Clinical trials seek to assess whether a medical strategy, treatment, or device is safe and effective for humans. These studies may also aim to assess how effective a medical approach is in the context of specific conditions or groups of people. Overall, clinical trials add to medical knowledge and provide reliable data to support health care decision-making and guidelines 1. Because of how important patient safety and well-being are, each new potential therapeutic must go through a thorough clinical trials process before receiving approval.
The idea for a clinical trial often originates in the research laboratory. After scientists assess new treatments or procedures in the lab and in animals, promising treatments are probed in the context of clinical trials in humans. As new treatments move through the different phases of a clinical trial, more and more information is obtained about the treatment and its risks and effectiveness. The research team often includes doctors, nurses, social workers, scientists, and clinical trial coordinators, with a principal investigator leading the work overall.
Clinical trials are composed of four phases focused on treatment, prevention, screening, diagnoses, supportive care, health services research, or basic science, and this process is rigorously adhered to 2. Each phase has a different goal and helps researchers address different questions. In phase I trials, researchers test a drug or treatment in a small group of people (20–80) for the first time ever. The aim is to shed light on its safety and identify any side effects. In phase II trials, the new drug or treatment is given to a larger group of people (100–300) to assess its effectiveness and further confirm its safety. In phase III trials, the new drug or treatment is administered to large groups of people (1,000–3,000) to validate its effectiveness and monitor any side effects. The drug or treatment is also compared to similar treatments. In phase IV trials, after a drug is approved by the Food and Drug Administration (FDA) and made available to the public, scientists track its safety in the general population, assessing more and more specific information about its benefits and optimal use. Clinical trials adhere to a protocol which is deliberately crafted to balance potential benefits and risks to participants and answer specific research questions.
There are a number of elements in how a study is designed that are considered to be the “gold standard” in research. For example, in clinical trials testing whether a therapeutic is effective, participants may receive a placebo, or an inactive product that resembles the test product under study, to compare to it to. Furthermore, the randomization of clinical trial participants minimizes any bias linked to assigning participants to one treatment or another. The randomization process can be modified for clinical trials that need to balance potential differences in certain demographics, such as by randomizing as much as possible while making sure the different groups have the same distribution of ages.
Blinded clinical trials, in which some or all of the people involved with the treatment and measuring and analyzing the data are unaware of which participants belong to which groups, are carried out to prevent members of the research team and/or study participants from unwittingly influencing the trials’ outcomes.
Most clinical trials in the U.S. are approved and monitored by an Institutional Review Board to ensure a reasonable balance of risks and benefits. These Institutional Review Board committees are key to protecting the rights and safety of research participants.
Clinical trial sponsors may be people, institutions, companies, government agencies, or other organizations that are responsible for initiating, managing or financing the clinical trial, but do not conduct the research.
Participants must read and sign an “informed consent” document before joining a trial outlining potential risks and benefits. Throughout the trial , risks are controlled and monitored; however, the nature of medical research studies makes a certain degree of risk inevitable 3. Especially in earlier stages of the clinical trials process, when less data is available, participants should be educated about what to expect and what could happen.
Regarding participant selection, it is essential that clinical trials include participants with a variety of lived experiences and living conditions, as well as race and ethnicity, age, and sex, among others. This is because many factors can influence how different people experience the same disease or treatment.
References
1. Learn About Clinical Studies – ClinicalTrials.gov. Available at: https://clinicaltrials.gov/ct2/about-studies/learn. (Accessed: 24th February 2023)
2. Step 3: Clinical Research | FDA. Available at: https://www.fda.gov/patients/drug-development-process/step-3-clinical-research. (Accessed: 24th February 2023)
3. The Basics | National Institutes of Health (NIH). Available at: https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics. (Accessed: 24th February 2023)
