Should you enroll in a clinical trial?

Volunteering for a study is a great public service, but don’t forget that you’re participating in an experiment. Know the risks, and ask plenty of questions.

(This article was first printed in the January 2004 issue of the Harvard Health Letter. For more information or to order, please go to http://www.health.harvard.edu/health.)

Clinical trials are the way that many of the most important questions in medicine get answered. Phase 1 trials, which include just a handful of subjects, are the first tests of a treatment in human beings. Then, in larger Phase 2 and 3 trials, the new treatment is carefully compared to a placebo or an alternative treatment. It’s the most dependable way to find out whether a drug, a device, or a therapy is effective.

The pros…

Many people sign up for altruistic reasons. By donating their time and effort (and bodies), they’re contributing to medical knowledge — no M.D. or Ph.D. required. According to a survey done in 2000, about 85% of volunteers participate in a study to help find a cure or to advance science. Evidence-based medicine has become a catchphrase. It means medical practice that’s based on statistically sound studies instead of individual preference or what’s been done in the past. Evidence-based medicine would grind to a halt without the thousands of unselfish people who volunteer every year for clinical trials.

There are also selfish reasons for participating. You might get a free physical exam and some additional tests. You’ll often get paid. It’s not a lot, but may be worth it if you have time on your hands. If you have a chronic health condition, you’ll probably learn a great deal more about it, which might open up new treatment possibilities or ways of coping with your condition unrelated to the trial (that is, apart from anything the trial is testing). Some people without health insurance sign up for a trial because it gives them access to medical care. Even if you have insurance, you may want to sign up if your plan limits coverage. And, of course, there’s the chance of getting a new treatment that works better than anything currently available.

…and the caveats

Private drug companies are paying for an increasing share of clinical trials — 70% of them, by some accounts. Whether that’s a good trend is controversial. Some argue that industry’s involvement speeds up testing and therefore practical application of research results and that systems in place manage to protect research subjects. Others see research increasingly tainted by profit motives and far too few safeguards. Regardless of who is right, you as the volunteer should have a clear idea of who is paying for the study.

Drug company research can be as intellectually sound and important to human health as any government study. But however altruistic your motives, remember that your contribution is also designed to help a bottom line in some way. Another thing: You can’t assume that a study taking place at a university or a nonprofit hospital is free of commercial entanglements. For example, the gene therapy trial at the University of Pennsylvania that resulted in the death of 18-year-old Jesse Gelsinger several years ago was government funded and vetted. But a key researcher and the university itself reportedly had a large financial stake in the biotech company that stood to benefit from the research.

Another reality of clinical trial research is that time is money. Each day a drug is in trials can cost a company millions because of high overhead costs. So there’s a great deal of competition for study subjects.

Drug companies pay doctors to work as “co-investigators” in a trial, often on a per-subject basis. The fees can range from $1,500 to $2,000 for each person enrolled. The fees are intended to cover lab, administrative, and other expenses.

Many people start a clinical trial with unrealistic expectations. For example, they believe (or will themselves into believing) that experimental therapy will work for them — although the premise of any trial is uncertainty about exactly that. They look past the risks, and even the best-intentioned investigators may downplay them, too. And, at least initially, many people have a misconception about the meaning of randomization, which is at the heart of the larger trials. Randomization means there’s a good chance you’ll be in the group that doesn’t get the treatment — the control group. Finally, people don’t realize that a study subject is not a patient in the usual sense. You can’t expect care tailored to your situation. A clinical trial is an experiment, and, like all scientifically valid experiments, it must follow rules (the study protocol). When you enroll in a study, you may be sacrificing some individualized care for the sake of science.