducting clinical trials obey these ethical guidelines.

Informed Consent

Before joining a trial, you should understand what your participation will involve. The study team will provide an informed consent document with detailed information about the study. The document will include details about the length of the trial, required visits, medications, and medical procedures. It will also explain the expected outcomes, potential benefits, possible risks, and other trial details. The study team will review the informed consent document with you and answer any questions you have. You can decide then or later if you want to take part in the trial.

If you choose to join the trial, you will be asked to sign the informed consent document. This document is not a contract; it verifies you understand the study and describes what your participation will include and how your data will be used. Your consent in a clinical trial is ongoing and your participation is voluntary. You may stop participating at any time.

Institutional Review Board Review

Institutional review boards (IRBs) review and monitor most clinical trials in the United States. An IRB works to protect the rights, welfare, and privacy of human subjects. An IRB usually includes a team of independent doctors, scientists, and community members. The IRB’s job is to review potential studies, weigh the risks and benefits of studies, and ensure that studies are safe and ethical.

If you’re thinking about volunteering for a clinical trial, ask if an IRB reviewed the trial.

What happens when a clinical trial ends?

When a clinical trial ends, researchers will analyze the data to help them determine the results. After reviewing the findings, researchers often submit them to scientific journals for others to review and build on.

Before your participation ends, the study team should tell you if and how you’ll receive the results. If this process is unclear, be sure to ask about it.

Where can I find more information?

This fact sheet covers the basics of clinical trials. To find more details and resources, visit  NIMH's clinical trials webpage .

For More Information

MedlinePlus  (National Library of Medicine) ( en español  )

ClinicalTrials.gov  ( en español  )

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health NIH Publication No. 23-MH-4379 Revised 2023

The information in this publication is in the public domain and may be reused or copied without permission. However, you may not reuse or copy images. Please cite the National Institute of Mental Health as the source. Read our copyright policy to learn more about our guidelines for reusing NIMH content.

Research Topics

The links below will take you to the Research pages for all of the Department of Medicine’s different Section and Centers. You can explore more specific research topics within those pages.

• Introduction
• Article Information

RCT indicates randomized clinical trial.

eFigure. Core Search Strategy for Structured Literature Search

eTable. Additional RCT Questions, Ranked by Overall Score

Nonauthor Collaborators

Data Sharing Statement

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Coon ER , McDaniel CE , Paciorkowski N, et al. Prioritization of Randomized Clinical Trial Questions for Children Hospitalized With Common Conditions : A Consensus Statement . JAMA Netw Open. 2024;7(5):e2411259. doi:10.1001/jamanetworkopen.2024.11259

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Prioritization of Randomized Clinical Trial Questions for Children Hospitalized With Common Conditions : A Consensus Statement

• 1 Department of Pediatrics, Intermountain Primary Children’s Hospital, Salt Lake City, Utah
• 2 Department of Pediatrics, University of Utah School of Medicine, Salt Lake City
• 3 Division of Hospital Medicine, Department of Pediatrics, Seattle Children’s Hospital, University of Washington, Seattle
• 4 Department of Pediatrics, Rochester General Hospital, Rochester, New York
• 5 Section of Hospital Medicine, Children’s Hospital Colorado, University of Colorado, Aurora
• 6 Section of Emergency Medicine, Children’s Hospital Colorado, University of Colorado, Aurora
• 7 Division of Hospital Medicine, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
• 8 The Hospital for Sick Children and Department of Pediatrics, University of Toronto, Toronto, Ontario, Canada
• 9 Pediatric Hospital Medicine, University of Florida COM-Jacksonville, Jacksonville
• 10 Department of Pediatrics, St Charles Hospital, Bend, Oregon
• 11 Division of Pediatric Hospital Medicine, Stanford University School of Medicine, Stanford, California
• 12 Dartmouth Health, Geisel School of Medicine at Dartmouth College, Lebanon, New Hampshire
• 13 Division of Pediatric Hospital Medicine, University of Nebraska Medical Center, Omaha
• 14 Division of Pediatric Hospital Medicine, University of California, San Francisco
• 15 Department of Pediatrics, Golisano Children’s Hospital, Rochester, New York
• 16 Department of Pediatrics, University of Rochester School of Medicine and Dentistry, Rochester, New York

Question   What are the most important and feasible randomized clinical trial (RCT) questions for children hospitalized with common conditions?

Findings   In this consensus statement, a 3-stage modified Delphi process involving multidisciplinary input from 46 individuals across 30 institutions produced 62 unique RCT questions, ranked according to importance and feasibility.

Meaning   These RCT questions can guide investigators and funders in conducting impactful trials to improve care and outcomes for children hospitalized with common conditions.

Importance   There is a lack of randomized clinical trial (RCT) data to guide many routine decisions in the care of children hospitalized for common conditions. A first step in addressing the shortage of RCTs for this population is to identify the most pressing RCT questions for children hospitalized with common conditions.

Objective   To identify the most important and feasible RCT questions for children hospitalized with common conditions.

Design, Setting, and Participants   For this consensus statement, a 3-stage modified Delphi process was used in a virtual conference series spanning January 1 to September 29, 2022. Forty-six individuals from 30 different institutions participated in the process. Stage 1 involved construction of RCT questions for the 10 most common pediatric conditions leading to hospitalization. Participants used condition-specific guidelines and reviews from a structured literature search to inform their development of RCT questions. During stage 2, RCT questions were refined and scored according to importance. Stage 3 incorporated public comment and feasibility with the prioritization of RCT questions.

Main Outcomes and Measures   The main outcome was RCT questions framed in a PICO (population, intervention, control, and outcome) format and ranked according to importance and feasibility; score choices ranged from 1 to 9, with higher scores indicating greater importance and feasibility.

Results   Forty-six individuals (38 who shared demographic data; 24 women [63%]) from 30 different institutions participated in our modified Delphi process. Participants included children’s hospital (n = 14) and community hospital (n = 13) pediatricians, parents of hospitalized children (n = 4), other clinicians (n = 2), biostatisticians (n = 2), and other researchers (n = 11). The process yielded 62 unique RCT questions, most of which are pragmatic, comparing interventions in widespread use for which definitive effectiveness data are lacking. Overall scores for importance and feasibility of the RCT questions ranged from 1 to 9, with a median of 5 (IQR, 4-7). Six of the top 10 selected questions focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis).

Conclusions and Relevance   This consensus statementhas identified the most important and feasible RCT questions for children hospitalized with common conditions. This list of RCT questions can guide investigators and funders in conducting impactful trials to improve care and outcomes for hospitalized children.

Randomized clinical trials (RCTs) are the criterion standard design to determine the efficacy or effectiveness of a given intervention. Although the number of RCTs involving adult participants has increased over time, the number of RCTs involving children has stalled in the last 2 decades. 1 The relative lack of RCTs including children results in pediatric clinicians more often having to make medical decisions based on observational studies, adult studies, or expert opinion.

Children are vulnerable to morbidity and mortality while hospitalized, making hospitalized children a priority population for RCTs. The pediatric RCTs that have led to the greatest recent improvements in outcomes for hospitalized children have focused on novel therapeutics for severe but relatively uncommon conditions, such as cystic fibrosis 2 and neuromuscular disease. 3 Randomized clinical trials for the most common pediatric conditions that lead to hospitalization are comparatively scarce. 4 For example, based on prevalence of disease, there are several hundred fewer RCTs than would be expected for childhood respiratory infections. 4

A first step toward conducting more RCTs involving common pediatric conditions for hospitalized children is to prioritize research questions for researchers and funders. The objective of this consensus statement was to identify the most important and feasible RCT questions for children hospitalized with common conditions.

In this consensus statement, we report on a 3-stage modified Delphi process conducted from January 1 to September 29, 2022 ( Figure ), and modeled after previous prioritization efforts incorporating clinician, researcher, and patient-parent perspectives. 5 - 7 Construction of RCT questions occurred during stage 1, followed by evaluation of trial question importance in stage 2 and feasibility in stage 3. This study was reviewed by the University of Utah institutional review board and received an exemption determination because the research included only interactions involving educational tests, survey procedures, interview procedures, or observation of public behavior. Participants provided written consent and were offered a $250 gift card for participating in the conference series.

We used purposive sampling to recruit study participants. The study team developed an initial expertise-based list of clinicians and researchers who were invited to participate. Patient-parent partners with affiliations to the study team and their institutions were also invited to participate. We then used a snowball sampling procedure to recruit participants with additional perspectives and expertise as needed. Participant race and ethnicity were reportedby the participants themselves. The US Department of Health and Human Services Office of Minority Health categories for race and ethnicity were used. These variables were collected as a measure of the diversity of conference participants. Ensuring a diversity of backgrounds and perspectives was prioritized in recruitment. For example, in addition to diversity of gender, race, and ethnicity, we sought diversity by clinical background (eg, nurses, pharmacists, and physicians) and practice environment (eg, children’s hospital and community hospital). Inclusion of community hospital participants was a priority because most children, particularly those hospitalized for common conditions, receive their care in settings other than freestanding children’s hospitals. 8

We began stage 1 of our modified Delphi process in January 2022 by identifying common pediatric conditions to serve as the basis for generating RCT questions. We used national hospitalization utilization data to identify the 10 most common pediatric conditions leading to hospitalization. 9 , 10 We excluded conditions for which surgical or other specialist clinicians may lead management (eg, appendicitis, diabetic ketoacidosis, and epilepsy). After applying this exclusion, we found that the 10 most common pediatric conditions (in decreasing order of prevalence) were birth hospitalization, bronchiolitis, pneumonia, asthma, mood disorders, cellulitis, neonatal hyperbilirubinemia, urinary tract infection, gastroenteritis, and septicemia. Together, these conditions accounted for more than 75% of all pediatric hospitalizations annually in the US. 9 , 10

A medical librarian (E.F.) performed structured literature searches for each condition. The medical librarian hand-searched the reference lists provided with each condition’s diagnosis and treatment summaries in Dynamed and UpToDate, 2 evidence-based clinical resources. Next, the librarian created structured literature searches in PubMed to capture relevant guidelines or evidence syntheses for each condition. The core strategy is available in the eFigure in Supplement 1 . Four authors (E.R.C., C.E.M., N.P., and S.V.K.) with pediatric hospital medicine expertise reviewed the search results and selected 3 to 5 high-yield resources for each condition that were most pertinent to understanding the evidence gaps related to diagnosis or management of each condition. For example, clinical practice guidelines with a section devoted to evidence gaps or research needs existed for most conditions and were included in the high-yield resources.

We divided participants into small groups (n = 10) for each condition, making sure that community and children’s hospital clinicians were represented in each small group. Each small group had at least 1 researcher with peer-reviewed publication expertise for that condition. Small groups had 2 months to review their high-yield resources, share additional resources with one another, and meet virtually to generate a list of at least 3 RCT questions for their assigned condition.

We conducted a virtual conference in June 2022 for stage 2 of our modified Delphi process. The purpose of stage 2 was to refine the RCT questions generated in stage 1 and evaluate them in terms of importance. Stage 2 was grounded within a framework of 6 guiding questions ( Box 1 ). This framework mirrored existing quality domains developed by the National Academy of Medicine. 11 Small groups took turns presenting their RCT questions to conference participants, sharing their impression of the importance of each question, receiving input from conference participants, and refining the questions. For each condition, patient-parent partners were asked to comment on patient centeredness of the presented RCT questions. We prioritized parent input within the discussion of each condition to ensure RCT questions were relevant and important to families. At the conclusion of the first conference, individual participants anonymously scored all the RCT questions for importance on a scale of 1 to 9, with 9 indicating the highest level of importance. To establish external validity of the generated questions, the RCT questions were then distributed for public comment to the American Academy of Pediatrics Society of Hospital Medicine listserv (approximately 4000 members). We invited listserv members to provide input and feedback on the generated questions through 3 mechanisms: (1) scoring each question on the same importance scale, (2) providing commentary on the questions, and (3) offering suggestions of additional important questions for consideration.

Framework for Evaluating the Importance of the RCT Question

To what extent will this RCT…

Answer a question that patients and their families care about (patient centeredness)?

Improve the patient experience?

Improve important clinical outcomes?

Increase efficiency or reduce variation in care delivery?

Improve safety?

Promote diversity, equity, and inclusion?

Abbreviation: RCT, randomized clinical trial.

We conducted a second virtual conference in September of 2022 for stage 3 of our modified Delphi process. The purpose of stage 3 was to incorporate feasibility into the prioritization of the RCT questions. The public comment and ratings of RCT questions generated in stage 2 were reviewed for incorporation and consideration. Small groups were given time to discuss the feasibility of their RCT questions guided by 3 core questions ( Box 2 ). Small groups group-scored each of their RCT questions on a feasibility scale of 1 to 9, where scores higher than 6 indicated RCT questions that were feasible for all 3 core feasibility questions, scores of 4 to 6 indicated RCT questions with a feasibility concern in 1 core feasibility question, and scores lower than 4 indicated RCT questions with a feasibility concern in more than 1 core feasibility question. Small groups then reported to the full group of conference participants, sharing their impressions and scores for the feasibility of each RCT question, with further input from conference participants.

Framework for Evaluating the Feasibility of the RCT Question

How straightforward, or not, would it be to administer the proposed intervention and control group requirements at your institution? Are the intervention and controls doable at your institution?

In your usual practice, how commonly do you care for patients who would be eligible for this trial (eg, weekly, monthly, and yearly)?

What challenges, if any, do you anticipate for collection of outcomes data in this trial?

At the conclusion of this conference, participants were provided the summary scores and comments related to the importance and feasibility of each question and asked to submit a final overall score for each question. This scoring was individual and anonymous, using a 1 to 9 scale, with higher scores indicating the most important and feasible RCT questions.

A total of 46 individuals from 30 different institutions participated in our modified Delphi process. Participants included children’s hospital (n = 14) and community hospital (n = 13) pediatricians, parents of hospitalized children (n = 4), other clinicians (ie, nurse and pharmacist; n = 2), biostatisticians (n = 2), and other researchers (eg, trialists and research coordinators; n = 11). Pediatric clinical expertise represented by these participants included hospital medicine (n = 27), general pediatrics (n = 3), infectious disease (n = 2), emergency medicine (n = 1), and critical care (n = 1). Participant geographic representation included Canada (n = 4) and all 4 US Census Bureau regions: West (n = 24), Midwest (n = 7), Northeast (n = 7), and South (n = 4). Conference participants who shared their demographic data (n = 38) self-identified as women (24 of 38 [63%]) and men (14 of 38 [37%]) and as having the following races and ethnicities: Asian ([4 of 38] 11%), Black or African American ([3 of 38] 8%), Hispanic ([2 of 38] 5%), Native Hawaiian or Other Pacific Islander ([1 of 38] 3%), and White ([30 of 38] 79%). A total of 115 listserv respondents provided comments and/or ratings of RCT questions generated in stage 2 of the modified Delphi process.

This process produced 62 unique RCT questions. Participant scores for the importance of each RCT question ranged from 1 to 9, with a median of 5 (IQR, 3-7). Participant overall scores, incorporating both importance and feasibility of the RCT question, ranged from 1 to 9, with a median of 5 (IQR, 4-7). The 10 RCT questions with the highest overall mean scores are summarized in the Table in a PICO (population, intervention, control, and outcome) format. The remaining 52 questions generated by this work and their mean scores are displayed in the eTable in Supplement 1 .

A total of 7 of 62 questions (11%) scored higher than 6 for importance. Five of those questions also had overall scores higher than 6, and all 5 of those questions were in the top 10 RCT questions by overall score. Six of the top 10 selected questions focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis). Two questions scored higher than 6 for importance but decreased below 6 after considering feasibility. These 2 questions involved antibiotic duration for children with bacteremia or meningitis and ultimately ranked 25th and 29th (eTable in Supplement 1 ).

For this consensus statement, we led a national, 3-stage modified Delphi process involving interdisciplinary, expert stakeholders, including patient-parent partners, to identify the most important and feasible RCT questions for children hospitalized with common conditions. Most of the RCT questions that we identified were pragmatic, comparing interventions in widespread use for which definitive effectiveness data are lacking. Our findings serve as an empirical foundation to guide investigators and funders in conducting impactful trials that improve outcomes for children hospitalized with common conditions.

The present research prioritization effort differs from existing prioritization studies in several fundamental ways. Some prior studies have focused on understanding condition prevalence, care variation, and costs for pediatric hospitalizations as a means to prioritize specific conditions for research. 9 , 12 , 13 Identification of condition-specific research topics or questions was outside the scope of these studies. Other prioritization studies have focused on broader (eg, all of pediatrics 6 ) or narrower (eg, chronic conditions 14 and palliative care 15 ) sets of conditions or settings that overlap with hospitalization (eg, emergency care 16 and patient safety 17 ). Nearly all existing prioritization studies, including a recent study focused on hospitalized children, 7 have produced priority topic areas (eg, What methods of communication are most effective between patients and clinicians?), as opposed to specific PICO-framed questions. Questions developed within existing prioritization studies were also not honed for a specific empirical research strategy, such as RCTs. To our knowledge, our research prioritization is unique in its focus on hospitalized children with common conditions and its development of specific PICO-framed questions designed to be readily answered in RCTs.

The questions that were prioritized in this process reflect broad goals of improving the value of care delivered to hospitalized children by avoiding unnecessary treatments. Six of the top 10 selected questions focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis). There is wide variation in antibiotic prescribing for these infections, raising concerns for potential antibiotic overtreatment and its detrimental effects for children (antibiotic-associated adverse effects), families (stress and costs), and communities (emergence of resistant organisms). 18 , 19 A growing body of RCT literature is demonstrating that less-aggressive antibiotic regimens are safe and effective for serious infections among adults (eg, pneumonia, bacteremia, osteomyelitis, and endocarditis), but trials on this topic remain rare for children, to our knowledge. 20 , 21

To address this ambitious list of RCT questions, investigators will likely need to augment traditional RCT designs with innovative and efficient RCT approaches. Many of the RCT questions developed in this study have natural overlap and could be considered for funding mechanisms that can support addressing multiple trial questions in a single application. For example, platform trials examine multiple research questions for a single condition. 22 Given that the present work generated multiple RCT questions for each condition, platform RCTs might be an efficient design to address more than 1 of these RCT questions at once. Similarly, factorial RCT designs could be used to address multiple questions at once when understanding the synergistic effect of 2 or more interventions is key. 23 As an example, 3 of the top 6 RCT questions developed here involve children hospitalized with urinary tract infections and their antibiotic regimens. The single condition entity and similar interventions could lend themselves to a platform or factorial design. Randomized clinical trial questions developed here may also lend themselves to a high-efficiency randomized controlled (HEROIC) trial approach, which uses a dispersed enrollment strategy to efficiently conduct pragmatic RCTs. 24

The consensus statement has several important limitations. First, these RCT questions will require further refinement as the trial is designed, including additional definitions for each intervention and careful consideration of the right primary outcome. Second, our consideration of feasibility focused on trial infrastructure (the ability to recruit, administer intervention or control, and collect data). This feasibility assessment was within the scope of conference participant skills and resources. During formal trial design or preparation, an additional feasibility assessment would be completed, including human participants protection review, confirmation of adequate numbers of eligible participants at proposed study sites, formal sample size estimates, power calculations, and budget or cost estimates. These considerations will undoubtedly influence the feasibility of each RCT question. Third, conference participants were diverse in many ways but did not represent all perspectives pertinent to RCT question generation in this population. Fourth, RCT question development was organized around common conditions. Undoubtedly, there are additional important and feasible questions that are not condition based (eg, RCTs comparing processes such as transitions of care) or that are focused on less-common conditions. Fifth, our 3-stage process could have been subject to anchoring bias, in which decisions made early in the process by smaller numbers of participants are difficult to reconsider later in the process. Sixth, investigators who pursue the RCTs described in this study will still contend with pervasive impediments to RCTs for children, such as relatively modest disease prevalence, lower frequency of objective outcome measures (eg, mortality), and fewer sources of funding compared with RCTs for adult populations. For example, the 2 questions that scored higher than 6 for importance but then ultimately decreased below 6 after considering feasibility involved less-common subpopulations of hospitalized children (children with bacteremia or meningitis). For RCT questions with particularly low feasibility scores, observational study designs might be more practical.

In this consensus statement, we have identified the most important and feasible RCT questions for children hospitalized with common conditions. These conditions are responsible for more than three-fourths of pediatric hospitalizations. Answering these pressing questions with RCTs has great potential to improve care and outcomes for hospitalized children.

Accepted for Publication: March 13, 2024.

Published: May 15, 2024. doi:10.1001/jamanetworkopen.2024.11259

Open Access: This is an open access article distributed under the terms of the CC-BY License . © 2024 Coon ER et al. JAMA Network Open .

Corresponding Author: Eric R. Coon, MD, MS, Department of Pediatrics, University of Utah School of Medicine, 100 N Mario Capecchi Dr, Salt Lake City, UT 84113 ( [email protected] ).

Author Contributions: Dr Coon had full access to all of the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis.

Concept and design: Coon, McDaniel, Paciorkowski, Jennings, Wilson, Kaiser.

Acquisition, analysis, or interpretation of data: Coon, McDaniel, Paciorkowski, Grimshaw, Frakes, Ambroggio, Auger, Cohen, Garber, Gill, Joshi, Leyenaar, McCulloh, Pantell, Sauers-Ford, Schroeder, Srivastava, Wang, Wilson, Kaiser.

Drafting of the manuscript: Coon, Frakes, Gill, Pantell.

Critical review of the manuscript for important intellectual content: Coon, McDaniel, Paciorkowski, Grimshaw, Frakes, Ambroggio, Auger, Cohen, Garber, Gill, Jennings, Joshi, Leyenaar, McCulloh, Sauers-Ford, Schroeder, Srivastava, Wang, Wilson, Kaiser.

Statistical analysis: Coon.

Obtained funding: Coon, Wilson, Kaiser.

Administrative, technical, or material support: Grimshaw, Ambroggio, Gill, McCulloh, Wilson, Kaiser.

Supervision: Wilson, Kaiser.

Conflict of Interest Disclosures: Dr Ambroggio reported receivng grants from Pfizer Inc outside the submitted work. Dr Cohen reported being a member of the Committee to Evaluate Drugs, which provides advice to the Ministry of Health on Ontario’s Public Drug Policy. Dr Gill reported receiving grants from Physicians’ Services Incorporated Foundation, Canadian Institutes of Health Research (CIHR), and Hospital for Sick Children outside the submitted work. Dr McCulloh reported receiving grants from the National Institutes of Health from the Office of the Director during the conduct of the study and grants from Merck Inc outside the submitted work. Dr Schroeder reported receiving grants from the Patient-Centered Outcomes Research Institute outside the submitted work. Dr Srivastava reported being a physician founder of the IPASS Patient Safety Institute, and his equity is owned by his employer, Intermountain Health, outside the submitted work; receiving grants from the Patient-Centered Outcomes Research Institute, National Institutes of Health, and Centers for Disease Control and Prevention; and receiving monetary awards, honoraria, and travel reimbursement from multiple academic and professional organizations for teaching and consulting on quality of care and spreading evidence-based best practices in health systems and pediatric hospital medicine. No other disclosures were reported.

Funding/Support: This study was financially supported by the Agency for Healthcare Research and Quality, US Department of Health and Human Services and the Division of Pediatric Hospital Medicine, the University of California San Francisco.

Role of the Funder/Sponsor: The funding sources had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.

Group Information: The members of the RCT conference series group are listed in Supplement 2 .

Disclaimer: The contents are those of the authors. They may not reflect the policies of the US Department of Health and Human Services or the US government.

Data Sharing Statement: See Supplement 3 .

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Researchers discover new pathway to cancer cell death from chemotherapy

by Netherlands Cancer Institute

Chemotherapy kills cancer cells. But the way these cells die appears to be different than previously understood. Researchers from the Netherlands Cancer Institute, led by Thijn Brummelkamp, have uncovered a completely new way in which cancer cells die: due to the Schlafen11 gene.

"This is a very unexpected finding. Cancer patients have been treated with chemotherapy for almost a century, but this route to cell death has never been observed before. Where and when this occurs in patients will need to be further investigated. This discovery could ultimately have implications for the treatment of cancer patients ." They published their findings in Science .

Many cancer treatments damage cell DNA. After too much irreparable damage, cells can initiate their own death. High school biology teaches us that the protein p53 takes charge of this process. p53 ensures repair of damaged DNA, but initiates cell suicide when the damage becomes too severe. This prevents uncontrolled cell division and cancer formation.

Surprise: Unanswered question

That sounds like a foolproof system, but reality is more complex. "In more than half of tumors, p53 no longer functions," says Brummelkamp. "The key player p53 plays no role there. So why do cancer cells without p53 still die when you damage their DNA with chemotherapy or radiation? To my surprise, that turned out to be an unanswered question."

His research group then discovered, together with the group of colleague Reuven Agami, a previously unknown way in which cells die after DNA damage. In the lab, they administered chemotherapy to cells in which they carefully modified the DNA. Brummelkamp says, "We were looking for a genetic change that would allow cells to survive chemotherapy. Our group has a lot of experience in selectively disabling genes , which we could perfectly apply here."

New key player in cell death

By switching off genes, the research group found a new pathway to cell death headed by the gene Schlafen11 (SLFN11). Principle investigator Nicolaas Boon said, "In the event of DNA damage, SLFN11 shuts down the protein factories of cells: the ribosomes. This causes immense stress in these cells, which leads to their death. The new route we discovered completely bypasses p53."

The SLFN11 gene is not unfamiliar in cancer research . It is often inactive in tumors of patients who do not respond to chemotherapy, says Brummelkamp. "We can now explain this link. When cells lack SLFN11 they will not die in this manner in response to DNA damage. The cells will survive and the cancer persist."

Impact on cancer treatment

"This discovery uncovers many new research questions, which is usually the case in fundamental research," says Brummelkamp.

"We have demonstrated our discovery in lab-grown cancer cells , but many important questions remain: Where and when does this pathway occur in patients? How does it affect immunotherapy or chemotherapy? Does it affect the side effects of cancer therapy? If this form of cell death also proves to play a significant role in patients, this finding will have implications for cancer treatments. These are important questions to investigate further."

Turning off genes, one by one

People have thousands of genes, many of which have functions that are unclear to us. To determine the roles of our genes, researcher Brummelkamp developed a method using haploid cells. These cells contain only one copy of each gene, unlike the regular cells in our bodies that contain two copies. Handling two copies can be challenging in genetic experiments, because changes (mutations) often occur in just one of them. This makes it difficult to observe the effects of these mutations.

Together with other researchers, Brummelkamp has been unraveling processes that are crucial in disease for years using this versatile method. For example, his group recently discovered that cells can make lipids in a different way than previously known.

They uncovered how certain viruses, including the deadly Ebola virus, manage to enter human cells. They delved into cancer cell resistance against specific therapies and identified proteins that act as brakes on the immune system , which is relevant to cancer immunotherapy.

Over the last years, his team discovered two enzymes that had remained elusive for four decades, and that turned out to be vital for muscle function and brain development.

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30 Medical Researcher Interview Questions and Answers

Common Medical Researcher interview questions, how to answer them, and example answers from a certified career coach.

Working as a medical researcher requires more than just an extensive understanding of the scientific method and clinical procedures. It demands critical thinking, creativity, and above all, an unwavering dedication to improving healthcare outcomes through rigorous research.

However, when it comes to landing that dream position in this challenging field, the interview process can often feel like a daunting experiment itself. That’s where we come in. In this article, we’ll decode some commonly asked medical researcher interview questions, providing you with insightful tips and sample answers to help you confidently articulate your skills, experience, and passion for medical research.

1. Can you describe a research project where you had to design and execute a complex study?

As a medical researcher, your ability to design and implement intricate studies is paramount. Your interviewer wants to see evidence of your methodological skills, creativity, and problem-solving abilities. They’re interested in how you approach research challenges and how you adapt when circumstances change or unexpected results occur. This question gives them insight into your thought process and how your skills might benefit their organization.

Example: “In one of my recent projects, I was tasked with assessing the efficacy of a new drug for Alzheimer’s disease. We designed a double-blind, placebo-controlled study involving 500 participants over two years.

The complexity lay in ensuring participant adherence to medication schedules and maintaining blinding protocols. Furthermore, we had to account for potential confounding variables like age, lifestyle factors, and genetic predispositions.

We used advanced statistical methods to analyze our data, which showed promising results for the drug. This project not only honed my research design skills but also underscored the importance of meticulous execution in clinical trials.”

2. What is your process for formulating a hypothesis and designing an experiment to test it?

The question aims to gauge your scientific acumen and problem-solving abilities. As a medical researcher, you’ll be expected to develop hypotheses and design experiments to test them regularly. Your ability to do this effectively and efficiently directly impacts the success of your research. Therefore, interviewers ask this question to understand your thought process, creativity, and analytical skills.

Example: “Formulating a hypothesis in medical research often begins with an observation or question about a particular phenomenon or treatment effect. This leads to a preliminary investigation, including literature review and data analysis, which helps shape the hypothesis.

To design an experiment, I consider factors such as the study population, control groups, variables, and methods of measurement. It’s crucial to ensure that the experiment is ethically sound, statistically valid, and practically feasible.

Once the experiment is conducted, results are analyzed against the original hypothesis. If the findings support it, further testing may be needed for validation. If they don’t, the hypothesis might need refining before repeating the process.”

3. How do you approach data analysis and interpretation in your research?

For a medical researcher, the ability to analyze and interpret data is key. It’s the core of what you do—you conduct experiments and studies, collect data, and then analyze that data to draw conclusions. It’s not enough to simply gather information; you need to be able to understand what that information means and how it fits into the larger context of your research. That’s why hiring managers want to know how you approach this critical aspect of your job.

Example: “In my research, I approach data analysis and interpretation through a systematic process. Initially, I define the specific objectives of the study to guide the analytical strategy.

I then collect high-quality, relevant data ensuring its integrity. Following this, I use statistical methods tailored to the dataset and research question. This could range from descriptive statistics to more complex predictive models.

Interpretation is done in the context of the original hypothesis and existing literature. It’s crucial not just to identify patterns but understand their implications for broader scientific understanding or clinical practice.

Throughout, transparency and reproducibility are key. I document every step, enabling others to verify results, contributing to robust, reliable science.”

4. How would you manage a situation where your research findings contradict existing medical knowledge?

This question is a test of your scientific integrity, your ability to handle unexpected results, and your courage to challenge existing paradigms. In the field of medical research, new discoveries often contradict or challenge established knowledge. Therefore, a researcher’s ability to objectively evaluate, validate, and communicate such findings is crucial in driving scientific progress and improving patient care.

Example: “In such a situation, I would first re-evaluate my methodology to ensure there are no errors. If the results still stand, I’d seek peer review for unbiased feedback. It’s crucial not to dismiss new findings just because they contradict existing knowledge. Instead, it should spark further investigation. Medical science is continually evolving and challenging established theories is part of that process. The key is to be transparent about the process and open to critique.”

5. How have you ensured ethical considerations are met in your previous research?

Ethics play a vital role in medical research. It’s not just about making groundbreaking discoveries, but also about ensuring that the methods used to reach those discoveries respect the rights, safety, and well-being of all participants involved. By asking this question, hiring managers are looking to assess your understanding and commitment to ethical research practices, which is an essential quality for anyone working in this field.

Example: “In my previous research, I ensured ethical considerations were met by strictly adhering to the guidelines set out in the Declaration of Helsinki. This included obtaining informed consent from all participants, ensuring their anonymity and confidentiality, and making sure that they understood they could withdraw at any time without consequence.

I also worked closely with an ethics review board to ensure our study design was ethically sound. Their input was invaluable in identifying potential ethical issues before we began collecting data.

Moreover, I prioritized transparency in our findings, sharing results openly while maintaining participant privacy. This not only fostered trust but also promoted accountability within our research team.”

6. Can you discuss a research project you led that significantly contributed to medical science?

Medical research is a field that relies heavily on innovation and progress. As such, interviewers want to know if you’ve made significant contributions in the past, as this can serve as a predictor for future success. They are interested in understanding your research skills, your ability to lead a team, your problem-solving capabilities, and how your work can impact the medical field. They also want to gauge your passion and dedication to advancing medical science.

Example: “I led a research project investigating the potential of CRISPR gene-editing technology in treating genetic diseases. We focused on cystic fibrosis, a disease currently without a cure.

Our team developed a method to deliver the CRISPR system into lung cells effectively. This was a significant challenge due to the mucus barrier in these patients’ lungs.

The results were promising. In lab tests, we successfully edited the faulty CFTR gene causing the disease. The study contributed valuable insights into using CRISPR for genetic disorders and paved the way for future clinical trials.”

7. How do you stay updated with the latest advancements in your field of medical research?

Keeping abreast of the latest advancements in medical research is a key aspect of the role. In a field that is constantly evolving and where new discoveries are made on a regular basis, hiring managers need to ensure that you’re someone who takes the initiative to stay updated. Your ability to keep up with recent findings, techniques, or technologies could potentially influence the success of your research projects.

Example: “To stay updated with the latest advancements in medical research, I regularly read peer-reviewed journals such as The New England Journal of Medicine and The Lancet. These publications provide cutting-edge findings from reputable researchers worldwide.

I also attend industry conferences, both physically and virtually, which offer opportunities to learn about new methodologies and technologies directly from their creators.

Participating in online forums and communities for medical researchers is another way I keep myself informed. They serve as platforms for knowledge exchange on current trends and challenges in our field.

Lastly, I am a member of professional organizations that provide continuous learning resources and updates on regulatory changes affecting medical research.”

8. Explain a situation where you had to make a critical decision during a research project. What was the outcome?

Deep in the heart of medical research, decisions must be made that could potentially impact the outcome of the entire study. Your ability to navigate these choppy waters confidently and effectively is a key quality that employers are looking for. They want to know that you can handle the pressure and make sound decisions when the stakes are high.

Example: “During a clinical trial, we noticed an unexpected side effect in some participants. It was crucial to decide whether to halt the study or adjust our approach.

After consulting with my team and reviewing data, I decided to modify the dosage instead of stopping the trial. This decision was based on the fact that the side effects were not life-threatening and the potential benefits outweighed the risks.

The outcome was positive; the adjusted dosage reduced the side effects while maintaining efficacy. This experience emphasized the importance of flexibility and swift decision-making in research.”

9. Can you describe a time when you had to manage a research budget effectively?

In the role of a medical researcher, you’re not just conducting experiments and analyzing data. You’re also in charge of managing resources, including the funding that makes your research possible. This question is designed to test your ability to balance scientific aspirations with financial realities, and to ensure that you can deliver results without exceeding your budget.

Example: “In one of my past projects, I was responsible for managing a tight budget while conducting clinical trials. We had to be very strategic about resource allocation.

To manage this effectively, I prioritized tasks based on their importance and cost-effectiveness. For instance, we utilized in-house resources wherever possible instead of outsourcing, which significantly cut costs.

Regular monitoring of the budget also played a crucial role in ensuring that we stayed within our limits. By doing so, we were able to complete the project successfully without exceeding our budget. This experience honed my skills in budget management in a research setting.”

10. How have you handled a situation where your research did not produce the expected results?

Science, by its very nature, is about exploration and discovery, and not every hypothesis will lead to the expected results. By asking this question, hiring managers are interested in understanding how you deal with the inevitable setbacks and unexpected outcomes that come with research. They want to know if you can critically analyze your own work, learn from your mistakes, and use these experiences to improve your future research.

Example: “In research, unexpected results are not uncommon. In such a case, I first reassess my methodology to ensure there were no errors in the process. If everything checks out, I interpret these findings as new insights rather than failures.

For instance, during a project on drug interactions, my team and I found that our hypothesis was incorrect. Instead of discarding the data, we re-evaluated it from different angles which led us to discover an entirely new interaction. This experience taught me that ‘unexpected’ can often mean ‘unexplored’.

It’s crucial to remain flexible and open-minded in scientific research because sometimes, the most significant discoveries come from the least expected outcomes.”

11. Describe your experience with grant writing and funding acquisition for research projects.

Securing funding is a crucial part of any researcher’s job, especially in the medical field. Without it, the important work you plan to do simply can’t happen. Hiring managers ask about your experience with grant writing and funding acquisition to gauge your abilities in this area. They want to know that you can effectively communicate the value and potential impact of your research to secure the necessary resources.

Example: “I have extensive experience in grant writing and funding acquisition for research projects. I’ve successfully secured grants from both private foundations and government agencies, including the National Institutes of Health.

My approach involves a deep understanding of the project’s objectives and aligning them with the funder’s mission. I focus on clear, concise writing that highlights the significance of the research, its potential impact, and our team’s expertise to execute it.

In terms of success rate, about 70% of my proposals have been funded, which has enabled the continuation of vital medical research. My ability to secure necessary funding is something I take pride in and would bring to this role.”

12. Discuss a time when you collaborated with professionals from other disciplines for a research project.

The field of medical research often involves a multidisciplinary approach, combining insights from various fields to solve complex health problems. Therefore, hiring managers want to know if you have the ability to successfully collaborate across disciplines. Your ability to work effectively in a team, respect diverse viewpoints, and integrate different perspectives to achieve a common goal is vital in this role.

Example: “During my PhD, I was part of a multi-disciplinary team investigating the genetic basis of Alzheimer’s disease. Our team included neurologists, geneticists, and bioinformaticians.

I collaborated with the neurologists to understand the clinical manifestations of the disease. The geneticists helped me grasp how certain genes could influence disease progression. With the bioinformaticians, we analyzed large genomic datasets.

This collaboration not only enriched our research but also broadened my understanding of the interplay between different disciplines in medical research.”

13. How do you handle disagreements or conflicts within your research team?

The dynamics of a research team are critical to its success. As a medical researcher, you’ll be part of a diverse team with varied opinions and perspectives. Disagreements are bound to happen. Hiring managers want to know if you have the emotional intelligence and the skills to resolve conflicts in a respectful, productive manner that maintains team cohesion and keeps the research project on track.

Example: “When disagreements arise within my research team, I believe in addressing them openly and respectfully. It’s crucial to listen to all perspectives, as each member brings unique expertise that can contribute to the solution.

I encourage open communication and promote a culture of respect for everyone’s ideas. This approach often leads to compromise or innovative solutions that we hadn’t previously considered.

If conflicts persist, involving a neutral third party like a supervisor or HR representative can be beneficial. They can provide an impartial perspective and help mediate the situation.

In essence, handling disagreements involves fostering a respectful environment, promoting open dialogue, and seeking external assistance when necessary.”

14. Discuss your experience with conducting clinical trials.

Clinical trials are a critical aspect of medical research. They are the final hurdle before a new treatment or drug becomes available to the public. As such, experience in conducting these trials is a highly sought-after skill. Employers want to know that you understand the intricacies of trial design, ethical considerations, and data analysis. Your ability to successfully navigate these complexities can directly affect the speed and efficiency of bringing new medical solutions to those who need them.

Example: “I have extensive experience in conducting clinical trials, particularly phase II and III studies. I’ve been involved in all stages of the process, from designing protocols to monitoring data collection.

My expertise lies in ensuring that trials are carried out ethically and efficiently while maintaining rigorous scientific standards. This includes managing patient recruitment, overseeing treatment administration, and analyzing trial results.

I also understand the importance of clear communication with stakeholders such as patients, healthcare providers, and regulatory authorities. I am adept at presenting complex data in an accessible manner, which is crucial for informed decision-making in clinical research.”

15. How do you ensure the accuracy and reliability of your research data?

As a medical researcher, your work could potentially influence health policy, medical practices, or even the development of new treatments and medicines. That’s why it’s essential to demonstrate a commitment to rigorous, accurate data collection and analysis. This question is a way for hiring managers to gauge your understanding of the importance of reliable, high-quality research and your ability to implement effective controls to ensure this.

Example: “To ensure the accuracy and reliability of research data, I adopt a multi-faceted approach.

I start with rigorous experimental design which includes clear objectives, appropriate sample sizes, and suitable controls. This helps minimize bias and increase the validity of results.

Data collection is standardized to maintain consistency across all measurements. Any deviations are documented for transparency.

Quality control measures such as calibration of equipment and validation of methods are implemented regularly.

Statistical analysis is employed to evaluate the significance of the findings. The use of robust statistical tools can help identify trends and patterns in the data that may not be immediately apparent.

Lastly, peer review is an important aspect. It provides an additional layer of scrutiny, ensuring the research methodology and conclusions drawn are sound.”

16. Can you describe a time when you had to adapt your research methods due to unforeseen circumstances?

In the unpredictable world of scientific research, things don’t always go according to plan. Your ability to adapt your research methods when faced with unexpected circumstances is a testament to your problem-solving skills and resilience. This question is designed to understand how you adjust your strategies, think on your feet, and handle challenges to keep your research on track.

Example: “During my PhD, I was investigating the efficacy of a new drug on lung cancer cells. However, after several weeks into the project, the supplier discontinued the specific cell line we were using.

I had to quickly adapt and find an alternative that would not compromise the integrity of our study. After extensive literature review, I identified another cell line with similar characteristics.

We ran preliminary tests to ensure it responded similarly to the original one. This unexpected change actually led us to discover some interesting differences between the two lines, adding depth to our research findings.”

17. How have you contributed to the development of research protocols in your previous roles?

This question is meant to gauge your hands-on experience in the critical stages of medical research. Developing a research protocol involves defining the purpose, scope, and methods of the study, which are all essential for its success. By asking this question, hiring managers are interested in understanding your ability to contribute to this process, showcasing your knowledge, critical thinking, and problem-solving skills.

Example: “In the realm of medical research, I have been involved in creating and refining protocols. For instance, during a clinical trial on a new drug, I helped design the protocol to ensure it met ethical standards and regulatory requirements.

I also contributed to developing data collection methods that were both effective and efficient. This included determining what information was necessary, how it should be collected, and how frequently.

Moreover, I’ve worked closely with teams to train them on these protocols, ensuring they understand their importance and how to adhere to them correctly. This not only ensures consistency but also improves the quality of our findings.”

18. What is your approach to presenting research findings to a non-scientific audience?

As a medical researcher, you may often find yourself in a position where you need to communicate complex scientific concepts to a wider audience. This could include patients, funding bodies, or even the general public. It’s important that you can demonstrate the ability to translate these complex ideas into language and concepts that non-scientists can understand and appreciate. That’s why hiring managers are keen to understand your approach to such situations.

Example: “When presenting research findings to a non-scientific audience, I focus on simplifying the complex data into understandable concepts. This involves using layman’s terms and avoiding jargon as much as possible.

I also find it effective to use visual aids such as infographics or charts. They can illustrate key points in an easily digestible format.

It’s crucial to highlight the relevance of the research to their lives or work. By connecting the information to something they are familiar with, it becomes more relatable and easier to comprehend.

Lastly, I encourage questions and feedback throughout the presentation. This not only ensures understanding but also promotes engagement from the audience.”

19. Have you ever faced a situation where you had to defend your research findings? How did you handle it?

As a medical researcher, your work is constantly being scrutinized by peers, regulatory bodies, and even the public. Not only does this question assess your ability to stand by your work and defend it with confidence, but it also provides insight into your ability to handle criticism, feedback, and pressure. It’s all about demonstrating resilience and commitment to scientific integrity.

Example: “Yes, during my post-doctoral research, I discovered a potential link between certain genetic markers and the development of Alzheimer’s disease. However, some colleagues were skeptical due to the small sample size.

I defended my findings by emphasizing the rigorous statistical analysis used and the promising preliminary results. I also proposed a larger study to further validate these findings.

This experience taught me the importance of constructive criticism and open dialogue in scientific research. It’s crucial to stand behind your work while remaining open to feedback for improvement.”

20. How do you handle the pressure and stress that comes with deadlines and high expectations in research?

The fast-paced, high-stakes world of medical research is not for the faint-hearted. It’s a field where the pressures of deadlines and the high expectations for accuracy and breakthroughs can be intense. As a potential candidate, hiring managers want to ensure you have the resilience, problem-solving skills, and coping mechanisms to thrive and deliver in such an environment. This question helps them assess your stress-management strategies and your ability to perform under pressure.

Example: “In managing stress and pressure, I prioritize my tasks based on their urgency and importance. This helps me to focus on what needs immediate attention.

I also believe in the power of teamwork. Collaborating with colleagues not only distributes workload but also allows for diverse perspectives that can enhance research quality.

Moreover, maintaining a balanced lifestyle is crucial. Regular exercise, good nutrition, and adequate rest help maintain mental clarity and resilience under pressure.

Lastly, I embrace challenges as opportunities for growth. High expectations inspire me to push beyond my limits and deliver exceptional results.”

21. Describe a time when your research findings had a direct impact on patient care.

The crux of medical research is to improve patient care, whether through developing new treatments, improving diagnostic techniques, or enhancing the understanding of diseases. Therefore, hiring managers want to ensure that you, as a medical researcher, are driven by the ultimate goal of positively impacting patient care. By sharing an instance where your research directly influenced patient care, you demonstrate your ability to make meaningful contributions to the field.

Example: “During my postgraduate studies, I was part of a team researching the efficacy of a new diabetes medication. Our findings indicated that this drug significantly improved blood sugar control and reduced side effects compared to existing treatments.

Based on our research, local physicians started prescribing this medication to their diabetic patients. This led to improved health outcomes in our community, including lower hospitalization rates due to complications from diabetes. It was rewarding to see how our work directly influenced patient care and overall public health.”

22. How do you ensure the confidentiality and privacy of subjects during clinical trials?

As a medical researcher, you’re entrusted with a great deal of sensitive information. Participants in clinical trials need to be confident that their personal and health information will be protected. That’s why interviewers want to know how you handle this critical aspect of the job. They’re looking for evidence of your commitment to ethical practices, and your understanding of the laws and regulations surrounding privacy and confidentiality in medical research.

Example: “Ensuring confidentiality and privacy in clinical trials is paramount. This can be achieved through anonymizing participant data, using only unique identifiers instead of personal information. Informed consent forms should clearly explain how personal data will be used and protected.

Strict access controls to sensitive data are also crucial. Only authorized personnel should have access, and they should be trained on data handling procedures.

Moreover, regular audits can help identify any potential breaches or weaknesses in the system. It’s essential to comply with all relevant regulations such as HIPAA in the US or GDPR in Europe, which provide robust frameworks for protecting patient data.”

23. What strategies do you use to manage multiple research projects simultaneously?

Multitasking is an essential part of being a medical researcher. With various projects running concurrently, each with their own timelines, objectives, and challenges, it’s critical to demonstrate you have the ability to manage multiple assignments without sacrificing quality or missing deadlines. This question is asked to assess your organizational abilities, time management skills, and how you prioritize tasks.

Example: “To manage multiple research projects, I prioritize tasks based on deadlines and importance. I also use project management tools to track progress and ensure timely completion.

Effective communication is key in coordinating with team members and stakeholders. Regular updates help keep everyone informed about the project status.

I believe in setting realistic goals and breaking down larger tasks into manageable sub-tasks. This helps in maintaining focus and reducing overwhelm.

Lastly, I always have a contingency plan in place for unexpected hurdles. This proactive approach allows me to stay ahead of any potential issues that may arise during the course of the research.”

24. Can you discuss a time when your research findings influenced policy change?

As a medical researcher, your work doesn’t end at the laboratory. It’s about using the findings to make a tangible, positive impact on health policies and practices. That’s why hiring managers want to see whether you have the ability to translate your research findings into actionable policy changes, thereby demonstrating your influence and effectiveness in the broader healthcare landscape.

Example: “During my work on a project investigating the impact of smoking on lung cancer, we found that passive smokers were at significant risk as well. Our research findings indicated that non-smokers living with smokers had an increased chance of developing lung cancer compared to those not exposed to secondhand smoke.

These findings triggered discussions with health policymakers. We presented our data and suggested stricter regulations on smoking in public places to protect non-smokers from involuntary exposure. Subsequently, this led to the implementation of more stringent anti-smoking laws in several states, including banning smoking in certain public areas. This experience demonstrated how impactful medical research can be in shaping health policies for the greater good.”

25. How do you approach problem-solving when you encounter obstacles in your research?

Problem-solving is at the heart of medical research. Whether you’re conducting clinical trials or studying medical data, you’ll inevitably encounter roadblocks. Your ability to navigate these challenges, adapt your plans, and continue to make progress is essential. This question is designed to assess your problem-solving skills, resilience, and adaptability. It also allows the interviewer to gain insight into your thought processes and how you handle pressure and setbacks.

Example: “When I encounter obstacles in my research, I first analyze the problem to understand its root cause. This often involves revisiting data, reassessing methodologies or consulting with colleagues for fresh perspectives.

Next, I explore alternative approaches or solutions. This could mean adopting new techniques, adjusting protocols, or even seeking external expertise when necessary.

Throughout this process, it’s important to maintain a flexible mindset and be open to learning from these challenges. Each obstacle is an opportunity to improve our understanding and refine our approach.”

26. Discuss your experience with peer-reviewed publications. How many have you authored or co-authored?

This question is a way for hiring managers to gauge your experience with the research and publishing process. Peer-reviewed publications are the gold standard in academic research, so your involvement in them demonstrates your ability to conduct high-quality research. Furthermore, your authorship or co-authorship of these publications can indicate your contributions to your field, your ability to collaborate with colleagues, and your dedication to advancing medical knowledge.

Example: “I have extensive experience with peer-reviewed publications in the field of medical research. I’ve authored 15 articles and co-authored another 10, each one focusing on different aspects of oncology. My work often involves complex laboratory experiments, data analysis, and collaboration with other researchers. The publication process has taught me the importance of meticulous attention to detail and clear communication of findings. It’s a rigorous but rewarding process that ensures the quality and credibility of my research.”

27. How proficient are you in using research software and technology?

The landscape of medical research is constantly evolving, with advancements in technology and software playing a significant role. Your ability to navigate and utilize these tools effectively is critical. It not only demonstrates your technical aptitude but also your adaptability to changing technology platforms. This question is asked to assess your readiness to jump right into the role and contribute to ongoing research effectively.

Example: “I am highly proficient in using research software and technology. I have extensive experience with statistical analysis tools such as SPSS, SAS, and R which are crucial for data interpretation. My skills also extend to bioinformatics software like BLAST and Bioconductor for genetic sequence analysis.

In terms of technology, I’m adept at utilizing laboratory information management systems (LIMS) for managing samples and associated data. I’ve also used electronic health record systems for patient data collection and analysis.

My proficiency extends beyond just usage; I understand the underlying principles guiding these tools, enabling me to troubleshoot issues or adapt to new software quickly. This technical savviness is complemented by my ability to stay updated on emerging technologies relevant to medical research.”

28. Can you describe a time when you challenged a traditional method or theory through your research?

Medical research is an ever-evolving field, and sometimes, progress means challenging established norms. Interviewers want to see that you have the courage, creativity, and scientific rigor to question traditional methods or theories when you believe there could be a more effective approach. Your ability to do so could lead to significant advances in medical science and patient care, which is the ultimate goal for any healthcare organization.

Example: “In my previous research on cancer treatments, I questioned the efficacy of chemotherapy as a standalone treatment. My team and I hypothesized that personalized medicine could potentially enhance treatment outcomes.

We conducted comprehensive genomic profiling on tumor samples to identify individual genetic mutations. This allowed us to recommend targeted therapies for each patient, which were designed to specifically attack their unique cancer cells.

The results showed significant improvement in patient recovery rates and reduced side effects compared to traditional chemotherapy. This research challenged the one-size-fits-all approach in cancer treatment, paving the way for more personalized care.”

29. How do you handle criticism and feedback on your research work?

As a medical researcher, you’ll be in the spotlight, with your work scrutinized by peers, stakeholders, and sometimes the public. Handling criticism and feedback effectively is essential to refining your research, improving your professional skills, and ensuring that your work stands up to rigorous examination. This question gives you an opportunity to demonstrate your resilience, openness to learning, and commitment to excellence in your research.

Example: “Criticism and feedback are crucial for growth in any field, including medical research. I view them as opportunities to improve my work and expand my knowledge base.

When I receive criticism, I first try to understand the perspective of the person providing it. This helps me grasp their point of view and apply it effectively.

I believe in maintaining an open dialogue with colleagues and mentors. If there’s something unclear or if I disagree, I discuss it openly to reach a constructive conclusion.

Ultimately, my goal is to produce high-quality research that contributes to medical advancements. Therefore, I welcome all feedback that aids this objective.”

30. How would you incorporate patient perspectives or experiences in your research?

Medical research isn’t just about cells, genes, and molecules. It’s about people. A project can only be successful if it acknowledges the needs and experiences of the patients it aims to help. By asking this question, hiring managers aim to gauge your ability to bridge the gap between the laboratory and the real world, ensuring the research is not only scientifically robust but also relevant and beneficial to the patients.

Example: “Incorporating patient perspectives in research is crucial to ensure its relevance and applicability. I would use qualitative methods, like interviews or focus groups, to gather patients’ experiences and views on the condition under study. This can provide insights into how a disease impacts their daily life, which may not be captured by quantitative measures alone.

Also, involving patients in designing the research itself can help tailor it to address their concerns. For instance, they could help formulate research questions, select outcomes of interest, or review study materials for clarity and appropriateness.

Moreover, Patient Reported Outcome Measures (PROMs) can be used to assess treatment effectiveness from the patient’s perspective. These tools measure aspects such as quality of life, symptom severity, and functional status, providing a more holistic view of the intervention’s impact.”

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Physiological Questions, Inventive Approaches

Four vp&s scientists receive schaefer research scholar awards, share this page.

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Four scientists at Columbia University Vagelos College of Physicians and Surgeons have received awards from the Schaefer Research Scholars Program , made possible through a bequest from Dr. Ludwig Schaefer. Each award consists of a $50,000 cash prize and up to $200,000 in direct research support.

Two awardees are full-time VP&S faculty and two are visiting faculty who are collaborating with VP&S faculty:

• Iok In Christine Chio , assistant professor, Institute for Cancer Genetics, Columbia University Vagelos College of Physicians and Surgeons
• Xuebing Wu , assistant professor of medicine, Columbia University Vagelos College of Physicians and Surgeons.
• Stéphane Nedelec , group leader, Institut du Fer à Moulin, Sorbonne Université (host: Hynek Wichterle, Pathology & Cell Biology)
• Ofer Yizhar , associate professor of neurobiology, Weizmann Institute of Science (host: Stavros Lomvardas, Biochemistry & Molecular Biophysics)

Since 2011, the annual Schaefer Scholar Awards have been given to research scientists who have distinguished themselves in the science of human physiology and whose current work is of outstanding merit with significant academic distinction.

Descriptions from the 2024 recipients:

Iok In Christine Chio

Project: “Methionine proteome reactivity in cancer induced cachexia”  

In her Schaefer project, Chio will use unique tools she has created to illuminate the basic mechanisms of cachexia, which could lead to new therapies that transform cancer care.

Cachexia is a severe wasting syndrome that accelerates the loss of muscle throughout the body. Patients weakened by this syndrome often cannot withstand the rigors of cancer therapy and thus experience greater mortality.

Chio’s lab has already uncovered a clue to the origins of cachexia. Loss of fat tissue often precedes muscle loss in cachexia, and some studies suggest that preserving fat can preserve muscle. Chio has found that fat loss in patients with cachexia seems to stem from an increase in an enzyme in fat tissue called MSRA.

Chio’s Schaefer project will test her hypothesis that MSRA promotes cachexia by modulating the methionine oxidation states of specific proteins in fat tissue. These proteins may serve as therapeutic targets or biomarkers of cachexia.

A unique and innovative chemoproteomic platform developed by Chio makes these new studies possible by allowing her to measure the oxidation states of individual methionine residues across the proteome.

Project: “Noncoding translation surveillance in tumor immunogenicity and immunotherapy ”

Wu’s Schaefer study could lead to the development of new cancer vaccines or therapies that improve the effectiveness of immunotherapies.

Immunotherapies are transforming cancer treatment but are ineffective for pancreatic cancer and many other tumors that are adept at hiding from the immune system.

The immune system can easily spot cancers covered with antigens that contain tumor-specific mutations. Tumors with fewer mutations, like pancreatic cancer, can more easily evade detection.

Recent studies have found that tumors can also be covered with “dark” antigens, which do not contain tumor-specific mutations but are generated by aberrant translation of noncoding sequences in cancer cells. Dark antigens, when present in large quantities, can trigger an attack from the immune system. Wu has found clues that cancers that evade the immune system might be suppressing the production of these dark antigens.

Wu will test the hypothesis that pancreatic cancer cells suppress the production of dark antigens by downregulating the BAG6 pathway, a process recently discovered in Wu’s lab, that partially degrades noncoding products and processes them into antigens. If so, he will then determine if increasing BAG6 expression will sensitize tumor cells to immunotherapies.

Stéphane Nedelec

Project: “Human motor neuron diversity and its implications for the etiology of amyotrophic lateral sclerosis”

With support from the Schaefer award, Nedelec will work with Hynek Wichterle’s lab to create improved human in vitro models of amyotrophic lateral sclerosis that should provide new insights into the disorder.

ALS is a late-onset disease that stems from the degeneration of muscle-innervating motor neurons, yet not all motor neurons exhibit the same vulnerability. Little is known about the mechanisms that underlie the differential resistance of adult neurons to ALS, in part due to limitations of existing models of the disease.

The researchers will test the capabilities of a groundbreaking organoid model recently developed in Nedelec’s lab. The 3D cellular system mimics several aspects of human trunk development, including the co-development of spine, spinal cord, muscle cells, and multiple subtypes of motor neurons corresponding to different locomotor circuits.

With the expertise of the Wichterle lab in analyzing motor neurons’ transcriptional programs and studying ALS physiopathology, they will determine if this new type of organoid is a better tool to model the maturation of neuromuscular circuits than current in vitro models of isolated motor neurons. If so, Nedelec will generate organoids from ALS patient-derived and control stem cells to better understand why motor neurons differ in their vulnerability to ALS. Ultimately, his project may open new therapeutic avenues for increasing motor neuron resistance to mutations causing the disease.

Ofer Yizhar

Project: “ A light-driven system for studying neuropeptide signaling using hybrid Opto-GPCRs ”

Oxytocin—a neuropeptide popularly known as the “love hormone”—promotes parenting behaviors and is essential in shaping social behavior throughout an animal’s life.  

Understanding the role of oxytocin in behavior requires molecular tools that can precisely manipulate oxytocin activity within animals, but current methods are either too slow or too broad.

Yizhar’s group recently developed a system that could be used to control circuits activated by the oxytocin receptor and other similar G-protein coupled receptors. The optogenetics system, called OptoGPCRs, deploys rhodopsins that occur naturally in various organisms into mammalian neurons, where they function like inhibitory GPCRs and can be controlled with light.

At Columbia, Yizhar will modify a naturally occurring OptoGPCR to match the profile of brain-resident neuropeptide receptors and validate the system in vitro. Yizhar will then collaborate with the Jones-Marlin, Kahn, and Abdus-Saboor labs at the Zuckerman Institute to assess the ability of this light-driven system to alter behavior, including the reactions of female mice to the sounds of their pups and social huddling among pups. Because the system is highly sensitive to light, OptoGPCRs can be activated non-invasively with overhead light and could become an important tool to study the physiological basis of neuropeptides in early-life social behaviors.

• Nebraska Medicine

Midwest Student Biomedical Research Forum

The Midwest Student Biomedical Research Forum provides an opportunity for young researchers from the health sciences, graduate, intern, and resident programs throughout the Midwest to present their research in the biomedical sciences.

 8:00 a.m. – 4:30 p.m.

Mike & Josie Harper Center Creighton University, 602 N 20th Street, Omaha, NE

MSBRF provides the option of presenting research in either an oral or poster format. The forum gives students an excellent opportunity to have their original work reviewed, evaluated, and recognized by their peers and established scientists. Each abstract submitted to the forum is subject to the same type of peer review applied to the selection of manuscripts for publication in major scientific journals.  Financial awards are given in several categories .

MSBRF has grown into an important assembly of young scientists. The previous MSBRF, held in March 2023, was a tremendous success with more than 180 students from Midwestern medical schools presenting their research at the forum.

The forum has been co-sponsored by the University of Nebraska Medical Center and Creighton University since 1987.

Keynote Presenter

Julie K. Freed, MD, PhD, FAHA, FCVS, is associate professor, executive vice chair, and director of clinical research in the Department of Anesthesiology at the Medical College of Wisconsin. Her presentation title is Translational Tales of Resiliency Within the Human Microvasculature.

Joining the Forum

Registration is now closed , cancel/refund policy.

The registration fee for an abstract that is not accepted will be refunded in the same form that the registration payment was received.

No other refunds will be processed.

If for any reason, you will not be able to present at the Forum, you must provide written notification, with the consent of your advisor, no later than Friday, Feb. 16, 2024.

Faculty Advisors

Director, Research Compliance Office; Professor, Department of Pathology, School of Medicine, Creighton University Medical Center

Assistant Professor Emeritus, Department of Pathology and Microbiology, University of Nebraska Medical Center

Planning Committee

The MSBRF Planning Committee is composed of students, faculty, and staff of UNMC and Creighton University.

MSBRF Student Committee

Amanda Christensen (UNMC) Emma Curran (Creighton) Anna Kosmach (UNMC) Justin Rudd (Creighton) Sarah Uhm (UNMC) Aron Lee (Creighton)

Joe Knezetic, PhD Charles Kuszynski, PhD

Administrative Contacts

Colin Dworak (CU) Brenda Ram, CMP, CHCP (UNMC)

Director, Educational Programs, Center for Continuing Education, UNMC

Phone: 402-559-9250

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• v.53(4); 2010 Aug

Research questions, hypotheses and objectives

Patricia farrugia.

* Michael G. DeGroote School of Medicine, the

Bradley A. Petrisor

† Division of Orthopaedic Surgery and the

Forough Farrokhyar

‡ Departments of Surgery and

§ Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, Ont

Mohit Bhandari

There is an increasing familiarity with the principles of evidence-based medicine in the surgical community. As surgeons become more aware of the hierarchy of evidence, grades of recommendations and the principles of critical appraisal, they develop an increasing familiarity with research design. Surgeons and clinicians are looking more and more to the literature and clinical trials to guide their practice; as such, it is becoming a responsibility of the clinical research community to attempt to answer questions that are not only well thought out but also clinically relevant. The development of the research question, including a supportive hypothesis and objectives, is a necessary key step in producing clinically relevant results to be used in evidence-based practice. A well-defined and specific research question is more likely to help guide us in making decisions about study design and population and subsequently what data will be collected and analyzed. 1

Objectives of this article

In this article, we discuss important considerations in the development of a research question and hypothesis and in defining objectives for research. By the end of this article, the reader will be able to appreciate the significance of constructing a good research question and developing hypotheses and research objectives for the successful design of a research study. The following article is divided into 3 sections: research question, research hypothesis and research objectives.

Research question

Interest in a particular topic usually begins the research process, but it is the familiarity with the subject that helps define an appropriate research question for a study. 1 Questions then arise out of a perceived knowledge deficit within a subject area or field of study. 2 Indeed, Haynes suggests that it is important to know “where the boundary between current knowledge and ignorance lies.” 1 The challenge in developing an appropriate research question is in determining which clinical uncertainties could or should be studied and also rationalizing the need for their investigation.

Increasing one’s knowledge about the subject of interest can be accomplished in many ways. Appropriate methods include systematically searching the literature, in-depth interviews and focus groups with patients (and proxies) and interviews with experts in the field. In addition, awareness of current trends and technological advances can assist with the development of research questions. 2 It is imperative to understand what has been studied about a topic to date in order to further the knowledge that has been previously gathered on a topic. Indeed, some granting institutions (e.g., Canadian Institute for Health Research) encourage applicants to conduct a systematic review of the available evidence if a recent review does not already exist and preferably a pilot or feasibility study before applying for a grant for a full trial.

In-depth knowledge about a subject may generate a number of questions. It then becomes necessary to ask whether these questions can be answered through one study or if more than one study needed. 1 Additional research questions can be developed, but several basic principles should be taken into consideration. 1 All questions, primary and secondary, should be developed at the beginning and planning stages of a study. Any additional questions should never compromise the primary question because it is the primary research question that forms the basis of the hypothesis and study objectives. It must be kept in mind that within the scope of one study, the presence of a number of research questions will affect and potentially increase the complexity of both the study design and subsequent statistical analyses, not to mention the actual feasibility of answering every question. 1 A sensible strategy is to establish a single primary research question around which to focus the study plan. 3 In a study, the primary research question should be clearly stated at the end of the introduction of the grant proposal, and it usually specifies the population to be studied, the intervention to be implemented and other circumstantial factors. 4

Hulley and colleagues 2 have suggested the use of the FINER criteria in the development of a good research question ( Box 1 ). The FINER criteria highlight useful points that may increase the chances of developing a successful research project. A good research question should specify the population of interest, be of interest to the scientific community and potentially to the public, have clinical relevance and further current knowledge in the field (and of course be compliant with the standards of ethical boards and national research standards).

FINER criteria for a good research question

Adapted with permission from Wolters Kluwer Health. 2

Whereas the FINER criteria outline the important aspects of the question in general, a useful format to use in the development of a specific research question is the PICO format — consider the population (P) of interest, the intervention (I) being studied, the comparison (C) group (or to what is the intervention being compared) and the outcome of interest (O). 3 , 5 , 6 Often timing (T) is added to PICO ( Box 2 ) — that is, “Over what time frame will the study take place?” 1 The PICOT approach helps generate a question that aids in constructing the framework of the study and subsequently in protocol development by alluding to the inclusion and exclusion criteria and identifying the groups of patients to be included. Knowing the specific population of interest, intervention (and comparator) and outcome of interest may also help the researcher identify an appropriate outcome measurement tool. 7 The more defined the population of interest, and thus the more stringent the inclusion and exclusion criteria, the greater the effect on the interpretation and subsequent applicability and generalizability of the research findings. 1 , 2 A restricted study population (and exclusion criteria) may limit bias and increase the internal validity of the study; however, this approach will limit external validity of the study and, thus, the generalizability of the findings to the practical clinical setting. Conversely, a broadly defined study population and inclusion criteria may be representative of practical clinical practice but may increase bias and reduce the internal validity of the study.

PICOT criteria 1

A poorly devised research question may affect the choice of study design, potentially lead to futile situations and, thus, hamper the chance of determining anything of clinical significance, which will then affect the potential for publication. Without devoting appropriate resources to developing the research question, the quality of the study and subsequent results may be compromised. During the initial stages of any research study, it is therefore imperative to formulate a research question that is both clinically relevant and answerable.

Research hypothesis

The primary research question should be driven by the hypothesis rather than the data. 1 , 2 That is, the research question and hypothesis should be developed before the start of the study. This sounds intuitive; however, if we take, for example, a database of information, it is potentially possible to perform multiple statistical comparisons of groups within the database to find a statistically significant association. This could then lead one to work backward from the data and develop the “question.” This is counterintuitive to the process because the question is asked specifically to then find the answer, thus collecting data along the way (i.e., in a prospective manner). Multiple statistical testing of associations from data previously collected could potentially lead to spuriously positive findings of association through chance alone. 2 Therefore, a good hypothesis must be based on a good research question at the start of a trial and, indeed, drive data collection for the study.

The research or clinical hypothesis is developed from the research question and then the main elements of the study — sampling strategy, intervention (if applicable), comparison and outcome variables — are summarized in a form that establishes the basis for testing, statistical and ultimately clinical significance. 3 For example, in a research study comparing computer-assisted acetabular component insertion versus freehand acetabular component placement in patients in need of total hip arthroplasty, the experimental group would be computer-assisted insertion and the control/conventional group would be free-hand placement. The investigative team would first state a research hypothesis. This could be expressed as a single outcome (e.g., computer-assisted acetabular component placement leads to improved functional outcome) or potentially as a complex/composite outcome; that is, more than one outcome (e.g., computer-assisted acetabular component placement leads to both improved radiographic cup placement and improved functional outcome).

However, when formally testing statistical significance, the hypothesis should be stated as a “null” hypothesis. 2 The purpose of hypothesis testing is to make an inference about the population of interest on the basis of a random sample taken from that population. The null hypothesis for the preceding research hypothesis then would be that there is no difference in mean functional outcome between the computer-assisted insertion and free-hand placement techniques. After forming the null hypothesis, the researchers would form an alternate hypothesis stating the nature of the difference, if it should appear. The alternate hypothesis would be that there is a difference in mean functional outcome between these techniques. At the end of the study, the null hypothesis is then tested statistically. If the findings of the study are not statistically significant (i.e., there is no difference in functional outcome between the groups in a statistical sense), we cannot reject the null hypothesis, whereas if the findings were significant, we can reject the null hypothesis and accept the alternate hypothesis (i.e., there is a difference in mean functional outcome between the study groups), errors in testing notwithstanding. In other words, hypothesis testing confirms or refutes the statement that the observed findings did not occur by chance alone but rather occurred because there was a true difference in outcomes between these surgical procedures. The concept of statistical hypothesis testing is complex, and the details are beyond the scope of this article.

Another important concept inherent in hypothesis testing is whether the hypotheses will be 1-sided or 2-sided. A 2-sided hypothesis states that there is a difference between the experimental group and the control group, but it does not specify in advance the expected direction of the difference. For example, we asked whether there is there an improvement in outcomes with computer-assisted surgery or whether the outcomes worse with computer-assisted surgery. We presented a 2-sided test in the above example because we did not specify the direction of the difference. A 1-sided hypothesis states a specific direction (e.g., there is an improvement in outcomes with computer-assisted surgery). A 2-sided hypothesis should be used unless there is a good justification for using a 1-sided hypothesis. As Bland and Atlman 8 stated, “One-sided hypothesis testing should never be used as a device to make a conventionally nonsignificant difference significant.”

The research hypothesis should be stated at the beginning of the study to guide the objectives for research. Whereas the investigators may state the hypothesis as being 1-sided (there is an improvement with treatment), the study and investigators must adhere to the concept of clinical equipoise. According to this principle, a clinical (or surgical) trial is ethical only if the expert community is uncertain about the relative therapeutic merits of the experimental and control groups being evaluated. 9 It means there must exist an honest and professional disagreement among expert clinicians about the preferred treatment. 9

Designing a research hypothesis is supported by a good research question and will influence the type of research design for the study. Acting on the principles of appropriate hypothesis development, the study can then confidently proceed to the development of the research objective.

Research objective

The primary objective should be coupled with the hypothesis of the study. Study objectives define the specific aims of the study and should be clearly stated in the introduction of the research protocol. 7 From our previous example and using the investigative hypothesis that there is a difference in functional outcomes between computer-assisted acetabular component placement and free-hand placement, the primary objective can be stated as follows: this study will compare the functional outcomes of computer-assisted acetabular component insertion versus free-hand placement in patients undergoing total hip arthroplasty. Note that the study objective is an active statement about how the study is going to answer the specific research question. Objectives can (and often do) state exactly which outcome measures are going to be used within their statements. They are important because they not only help guide the development of the protocol and design of study but also play a role in sample size calculations and determining the power of the study. 7 These concepts will be discussed in other articles in this series.

From the surgeon’s point of view, it is important for the study objectives to be focused on outcomes that are important to patients and clinically relevant. For example, the most methodologically sound randomized controlled trial comparing 2 techniques of distal radial fixation would have little or no clinical impact if the primary objective was to determine the effect of treatment A as compared to treatment B on intraoperative fluoroscopy time. However, if the objective was to determine the effect of treatment A as compared to treatment B on patient functional outcome at 1 year, this would have a much more significant impact on clinical decision-making. Second, more meaningful surgeon–patient discussions could ensue, incorporating patient values and preferences with the results from this study. 6 , 7 It is the precise objective and what the investigator is trying to measure that is of clinical relevance in the practical setting.

The following is an example from the literature about the relation between the research question, hypothesis and study objectives:

Study: Warden SJ, Metcalf BR, Kiss ZS, et al. Low-intensity pulsed ultrasound for chronic patellar tendinopathy: a randomized, double-blind, placebo-controlled trial. Rheumatology 2008;47:467–71.

Research question: How does low-intensity pulsed ultrasound (LIPUS) compare with a placebo device in managing the symptoms of skeletally mature patients with patellar tendinopathy?

Research hypothesis: Pain levels are reduced in patients who receive daily active-LIPUS (treatment) for 12 weeks compared with individuals who receive inactive-LIPUS (placebo).

Objective: To investigate the clinical efficacy of LIPUS in the management of patellar tendinopathy symptoms.

The development of the research question is the most important aspect of a research project. A research project can fail if the objectives and hypothesis are poorly focused and underdeveloped. Useful tips for surgical researchers are provided in Box 3 . Designing and developing an appropriate and relevant research question, hypothesis and objectives can be a difficult task. The critical appraisal of the research question used in a study is vital to the application of the findings to clinical practice. Focusing resources, time and dedication to these 3 very important tasks will help to guide a successful research project, influence interpretation of the results and affect future publication efforts.

Tips for developing research questions, hypotheses and objectives for research studies

• Perform a systematic literature review (if one has not been done) to increase knowledge and familiarity with the topic and to assist with research development.
• Learn about current trends and technological advances on the topic.
• Seek careful input from experts, mentors, colleagues and collaborators to refine your research question as this will aid in developing the research question and guide the research study.
• Use the FINER criteria in the development of the research question.
• Ensure that the research question follows PICOT format.
• Develop a research hypothesis from the research question.
• Develop clear and well-defined primary and secondary (if needed) objectives.
• Ensure that the research question and objectives are answerable, feasible and clinically relevant.

FINER = feasible, interesting, novel, ethical, relevant; PICOT = population (patients), intervention (for intervention studies only), comparison group, outcome of interest, time.

Competing interests: No funding was received in preparation of this paper. Dr. Bhandari was funded, in part, by a Canada Research Chair, McMaster University.

Clinical Research Coordinator II

• Madison, Wisconsin
• SCHOOL OF MEDICINE AND PUBLIC HEALTH/CLINICAL RESEARCH
• Staff-Full Time
• Opening at: May 23 2024 at 09:50 CDT
• Closing at: Jun 6 2024 at 23:55 CDT

Job Summary:

UW Clinical Trials Institute (CTI) is a partnership between the University of Wisconsin School of Medicine and Public Health (SMPH) and UW Health. It supports clinical research professionals, investigators, and participants to advance discovery and research in the prevention, detection, and treatment of disease to help improve the health of individuals, families, and communities across Wisconsin and beyond. Our exceptionally talented workforce provides care, support, and provide information to clinical trials participants; resources, training, and guidance to research professionals; and opportunities for industry partners to advance groundbreaking medical research. The CTI has an exciting opportunity for an experienced, full-time clinical research coordinator to join our growing team. The ideal candidate is intrinsically motivated, detailed oriented, an enthusiastic problem solver, and works well in a multi-disciplinary team to achieve a common goal. This role directly interacts with current and prospective participants of all ages and backgrounds, so the successful applicant is a clear, compassionate communicator with a strong customer service attitude. Additional responsibilities of the role include executing the procedures of the research protocol, safeguarding the integrity of study data, and actively participating in the screening, recruitment, and retention of our clinical trial participants. Senior members of the team are also expected to lend their experience to assist in training and mentoring new and less experienced members of the team. This is an on-site position with standard business hours expected during the workweek. However, flexibility is essential, as occasional early mornings, evenings, or weekends may be required to accommodate the schedules of the research participants. Additionally, the role may involve travel between research sites within the metro Madison area. If you are ready to make a meaningful impact in the field of clinical research, we encourage you to apply today! At CTI we consider diversity broadly, understanding each person's life and community experiences makes them unique. We believe diverse abilities/disabilities, racial/ethnic and cultural backgrounds, age, genders and gender identities and expressions, educational experiences and life stories drive true innovation and an exceptional work environment.

Responsibilities:

• 10% Schedules logistics, determines workflows, and secures resources for clinical research trials
• 15% Recruits, screens, selects, determines eligibility and enrolls trial participants, collects more complex information, performs medical assessments, and answers varied questions under supervision of a medically licensed professional. Performs medical assessments after completion of required training/certification, such as collecting vital signs, obtaining weight, and performing phlebotomy
• 15% Manages research workflow components, collects, prepares, processes, and submits participant data and samples to appropriate entities, adhering to research protocols
• 10% Identifies work unit resources needs and manages supply and equipment inventory levels
• 10% Serves as a main point of contact for clinical research participants, providing information regarding research procedures, expectations, and informational inquiries. Ensures participants follow the research protocol and alerts Principal Investigator of issues
• 5% Performs quality checks
• 10% Identifies resource needs and develops clinical research documents, such as job aids, tools, processes, procedures, and training. Contributes to the development of protocols
• 5% May assist with training of staff
• 10% Reconcile research billing, payments, and invoices and financial issues in concert with CTI financial specialists
• 10% Participate in quality and process improvement endeavors within the CTI; contribute to the development of new workflows and demonstrate flexibility in an evolving environment.

Institutional Statement on Diversity:

Diversity is a source of strength, creativity, and innovation for UW-Madison. We value the contributions of each person and respect the profound ways their identity, culture, background, experience, status, abilities, and opinion enrich the university community. We commit ourselves to the pursuit of excellence in teaching, research, outreach, and diversity as inextricably linked goals. The University of Wisconsin-Madison fulfills its public mission by creating a welcoming and inclusive community for people from every background - people who as students, faculty, and staff serve Wisconsin and the world. For more information on diversity and inclusion on campus, please visit: Diversity and Inclusion

Preferred Bachelor's Degree

Qualifications:

- At least 2 years of prior clinical research coordination experience.

- Health or science related college degree or equivalent education and experience. - Previous experience with basic clinical assessments including phlebotomy. - Previous experience with research applications used in the UW CTI: OnCore, eBinders (Florence), Advarra Payments, EPIC, etc. - Experience with training new staff.

Full Time: 100% It is anticipated this position requires work be performed in-person, onsite, at a designated campus work location.

Appointment Type, Duration:

Ongoing/Renewable

Minimum $50,000 ANNUAL (12 months) Depending on Qualifications

Additional Information:

Required: - Ability to work with minimal supervision with diverse teams of physicians, coordinators, collaborators, and study sponsors in a diplomatic, collaborative, and effective manner. - Ability to effectively multi-task, work well under time multiple time constraints, and meet competing deadlines. - Excellent interpersonal, oral, and written communication skills - Self-motivated, exceptional attention to detail, and able to exercise good judgement. - Understanding of medical terminology. - Strong organizational skills including the use of Microsoft applications (word, excel, etc.) - Able to quickly problem solve and resolve conflict. - Travel to multiple sites required, must have reliable transportation. - Valid Driver's License - Current ACRP or SoCRA Certification or ability to become certified within 6 months of hire. The selected applicant will be responsible for ensuring eligibility for employment in the United States on or before the effective date of the appointment. University sponsorship is not available for this position This position has been identified as a position of trust with access to vulnerable populations. The selected candidate will be required to pass an initial caregiver check to be eligible for employment under the Wisconsin Caregiver Law and every four years.

How to Apply:

To apply for this position, please click on the "Apply Now" button. You will be asked to upload a current resume/CV and a cover letter briefly describing your qualifications and experience. You will also be asked to provide contact information for three (3) references, including your current/most recent supervisor during the application process. References will not be contacted without prior notice.

Jennifer Wilkie [email protected] 608-262-8025 Relay Access (WTRS): 7-1-1. See RELAY_SERVICE for further information.

Official Title:

Clin Res Coord II(RE016)

Department(s):

A53-MEDICAL SCHOOL/CLINICAL RESEARCH

Employment Class:

Academic Staff-Renewable

Job Number:

The university of wisconsin-madison is an equal opportunity and affirmative action employer..

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Adhesive coatings can prevent scarring around medical implants

When medical devices such as pacemakers are implanted in the body, they usually provoke an immune response that leads to buildup of scar tissue around the implant. This scarring, known as fibrosis, can interfere with the devices’ function and may require them to be removed.

In an advance that could prevent that kind of device failure, MIT engineers have found a simple and general way to eliminate fibrosis by coating devices with a hydrogel adhesive. This adhesive binds the devices to tissue and prevents the immune system from attacking it.

“The dream of many research groups and companies is to implant something into the body that over the long term the body will not see, and the device can provide therapeutic or diagnostic functionality. Now we have such an ‘invisibility cloak,’ and this is very general: There’s no need for a drug, no need for a special polymer,” says Xuanhe Zhao, an MIT professor of mechanical engineering and of civil and environmental engineering.

The adhesive that the researchers used in this study is made from cross-linked polymers called hydrogels, and is similar to a surgical tape they previously developed to help seal internal wounds. Other types of hydrogel adhesives can also protect against fibrosis, the researchers found, and they believe this approach could be used for not only pacemakers but also sensors or devices that deliver drugs or therapeutic cells.

Zhao and Hyunwoo Yuk SM ’16, PhD ’21, a former MIT research scientist who is now the chief technology officer at SanaHeal, are the senior authors of the study, which appears today in Nature . MIT postdoc Jingjing Wu is the lead author of the paper.

Preventing fibrosis

In recent years, Zhao’s lab has developed adhesives for a variety of medical applications, including double-sided and single-sided tapes that could be used to heal surgical incisions or internal injuries. These adhesives work by rapidly absorbing water from wet tissues, using polyacrylic acid, an absorbent material used in diapers. Once the water is cleared, chemical groups called NHS esters embedded in the polyacrylic acid form strong bonds with proteins at the tissue surface. This process takes about five seconds.

Several years ago, Zhao and Yuk began exploring whether this kind of adhesive could also help keep medical implants in place and prevent fibrosis from occurring.

To test this idea, Wu coated polyurethane devices with their adhesive and implanted them on the abdominal wall, colon, stomach, lung, or heart of rats. Weeks later, they removed the device and found that there was no visible scar tissue. Additional tests with other animal models showed the same thing: Wherever the adhesive-coated devices were implanted, fibrosis did not occur, for up to three months.

“This work really has identified a very general strategy, not only for one animal model, one organ, or one application,” Wu says. “Across all of these animal models, we have consistent, reproducible results without any observable fibrotic capsule.”

Using bulk RNA sequencing and fluorescent imaging, the researchers analyzed the animals’ immune response and found that when devices with adhesive coatings were first implanted, immune cells such as neutrophils began to infiltrate the area. However, the attacks quickly quenched out before any scar tissue could form.

“For the adhered devices, there is an acute inflammatory response because it is a foreign material,” Yuk says. “However, very quickly that inflammatory response decayed, and then from that point you do not have this fibrosis formation.”

One application for this adhesive could be coatings for epicardial pacemakers — devices that are placed on the heart to help control the heart rate. The wires that contact the heart often become fibrotic, but the MIT team found that when they implanted adhesive-coated wires in rats, they remained functional for at least three months, with no scar tissue formation.

“The formation of fibrotic tissue at the interface between implanted medical devices and the target tissue is a longstanding problem that routinely causes failure of the device. The demonstration that robust adhesion between the device and the tissue obviates fibrotic tissue formation is an important observation that has many potential applications in the medical device space,” says David Mooney, a professor of bioengineering at Harvard University, who was not involved in the study.

Mechanical cues

The researchers also tested a hydrogel adhesive that includes chitosan, a naturally occurring polysaccharide, and found that this adhesive also eliminated fibrosis in animal studies. However, two commercially available tissue adhesives that they tested did not show this antifibrotic effect because the commercially available adhesives eventually detached from the tissue and allowed the immune system to attack.

In another experiment, the researchers coated implants in hydrogel adhesives but then soaked them in a solution that removed the polymers’ adhesive properties, while keeping their overall chemical structure the same. After being implanted in the body, where they were held in place by sutures, fibrotic scarring occurred. This suggests that there is something about the mechanical interaction between the adhesive and the tissue that prevents the immune system from attacking, the researchers say.

“Previous research in immunology has been focused on chemistry and biochemistry, but mechanics and physics may play equivalent roles, and we should pay attention to those mechanical and physical cues in immunological responses,” says Zhao, who now plans to further investigate how those mechanical cues affect the immune system.

Yuk, Zhao, and others have started a company called SanaHeal, which is now working on further developing tissue adhesives for medical applications.

“As a team, we are interested in reporting this to the community and sparking speculation and imagination as to where this can go,” Yuk says. “There are so many scenarios in which people want to interface with foreign or manmade material in the body, like implantable devices, drug depots, or cell depots.”

The research was funded by the National Institutes of Health and the National Science Foundation.

School of Engineering first quarter 2024 awards

Mit news in the news, eleven from mit awarded 2024 fulbright fellowships, professor emeritus jerome connor, pioneer in structural mechanics, dies at 91, mit conductive concrete consortium cements five-year research agreement with japanese industry, 3 questions: a shared vocabulary for how infectious diseases spread.

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WCRI Medical Price Index for Workers’ Compensation, 16th Edition (MPI-WC)

By rebecca (rui) yang, olesya fomenko.

May 16, 2024 Related Topics: Medical Costs , Medical Price Index

Since medical costs are a constant focus for policymakers and system stakeholders in state workers’ compensation systems, this annual study provides comparisons of prices paid for medical professional services across 36 states from 2008 to 2023. The study focuses on professional services (evaluation and management, physical medicine, surgery, major and minor radiology, neurological testing, pain management injections, and emergency care) billed by physicians, physical therapists, and chiropractors.

Unlike other indices measuring how prices for professional services change, this index is a more relevant benchmark of medical inflation in workers’ compensation as it focuses on those services commonly provided to workers with injuries. Since workers’ compensation price regulations are set at the state level, this study provides a state-level price index to help policymakers and stakeholders conduct meaningful comparisons of prices paid across states and to monitor price trends in relation to changes in fee schedules.

This edition covers 36 states that represent 88 percent of the workers’ compensation benefits paid in the United States. These states are Alabama, Arizona, Arkansas, California, Colorado, Connecticut, Delaware, Florida, Georgia, Illinois, Indiana, Iowa, Kansas, Kentucky, Louisiana, Maryland, Massachusetts, Michigan, Minnesota, Mississippi, Missouri, Nebraska, Nevada, New Hampshire, New Jersey, New Mexico, New York, North Carolina, Oklahoma, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Virginia, and Wisconsin.

WCRI Medical Price Index for Workers’ Compensation, 16th Edition (MPI-WC) . Rebecca Yang and Olesya Fomenko. May 2024. WC-24-20.

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• How do prices paid for medical professional services for treating workers with injuries in my state compare with other states?
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The increasing costs of medical care for treating injured workers have been a focus of public policymakers and system stakeholders in many states. To help them conduct meaningful comparisons of prices paid across states, and to monitor the price trends in relation to changes in fee schedules and network participation, this annual study creates an index for the actual prices paid for professional services across based on a marketbasket of commonly used services for treating injured workers.

The increasing costs of medical care for treating injured workers have been a focus of public policymakers and system stakeholders in many states. Download our 31-state study to compare medical prices paid across states and to monitor trends in relation to policy choices and changes in fee schedules.