Clinical trials are the foundation of modern medical advancements, yet mistrust and lack of awareness lead to lower participation from volunteers.
The foundation of medical advancements
Clinical trials are the foundation of the medical advancements we enjoy today: cancer treatments, vaccines, and diagnostic tools, to name a few. Yet, people know very little about them until they face a serious diagnosis. This lack of knowledge, coupled with incorrect perceptions, has real consequences. Promising treatments move more slowly through development, patients miss opportunities for cutting-edge care, and crucially, results from trials without the right mix of participants have limited applicability.
Misperceptions
One of the most common misconceptions is that clinical trials are only for severely ill patients; that medical teams recommend trials to patients only when there’s little hope left. The fear of being used as a guinea pig is another very pervasive and frequently cited concern. This fear appears consistently across patient populations, regardless of prior research experience. People sometimes feel they’ll probably get a placebo, and being in the trial won’t help them.
The clinical center at the National Institutes of Health (NIH) reports that many fear the potential side effects. Statistics show that only 1% of the U.S. population participates in clinical trials. Paradoxically, almost 80% of the general public had a positive view of clinical research despite these fears and incorrect perceptions!
“Community mistrust and lack of awareness are major barriers to participation in clinical trials, particularly in underrepresented communities,” says Professor Lisa Goldman Rosas at Stanford’s School of Medicine, an expert in Epidemiology and Population Health. “History plays a big role, and we have to work hard to educate the community and gain people’s trust,” she adds.
What is a clinical trial?
Clinical trials are carefully designed research studies to test new medical interventions – drugs, devices, procedures, or preventive measures – to determine if they are safe and effective before becoming widely available. They play a pivotal role in advancing medical research and developing new treatments. Every prescribed drug in the U.S. has gone through this rigorous testing process, sometimes taking a decade or longer before the FDA approves the drug, and people can benefit from treatment.
In these trials, human subjects are assigned to one or more interventions (which could include a placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.
The study follows these subjects using a predefined, reviewed, and approved process, described in a written protocol finalized before the study begins and research subjects are enrolled, detailing how subjects will be assigned to the different groups or “arms” of the trial.
A fundamental, guiding principle in clinical-trial design is that the participant’s welfare must always take precedence over the interests of science and society; ethical considerations must always come first. This forms the foundational design framework, and all trial protocols are subject to ethical review and oversight by independent Institutional Review Boards. This is mandated by federal law for all clinical research involving human subjects in the US and many other countries.
Types of clinical trials
Approximately 25% of all clinical trials are observational. Researchers watch and record, but do not intervene. The landmark Framingham Heart Study is a great example. It began in 1948, following over 5,000 residents of Framingham, Massachusetts. This research study tracked cardiovascular disease development over decades and identified major risk factors such as smoking, high cholesterol, high blood pressure, obesity, and diabetes. The study is still ongoing with multiple generations now enrolled.
The remaining 75% test medical interventions; about 40% test drugs, medications, or vaccines; and 10% test medical devices such as implants, surgical instruments, or diagnostic tools. Interventions such as procedures, behavioral interventions, surgery, radiation, and diagnostic tests make up the balance.
Phases of a clinical trial
Drug development typically has several phases, using trials of different designs. The initial phase tests safety and dosage in small groups to explore optimal dosing and dose-related short-term toxicity, including whether patients can tolerate the new drug.
If a safe or tolerable dose range is found, the trial moves to Phase II, which evaluates effectiveness and side effects in larger groups, in which usually all subjects have the condition being addressed. The focus shifts from safety to efficacy in specific patient populations, while continuing to monitor safety.
This phase must demonstrate that the treatment delivers clinically meaningful results, and also shows enough promise to warrant the significant investment usually required to continue the trials.
Phase III trials compare the new treatment to placebo, current standard care, or other treatment modalities, in large populations. They are high-stakes, typically accounting for 50-70% of total clinical development costs. Only about 1 in 3 drugs move forward to Phase III; what looks promising in early testing may not translate to clinical benefit.
Once Phase III is complete and regulatory approval for use is obtained, Phase IV is typically used to monitor real-world use of the new drug (or device or intervention), using observational data from medical records, registries, or adverse-event databases.
The vital role of volunteers
Clinical trials investigating an intervention for a specific condition need to enroll patients with that condition. Prevention trials enroll people who may be at risk for that condition. By assembling a group of volunteers that is representative of the broader community, researchers can better ensure the results apply to that community.
Education, awareness, and building of trust are needed, especially among the underrepresented members of our community. Individuals who willingly participate in clinical research to drive medical advancement are critical in drug development and other medical advancements.
Can I volunteer for a trial if I’m healthy?
Healthy volunteers can play a crucial role. For most new medications (excluding drugs for cancer and some toxic biologics), healthy volunteers can participate in Phase I trials to establish safety profiles, determine appropriate dosing ranges, and study how drugs are absorbed, distributed, metabolized, and excreted in the human body – without the confounding effects of disease.
They provide essential baseline data that defines what “normal” looks like, enabling researchers to later distinguish drug effects from disease symptoms in patient populations. Beyond drug testing, healthy volunteers contribute to observational studies, diagnostic test development, prevention trials, and research on normal physiology.
Nearly 60% of healthy volunteers cite societal benefits as their primary motivation, recognizing that their participation accelerates medical advances that will benefit future patients, including their own loved ones and communities. By volunteering before they themselves face health challenges, these individuals help establish the safety standards that protect all future clinical trial participants and ensure that promising treatments can reach patients who need them faster and more safely.
Clinical trials matter
Clinical research should be seen as a social good, vital to the mission of enhancing health, lengthening life, and reducing the burdens of illness and disability, providing insights and answers about the safety and effectiveness of drugs and other therapies. Improved knowledge and awareness of clinical trials will help all of us understand the true benefits and risks, make informed decisions about the value of participation, and advocate for them in our communities and networks.
As Dr. Francis Collins, the distinguished physician-geneticist renowned for leading the international Human Genome Project to its completion, and for his record-breaking tenure as the Director of the NIH from 2009 to 2021, said: “signing up for a clinical trial may benefit medical research and help future generations. But it is not strictly an altruistic endeavor. In many instances, trial participants do gain personal advantages, such as improved disease outcomes or better health.”
