Clinical Trials: Testing Medical Products in People

by Food and Drug Administration (FDA)

Carolyn Meritt was preparing to undergo a stem cell transplant in 2001 to treat mantle cell lymphoma — a rare cancer of the lymph nodes — when her doctor told her about a clinical trial being conducted by the National Institutes of Health (NIH). He suggested that she consider becoming a participant.

"I didn't even know what a clinical trial was," Meritt says. That was only one of many questions she would need answered before she could decide whether or not to enroll.

But Meritt's white blood counts were rising. She would need treatment soon. She could proceed with the stem cell transplant — the only existing therapy — or, as long as she felt well enough, she could hold out hope and participate in the clinical trial, an option open to her only if she had no prior treatment. But how would she decide?

Predicaments like Meritt's are not uncommon. "We get this kind of call just about every day," says Theresa Toigo, director of the Food and Drug Administration's Office of Special Health Issues (OSHI). "The patients have serious and life-threatening diseases. They're afraid. They want some options, but they're not sure where to go. We try to provide the information patients and their families need to make important decisions in such difficult situations."

What Is A Clinical Trial?

A common thread linking such calls, says Toigo, is clinical trials. Clinical trials, also known as clinical studies, test potential treatments in human volunteers to see if they should be approved for wider use in the general population. A treatment could be a drug, medical device, or biologic, such as a vaccine, blood product, or gene therapy. Potential treatments, however, must be studied in laboratory animals first to determine potential toxicity before they can be tried in people. Treatments having acceptable safety profiles and showing the most promise are then moved into clinical trials.

Although "new" may imply "better," it is not known whether the potential medical treatment offers benefit to patients until clinical research on that treatment is complete. Clinical trials are an integral part of new product discovery and development, and are required by the FDA before a new product can be brought to the market.

The FDA is committed to protecting the participants of clinical trials, as well as providing reliable information to those interested in participating. Recently, unethical behavior on the part of some researchers has shaken the public trust and prompted the federal government to establish regulations and guidelines for clinical research to protect participants from unreasonable risks.

Although efforts are made to control risks to clinical trial participants, some risk may be unavoidable because of the uncertainty inherent in clinical research involving new medical products. It's important, therefore, that people make their decision to participate in a clinical trial only after they have a full understanding of the entire process and the risks that may be involved.

Why Participate in a Clinical Trial?

People volunteer to participate in clinical trials for different reasons. Some volunteer because they want to help advance medical knowledge. Others have tried all available treatments for their condition without success.

In a spring 2000 Harris Poll of cancer clinical trial participants, 76 percent of the respondents said they participated because they believed that the trial offered the best quality of care for their disease. Helping other people and receiving more and better attention for their own specific disease were other reasons cited.

"I was going ahead with the stem cell transplant because I thought I had no other options," Meritt admits. "Part of the reason I decided to do this study was so that maybe what they learned through me would help other people with mantle cell lymphoma." Others enter clinical trials in hopes of finding a potential treatment, after traditional therapies fail.

People should not, however, be tempted to enroll in a clinical trial simply because a potential treatment is being offered free during a study, or because of the promise of money, says David Banks, an FDA pharmacist.

"People lured by compensation may overlook the known risks," Banks says. "Or [they may fail] to adequately appreciate the potential for discovery of serious new side effects during clinical testing of a new treatment." Banks also says that clinical trials "are generally not a means for patients to receive long-term treatment for their chronic disease." Still, he adds, "clinical trials often represent an option to seriously consider."

Who Can Participate?

It's important to test medical products in the people they are meant to help. In the past, most new drug testing had been done on white men. Groups such as women, African-Americans, and Hispanics often were not adequately represented. It's important to test medical products in a wide variety of people because drugs can work differently in people of various ages, races, ethnicity, and gender. The FDA seeks to ensure that people from many different groups are included in clinical trials.

Trial guidelines, or eligibility requirements, are developed by the researchers and usually include criteria for age, sex, type and stage of disease, previous treatment history, and other medical conditions. Some trials involve people with a particular illness or condition to be studied, while others seek healthy volunteers. Inclusion or exclusion criteria — medical or social standards used to determine whether a person may or may not be allowed to enter a clinical trial — help identify appropriate participants and help to exclude those who may be put at risk by participating in a trial.

Volunteering for a clinical trial is no guarantee of acceptance. Similarly, there's no guarantee that an individual in a clinical trial will receive the drug or medical product being studied.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.