How patients contribute to medical progress through research

What does taking part in research mean?

Taking part in research means volunteering as a subject of a study, while the related but distinct idea of "involvement" means using your experience of a condition to help shape how research is designed, run or communicated. Cancer Research UK distinguishes three related activities: participation, where patients or healthy volunteers are the subject of a study; engagement, where research information and knowledge are shared with the public; and involvement, where patients or the public help define research questions, design studies or develop patient-facing documents^[1].

Both healthy people and people with a diagnosed medical condition can take part in clinical research, and participation is always voluntary; participants can withdraw at any time and for any reason. Every medicine, device, diagnostic test and technique used in medicine today was tested in volunteers before it became part of routine care^[2].

What types of clinical research exist?

Clinical research is divided into two broad categories: observational studies and clinical trials, also called interventional studies. Observational studies aim to identify and analyse patterns in medical data or biological samples, such as tissue or blood, without testing an intervention. Clinical trials test the safety and effectiveness of medical interventions, such as medications, procedures and tools, in living people^[2].

Within these two categories, several specific study types exist^[2]:

• Biospecimen studies — collection and storage of biological material such as blood, tissue or cells in a biorepository, together with a participant's medical information and written consent;
• Healthy volunteer studies — people with no known major health issues who help researchers see how a study drug is absorbed and removed from the body, usually early in development;
• Surveys — used to study health behaviours, practices and opinions;
• Clinical trials — test drugs, surgical procedures, medical devices, new ways of using existing treatments, or new ways of changing health behaviours.

Clinical trials themselves have different goals depending on the medical question asked: treatment trials test the safety and effectiveness of new treatments, including medications, surgical procedures and new combinations of existing treatments; prevention trials study interventions such as vaccines, medications and lifestyle practices^[2].

What must a clinical trial satisfy before you can take part?

A clinical trial of a medicinal product is defined as any study involving human subjects for the purpose of discovering or confirming clinical or pharmacological effects, identifying adverse reactions, or tracking the absorption, distribution, metabolism and excretion of one or more investigational medicinal products, with a view to their safety and efficacy. Before a trial can start, it must be justified by preclinical or previous clinical data, scientifically justified and described in a clinical trial protocol. It must also be based on ethical principles, conducted by appropriately qualified staff, and carried out at a designated research centre^[3].

Clinical trials are also classified by their sponsor and purpose. Commercial trials are conducted to obtain or amend a marketing authorisation for a medicinal product or medical device, and pharmaceutical or biotechnology companies act as their sponsors. Non-commercial trials are conducted to advance clinical practice and search for new treatments^[3].

Conducting clinical trials enhances physicians' medical knowledge and helps ensure more effective clinical practice, and their results are also taken into account when decisions are made about reimbursing medicines from public funds^[3].

How does taking part differ from helping to shape research design?

Cancer Research UK states that when patients participate as subjects, "the research is being done to them, not with them," whereas involvement means working with patients at any stage of a project to define research questions, plan and design a study, develop patient-facing documents, or share progress. Researchers are expected to involve people affected by a condition and members of the public whenever their work is public-facing, whenever patients or healthy individuals will be study subjects, whenever samples are being taken from patients, or whenever there are ethical issues or implications associated with the research^[1].

A multi-stakeholder survey conducted in Switzerland between January and July 2019 by the European Patients' Academy on Therapeutic Innovation Switzerland (EUPATI CH) received 55 online responses and 14 semi-structured interviews from patient representatives, academic researchers, pharmaceutical industry staff, healthcare professionals and government agency representatives. Thirty-eight percent of respondents considered patient involvement in Switzerland to be limited or absent. The main barriers identified were a lack of funds and human resources, cited by 65% of respondents, followed by a lack of information and education on how to become a patient advocate, cited by 21%^[4].

How has patient input shaped medicines regulation?

The European Medicines Agency (EMA) has stated that since patients are the ones who will take a medicine, it makes sense to incorporate their values and knowledge at different stages of the process of developing, assessing, licensing and monitoring medicines. On 26 September 2013, the EMA held a workshop at its London headquarters that brought together patients, consumers, healthcare professionals and pharmaceutical industry representatives with members of the Agency's scientific committees. The aim was to explore what patients can contribute to the assessment of benefit and risk that lies at the heart of the regulatory process^[5].

The EMA's report identifies the response to HIV as a seminal example of patient-driven change: intense, well-informed patient activism from the 1980s onwards forced both the pharmaceutical industry and regulators to reconsider how they interacted with patients. The European Community Advisory Board (ECAB), a working group of the European AIDS Treatment Group, was set up in 1997 as a forum for interactions between patients, the pharmaceutical industry and regulators, aiming for more constructive dialogue and more timely availability of safe and effective therapies^[5].

What medical advances have come from patient participation?

Clinical trials led at Johns Hopkins Medicine have produced several documented medical milestones over the past three decades^[6].

Year	Type	Advance
1995	Research	Clinical trials helped find hydroxyurea, sickle cell anaemia's first effective treatment
2015	Research	A large study showed a drug aimed at the immune system may help people with lung cancer survive longer
2016	Milestone	A clinical trial showed the benefits of a kidney transplant from a living, incompatible donor
2018	Research	Researchers developed a blood test to screen for eight types of cancer
2021	Document	A large study found that students who received eyeglasses through a school-based programme scored higher on reading and maths tests

^[6]

Results of clinical studies are submitted to and published in biomedical research journals, and a central database of published research, PubMed, is run by the National Library of Medicine, part of the National Institutes of Health^[6].

Why does the diversity of participants matter?

Research results are more likely to apply to a diverse population when the group of study participants itself reflects that diversity. A 2020 report from the U.S. Food and Drug Administration found that 75% of research participants were white, while white people made up 60% of the U.S. population; 8% of participants were African-American/Black, against 13% of the population; 11% were Latino/Hispanic, against 18% of the population; and 6% were Asian, matching the 6% share of Asian Americans in the population^[7]. This is a United States-specific figure and is cited here as an illustration of a broader pattern, not as a description of participation rates in Europe.

Race, disability, socioeconomic status and other demographic factors can affect a person's risk of developing certain conditions, and can also affect how they respond to medical interventions and their overall health outcomes. Research participants should represent the people most affected by the disease or condition under study; for example, certain cancers and sickle cell disease occur more often in people of colour, which makes their participation in related clinical studies particularly important^[7].

Johns Hopkins Medicine has organised a Community Research Advisory Council, made up of community leaders who provide feedback to research teams on how to design and conduct studies in ways that provide value to the entire community. During the 2020 COVID-19 vaccine trials, researchers recruited participants in communities outside the medical centres where they usually look for volunteers, including working with church leaders in Baltimore to reach the local Latinx community^[7].

What do participants themselves say about taking part?

In the United Kingdom, an adult Participant Research Experience Survey (PRES) collected 29,664 responses during the 2024/25 period. Of these respondents, 69% took part in research for the first time, and 91% said they would take part in research again. In the children and young people's survey, 92% strongly agreed or agreed that they felt valued by staff for taking part in a research study, and 85% agreed or strongly agreed that they knew how to contact someone from the research team if they had questions or concerns^[8].

People who take part in research report several distinct motivations: making a difference by helping improve treatments and quality of life, supporting research into a condition they care about, gaining access to new treatments, and taking an active role in their own health and care^[8].

How can you start taking part in research?

Potential volunteers are carefully screened to confirm they meet the requirements of a specific study before they begin, and most exclusions relate to safety considerations. Clinical research studies need people of every age, health status, race, gender, ethnicity and cultural background, because a broader range of participants increases the likelihood that resulting treatments and procedures will be safe and effective for everyone^[2].

Both patients with a diagnosed condition and healthy volunteers with no known major health issues are needed for research. For new medications, it is generally considered safer and easier to begin testing in healthy individuals, in studies that look at how a drug affects the body and how it is absorbed and removed^[2].

Reliable and complete information about clinical trials is important throughout the treatment process, and patients should ideally receive this information from the beginning of diagnosis so they can make informed decisions about their treatment path. Clinical trials are sometimes treated as a treatment of last resort, which can make it harder for patients to weigh their risks and benefits realistically^[3].

When should you talk to your doctor or care team about taking part in research?

Participation in clinical research is always voluntary, and a participant can leave a study at any time and for any reason, without needing to justify the decision^[2]. Ask your doctor or care team for information about a specific trial's requirements, and about how a study relates to your diagnosis, before agreeing to take part^[3].

Cancer Research UK advises researchers that if they are unsure whether to involve people affected by a condition, or members of the public, in shaping a piece of research, the simplest approach is to ask them^[1]. This underlines that meaningful involvement depends on researchers actively seeking out patients' and the public's views, rather than assuming what they want.

Summary

Taking part in research ranges from being the subject of a study to helping design the questions a study asks in the first place. Both roles are documented as contributing directly to medical progress, from the first effective treatment for sickle cell anaemia to blood tests that screen for multiple cancers.

Diversity among participants, clear information at the point of diagnosis, and open communication with your care team are recurring themes across patient organisations, hospitals and regulators. Before agreeing to take part in any study, ask about its purpose, its requirements, and your right to withdraw at any time.

This article is for general information only and does not replace individual medical advice. Always discuss a specific clinical trial or research study with your doctor or care team before making a decision.

1. [1] Patient and public involvement in research - Cancer Research UK (accessed 2 June 2026) — https://www.cancerresearchuk.org/for-researchers/how-we-deliver-research/patient-and-public-involvement-in-research
2. [2] Clinical Research: What Is It? - Johns Hopkins Medicine (accessed 2 June 2026) — https://www.hopkinsmedicine.org/research/understanding-clinical-trials/clinical-research-what-is-it
3. [3] Patient in Clinical Trials - Information Folder, Updated Version 3.0 - Agencja Badań Medycznych (ABM) (accessed 2 June 2026) — https://abm.gov.pl/download/1/8525/Informationfolder-updatedversion30.pdf
4. [4] https://ch.eupati.eu/wp-content/uploads/sites/48/2021/12/fmed-08-795659.pdf
5. [5] The patient's voice in the evaluation of medicines: How patients can contribute to assessment of benefit and risk - European Medicines Agency (accessed 2 June 2026) — https://www.ema.europa.eu/en/documents/report/report-workshop-patients-voice-evaluation-medicines_en.pdf
6. [6] Clinical Research: Medical Advancements from Johns Hopkins Medicine - Johns Hopkins Medicine (accessed 2 June 2026) — https://www.hopkinsmedicine.org/research/understanding-clinical-trials/clinical-research-medical-advancements
7. [7] Why It's Vital That All People — Including People of Color — Take Part in Clinical Research Studies - Johns Hopkins Medicine (accessed 2 June 2026) — https://www.hopkinsmedicine.org/research/understanding-clinical-trials/poc-and-clinical-trials
8. [8] Why taking part matters - Be Part of Research (accessed 2 June 2026) — https://bepartofresearch.nihr.ac.uk/take-part-in-research/why-taking-part-matters/