Should You Participate in a Clinical Drug Trial?

Do you know someone who has participated in a clinical drug trial? Years ago, your answer would probably have been no, but that may have changed. In recent years, the number of clinical drug trials has increased dramatically and researchers are looking for participants.

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According to ClinicalTrials.gov, as of late August, more than a quarter million trials had been registered worldwide (all 50 states and 200 countries) since the count began in 2000. But before you decide to participate in one, you'll want to know the pros and cons, noted below.

Clinical trials are research studies that track how well a new drug or other therapy works. Trials also document any harmful side effects and how well participants tolerate the drug or therapy.

The goals of clinical drug trials are to determine better ways to prevent, detect, diagnose and treat diseases and other health conditions. Without clinical research, new drugs would not be developed and made available to those who need them. By the time doctors prescribe drugs, they have been through numerous clinical trials, years in the making, before they are ever made available to patients.

All clinical trials are submitted to an institutional review board before startup to determine if they are reasonable from an ethical and safety standpoint.

The National Institutes of Health and the U. S. Food and Drug Administration (FDA) provide additional information about clinical trials.

Says Dr. Ronald K. Mayfield, medical director and an investigator at Mountain View Clinical Research in Greer, S.C., whose involvement with clinical research spans nearly 30 years: "Participation in clinical trials is not without risks; however, patients are closely monitored and there are often health benefits. Patients receive education about their disease, access to a drug that might not be available because of cost or availability outside the trial, and, in many cases, see improvement because they are paying more attention to their health."

Prior to beginning a trial, participants must read the purpose and requirements and sign a consent form. A screening process follows, which usually includes basic testing and determination of factors that may determine if a person should be included or excluded, which are specific to each study.

A trial may include run-in and follow-up phases during which participants aren't receiving the drug, but are documenting aspects of their health and being monitored. During the trial, in addition to regular clinic visits, some participants are required to keep journals, call in periodically or log in to an online site daily to answer questions.

Lisa Cox received a diagnosis of Parkinson's disease in 2009. Since then, she has participated in three clinical trials.

"Because I trusted my doctor, I was confident he wouldn't involve me in something that might harm me. He had years of experience with research so I had no fears about participating in a clinical trial he suggested," Cox says.

The first clinical trial she participated in came a year and a half after her diagnosis. The trial was located in another state, where she traveled once a month for testing to make sure the drug wasn't having an adverse effect on her vision or producing skin cancer. Occasionally, Cox had to wear a heart monitor.

"There were times when being part of the study that required me to drive to another state was inconvenient, but if people don't volunteer for trials, we don't move forward with research," Cox says. "One of the difficult things about being in a trial is you don't know if you are actually getting the drug. Initially, you may be experiencing a placebo effect and not really on the drug. Another thing that is challenging is the amount of information you're required to divulge about yourself, your body functions and personal life. If someone is a very private person, I can see how this would feel extremely invasive."

After a little over a year, Cox's doctor suggested she come off the study because she wasn't experiencing an appreciable difference in symptoms. She never knew if she was on the drug. Often, trials are blind studies in which participants don't know what drug is being tested, if they are on it or what its strength is.

Cox later participated in shorter studies. "Many people don't realize a clinical trial can be short — a day or even a few hours — and that volunteers are also needed to serve as control subjects. For one study, my husband and I drove to Birmingham, Alabama, for just a few hours of testing. My husband participated as a control."

Researchers find clinical trial participants several ways.

"At Mountain View, we actively seek volunteers," Mayfield says. "We have a recruitment division that works with doctors in the area for referrals, [and] advertises and targets social media, which has proven very effective. We refer to our volunteers as heroes because they provide an important service."

Dr. John B. Buse, director of the North Carolina Translational and Clinical Sciences Institute and chief of the Division of Endocrinology at the University of North Carolina, says: "My most successful method of recruiting volunteers has been through my own practice. We also communicate with the primary care doctors who refer patients to us."

For those interested in looking for trials on their own, ClinicalTrials.gov has the most comprehensive listing of public and private clinical studies worldwide. Buse also suggests looking at sites related to your particular disease or condition (for example, type 1 diabetes) or browsing the sites of health care systems in your area. Another option is Patient-Centered Outcomes Research Institute (PCORI).

Buse gives several reasons why he feels people participate in clinical research. "Often, volunteers want to be a part of providing better care for people in the future, particularly if they have a disease or condition that might predispose the next generation of their family. Also, clinical trials provide access to new therapies if what people are currently doing is inadequate or they can't afford a drug."

Cox adds her own perspective: "I feel anyone who volunteers for research is a pioneer and I appreciate all who have gone before me."

"While it is challenging to put studies together and have them approved, we are finding better answers more efficiently and quickly and seeing progress in treatment and care. That is rewarding," Buse says.