A clinical trial is a research study that involves human volunteers. Heralded as the best, if not the only way, to advance medicine, clinical trials are designed to evaluate new ways to prevent, detect, or treat disease. (1,2)
Every prescription medicine on the market and every vaccine in use was once the subject of a clinical trial. Countless lives have been saved by penicillin and the smallpox vaccine, to reach back in history and name just two. Current advances include a long list of immunotherapies now approved for use by the U.S. Food and Drug Administration (FDA) to treat lung cancer, as well as other cancers.
Only prescription medications require clinical trials before approval by FDA. Over-the-counter medications and supplements typically do not require a clinical trial, according to the Congressional Research Service.
The number of clinical trials being conducted in the United States and around the world is higher than it’s ever been. A total of 439,377 studies are currently registered with ClinicalTrials.gov, one of the largest databases of clinical trials in existence. Of these, 63,922 are actively recruiting participants. That’s up from just 1,255 trials, in total, when the National Institutes of Health (NIH) website first became available to the public in early 2000. (3,4)
Yet despite efforts by both pharmaceutical companies and public health agencies to educate people about the potential benefits of clinical trials and to recruit volunteers, public awareness about clinical trials remains relatively low — as does participation, particularly among women, children, and minorities, who tend to be underrepresented in research. (5,6,7)
One study found that while women were well-represented in trials of drugs for hypertension and atrial fibrillation, the numbers of those participating in studies involving heart failure, coronary artery disease, and acute coronary syndrome were significantly lower than the relative numbers of women affected by those conditions in the general population. (8) Cardiovascular disease continues to be the leading cause of death among women (and men) in the United States.
Indeed, recruitment in general is considered a major challenge for those leading these trials, and many studies never get off the ground due to a lack of volunteers. Studies involving cancer therapies are known for being hard hit. Over 18,000 cancer clinical trials are in the recruitment stage, but estimates put the rate of participation among cancer patients at as low as 6 percent of potential trial candidates.
Among commonly cited reasons why some cancer patients may not enroll in clinical trials: simple lack of awareness among potential volunteers, mistrust of the process and fear of being a “guinea pig,” fear of not receiving the new therapy but rather the “standard of care” comparison therapy, restrictive inclusion criteria — for example, those who have had prior chemotherapy may be ineligible — and fear of side effects of new drugs. (9,10)
Should You Participate in a Clinical Trial?
People say they participate in clinical trials for a variety of reasons, including to play an active role in their healthcare; to help others by contributing to medical research; to gain access to treatments before they are widely available; to get medical attention and care they might not ordinarily receive; and even to earn money. (14,15)
Satisfaction rates among people who have participated in a clinical trial run high: One report found that 95 percent of those who had taken part in a trial in the past said they would consider joining another in the future. (16)
Still, many potential first-time volunteers never move forward. There are a number of reasons for that. Some, particularly minorities, are disturbed by memory of missteps in the past, primarily the Tuskegee Syphilis Experiment (1932–1972), in which African-American men were told by the U.S. Public Health Service that they were being treated (at no charge) for syphilis, but who in fact received no treatment at all for the communicable and often deadly disease. (17)
Still others are cognizant of past trials that resulted in products or treatments that subsequently turned out to be problematic for some reason, such as thalidomide, the sedative marketed to pregnant women in the late 1950s for morning sickness that caused thousands of severe birth defects; and the Dalkon Shield, the intrauterine device (IUD) used in the early 1970s that prompted more than 200,000 lawsuits by women harmed by the device.
Other commonly cited concerns include: fears about side effects; doubts that the therapy will be any better than the standard of care; and worry about receiving a placebo — as in, why would anyone want to risk getting a placebo, especially if they are very ill? (14)
While experts in the field acknowledge that the risks of enrolling in a clinical trial are real and entail many unknowns, they also say that some concerns are unfounded. For example, placebos are rarely used in trials involving patients with more advanced or serious illness, says Kenneth Getz, founder of CISCRP and deputy director of the Tufts Center for the Study of Drug Development, explaining that most use an active comparison drug, typically the FDA-approved standard of care. In addition, volunteers are free to withdraw from a trial at any time and for any reason. (18)
Finding a Trial That Suits Your Needs
Clinical studies are conducted in a variety of types of locations, including hospitals, universities, doctors’ offices, and community clinics. The location depends on who’s conducting the study. Funders can include governmental agencies, industry groups, individuals, universities, and other organizations, such as foundations and not-for-profits. Locations now exist in all 50 states and in more than 200 countries around the world. (4)
Trials can be found for virtually every condition imaginable. While many of the most commonly publicized trials often involve high-stakes therapies, such as those for late-stage cancer, lower-stakes trials are plentiful. Registered trials include treatments for everything from acne to insomnia to anxiety. (4)
And they’re not all about pharmaceuticals. Many clinical trials involve interventions with therapies such as botanicals, mineral supplements, and acupuncture — even aromatherapy and yoga for hot flashes, for example. (4)
People with a preference for lifestyle interventions to prevent or treat illness can help play a role in moving those disciplines forward as well. A number of trials involving diet and exercise have been registered.
These include studies that have now been completed assessing the effects of low-fat vegan versus Mediterranean diets on body weight and insulin sensitivity, run by the Physicians Committee for Responsible Medicine, and the effects of intermittent fasting in subjects consuming a Mediterranean or Western diet, performed by the Washington University School of Medicine on the prevention and treatment of age-related diseases. (4)
In the end, whether to participate in a trial is a personal decision, but perhaps the most important takeaway is that the decision be an informed one. Whether the thinking is “buyer beware” or “well-informed is well-armed,” the consensus is that it’s best to gather as much information and input as possible from trusted and valued sources, including doctors and other health professionals as well as family and friends.
“Our motto is ‘education before participation,’” says Getz. “We encourage patients and their families and friends to gather the facts, speak with other patients and professionals, and ask a lot of questions.”
There are several searchable databases that offer listings of available clinical trials, including:
Additionally, you may find searchable listings at local healthcare centers, on the websites of disease specific organizations, and even advertised in local newspapers. (14,19,20,21,22)
Things to Keep in Mind When Considering a Clinical Trial
In most cases, participating will be at no cost to you. Sometimes, insurance may be billed for some devices, drugs, or services. These should be spelled out in the informed consent process.
What to Ask Can you spell out what the cost will be to me?
Some clinical trials will not require travel. Others will require regular check-ins. Find out what will be required of you beforehand.
What to Ask Will my travel costs be covered?
A treatment trial may be comparing a new treatment to one that is the standard, or to a placebo. You have no control over which arm of the trial you are assigned to.
What to Ask Am I willing to participate if I’m not getting the new treatment?
You can opt out of a trial at any time.
What to Ask How do I handle the situation if I decide to opt out part way through the trial?
Some trials involve years of follow-up.
What to Ask When does the trial end, and how long will my obligation last?
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