Should You Join a Research Study? 9 Tips for Volunteers in Clinical Trials

Mary Collins has had type 1 diabetes for half her lifetime. The autoimmune disease, while manageable, is costly and unpleasant for the 55-year-old self-employed seamstress from Mountain Rest, S.C. So Collins, who lacks health insurance and hates the insulin shots she has to give herself before every meal, jumped at a chance a few years ago to join a research study. It provided her with free medical supplies, reimbursed her for travel to her appointments at the study site, paid her $1,200 outright, and—most appealingly—let her try out an inhaled form of the medication she'd always had to take by needle.

Collins was to participate in the industry-sponsored trial, which was designed to test a company's unproven delivery device for inhaled insulin, for a year. After 6 months, however, she dropped out of the study, concerned that her blood glucose levels were fluctuating to an unhealthy degree. Despite that outcome—and a hassle she experienced while seeking compensation that was due her—she has no regrets about giving the clinical experiment a try. "Even though I did have a problem," she says, "it would not stop me from doing it again." Medical research is an enormous—and enormously important—enterprise. Without a steady stream of volunteers like Collins, it would grind to a halt, making new therapies impossible to develop. "The reason we have [today's] standard-of-care drugs . . . is because people went into clinical trials and proved their efficacy," says Michael Caligiuri, a medical oncologist and CEO of the James Cancer Hospital at Ohio State University.

That doesn't mean everyone should join a clinical trial, however. For one thing, no trial is completely risk free. Ten years ago this month, in one of the most infamous research disasters in modern times, Jesse Gelsinger had a fatal reaction to an experimental gene therapy. The 18-year-old's death led to allegations of ethical misconduct directed at the study's leaders and the University of Pennsylvania, which ran the trial. (The parties involved reached an out-of-court settlement.)

Even ethically unassailable studies have risks. "Bad things can happen even when [trials] are done well," says Christine Grady, a bioethicist at the National Institutes of Health's research hospital, known as the NIH Clinical Center. In a 2005 analysis of 460 early stage cancer trials, researchers led by Grady found that 1 in every 7 participating cancer patients suffered serious side effects from drug toxicity. One in every 200 volunteers died from it. (That kind of trial may be particularly risky because of the nasty nature of many cancer medications.) And while many volunteers hope participating in a research study will improve their health, there's no evidence that that's likely. In the same 2005 study, doctors observed clinical improvements in only 4.4 percent of patients receiving the most unproven therapies; more patients were harmed than helped. And although a trial might give a patient access to a new drug, that drug isn't necessarily better than (or even as good as) standard therapies that have already been tested and proved.

Whether a particular medical study is right for a particular patient depends on the person's health condition, personal priorities, what alternative treatments are available, and how thoroughly the experimental therapy has been studied so far, among other factors. Here are nine tips for people considering volunteering for a clinical trial:

Understand the researchers' goals. A doctor's sole purpose is to help you get better, right? Not when she's wearing the hat of a medical researcher. A researcher's foremost responsibility, unlike that of a typical physician, is to ensure the scientific and ethical integrity of a medical experiment. Yet many patients wrongly hold a "presumption that being in a trial is [automatically] good for you," says Deborah Zarin, head of clinicaltrials.gov, a government database of clinical trials. That misunderstanding, called the therapeutic misconception, may lead some patients to agree to tests and procedures that they'd refuse if they really thought about the implications for their health.