Clinical trials are designed to evaluate how well new or existing medical, surgical, or lifestyle interventions work, how they compare with each other, and whether they’re safe. You might consider joining a clinical trial if, for example, you have an illness that hasn’t responded to existing treatments. In such cases, you’d have the opportunity to possibly receive the latest medications and therapies provided by top specialists at leading health care facilities.
Here are a few potential benefits you can gain from participating in a clinical trial:
- You could receive a treatment that’s generally not available to the public and possibly more effective than the standard treatment.
- You could gain access to therapies not available to the general public.
- You’ll often receive more frequent monitoring of your condition than you’d normally receive from your own doctors. (You’ll likely continue to see your regular doctor for other health matters.)
- You may feel a greater sense of control over your condition.
- You’ll contribute to research that may help others (as well as yourself).
Yet, despite such benefits, only a small percentage of eligible people enroll in clinical trials. A study published in the November 2019 Journal of the National Comprehensive Cancer Network found thatfewer than 5 percent of eligible patients are enrolling in clinical trials for cancer treatments, even though, in some cases, enrollment might increase life expectancy.
What is a clinical trial?
There are different types of clinical trials based on the objective of the experiment:
- Treatment trials test new interventions like drugs or a procedure that isn’t yet available or new combinations of existing interventions.
- Prevention trials target reducing the risk of developing a certain condition or of the condition returning.
- Screening trials evaluate new ways to detect diseases and health conditions.
- Diagnostic trials study new procedures or tests for diagnosing conditions.
- Quality-of-life trials study methods with the potential to help improve the comfort and quality of life of people with certain conditions.
- Behavioral trials study how to get people to change behaviors to improve their health.
Government agencies, medical institutions, pharmaceutical companies, foundations, and individual researchers are among those who sponsor clinical trials by initiating, managing, or financing them. They’re conducted in a variety of locations, including hospitals, universities, doctors’ offices, and community clinics.
Clinical trials are usually initiated with laboratory and animal studies. If results are promising, recruitment for participants begins.
What to expect
Applying for participation in a clinical trial doesn’t guarantee acceptance. You must fulfill all the study criteria, which may include factors such as age, degree of illness, and general health status. If you’re accepted, you’ll undergo what’s known as an informed consent process.
The research team will inform you of the trial’s possible benefits, its specific procedures and any potential discomfort, side effects, and risks. The team checks your health at the beginning of the trial, provides instructions, monitors your health regularly, and might continue to monitor you after the study is completed.
The amount of time the trial requires is highly variable. Protocols may include such things as frequent checkups, overnight hospital stays, and semiannual or annual evaluations that might continue for years. You’re free to withdraw from a clinical trial at any time during the study’s course.
Some trials pay participants; others reimburse for research-related expenses, such as travel to the evaluation site; still others offer no payment. Usually, the study sponsors cover treatment costs (including visits for evaluation or treatment and any tests performed solely for the study). The Affordable Care Act requires health insurers to cover participants’ routine costs.
How clinical trials are designed
The gold standard of clinical trial design is a randomized, double-blind study. Participants assigned to a treatment group receive the experimental treatment or procedure. Participants in a control group receive a placebo or the standard treatment or procedure they would be given if they weren’t participating in the study. Those assigned to receive a placebo take an inactive pill or undergo sham treatment that resembles the intervention being tested, but it has no effect on the condition.
In this type of trial, neither the patient nor the doctor knows who gets what until the end of the trial. This is to control for the placebo effect—when people who think they’re taking real medication, for example, get better even though they’ve only taken “sugar pills.” In a single-blind trial, only the doctors, and not the patients, know which participants are getting the standard or investigative treatment.
Placebos are never used when a patient could benefit from a known, effective treatment; thus, many studies, including most cancer trials, don’t have a placebo group. Researchers generally agree that placebos aren’t appropriate if there is a treatment available that prolongs life or preserves quality of life. In these situations, investigative treatments are compared with the standard therapy. However, a placebo group may be used if there’s no standard therapy with which to compare the experimental treatment or if the lack of treatment poses no danger to the participants.
People generally tend to assume that by enrolling they’ll receive the investigative treatment, but this might not be the case. They may be randomly assigned to the control group and usually won’t learn which group they were in until after the trial ends.
Informed Consent for Clinical Trials: What to Know
Before enrolling in a clinical trial, you’ll be given a document that details the purpose, risks, and benefits of the study, exactly what will be required, and for how long. Here are the specific questions it should answer.
Clinical trial phases
Specific procedures vary with the nature of the trial. Trials are generally conducted in four separate phases that can be modified depending on the specific treatment and condition being studied. The length of each phase varies among studies. The following is a typical path followed by many studies, particularly for investigational drugs or medical devices such as pacemakers:
- Phase I. Investigators test an experimental treatment or procedure in a small group (under 80 people) of often healthy volunteers to evaluate safety, identify side effects, and determine a safe dosage range, if applicable.
- Phase II. Researchers study the effectiveness and short-term side effects, if any, of the new intervention and further evaluate safety in 100 to 300 people being treated.
- Phase III. During this phase, researchers compare an experimental treatment, a new drug combination, or a new procedure with the best existing treatment in 1,000 to 3,000 people with the condition being studied. This phase is designed to confirm effectiveness, monitor side effects, and collect additional safety information.
- Phase IV. Trials in this phase are conducted after FDA approval and the product is being marketed to evaluate the long-term effects of a procedure or treatment in a real-world setting.
Just as there are benefits to enrolling in a clinical trial, there are risks and disadvantages as well:
- You won’t know whether the treatment will have side effects or complications, which can range from mild, such as dry mouth, to severe, such as a heart attack or, in very rare cases, premature death, especially in early-phase studies.
- You may be required to discontinue your standard treatment without any assurance that the new therapy will be helpful.
- Some clinical trials require a significant time commitment.
- If the study isn’t near your home, travel and hotel expenses may be prohibitive, although some trials reimburse for those costs.
Despite the potential drawbacks, enrolling in a clinical trial can yield personal benefits—as well as help countless others lead longer, healthier lives—that often outweigh the negatives.
Research in clinical trials must follow strict scientific and ethical guidelines. An institutional review board (IRB) made up of doctors, researchers and other experts, and community and patient advocates approves and regularly reviews most clinical trials. Board members aren’t involved in the trial and don’t receive remuneration.
In addition to an IRB, an expert scientific panel supervises many trials. These data and safety monitoring boards or committees can stop a trial if the study is causing unexpected harm to participants or it becomes clear that one treatment is superior to the other and should be made available to everyone in the study.
How to Find a Clinical Trial
If you are interested in joining a clinical trial, here are tips to get the ball rolling.
This article appeared in the May 2020 issue of UC Berkeley Health After 50.
Also see Connecting Patients and Clinical Trials.
Published May 20, 2020