From the Archives: Spring 2008 issue of Lupus Now
UNDERSTANDING CLINICAL TRIALS: Clinical trial participants make a difference in lupus research by Bruce Goldfarb
Medical science progresses by small, incremental steps. Every advance in the understanding of a disease such as lupus -- its causes, its effects on the body, the development of new drugs and treatments -- is based on research.
Every year, more than 80,000 clinical trials are conducted in the United States. In the first article in this series on clinical trials (in the Fall 2007 issue of Lupus Now), we explained that there are five types of clinical trials. The type of clinical trial that evaluates a new drug or therapy is called a treatment trial.
In order for the U.S. Food and Drug Administration (FDA) to approve a drug and make it available to people, the drug must be proven safe and effective by well-controlled treatment trials. To conduct these trials and bring new therapies to market, researchers need people with lupus to participate -- lots of them.
T.C., a 26-year-old pre-med student living in the East Bay area of San Francisco, fills an invaluable role in the pursuit of lupus research by participating in a clinical study.
"This is the only way medicine moves forward, if people participate in these clinical trials,” says T.C., who asked that her full name not be used.
T.C., who was diagnosed with lupus four years ago, learned from her rheumatologist about a clinical trial of a potential new drug for lupus -- it is one of 20 or more possible new treatments that are currently in the drug development pipeline. Her doctor posed this experimental treatment as an option during one of T.C.’s regular office visits.
"My treatment was going to change anyway because I had a flare while on methotrexate [Folex®, Rheumatrex®]," she says. The trial offered her the chance to try rituximab (Rituxan®) instead. "It was the right timing, and there was a possibility that it might work. I had the opportunity, and I knew I had the time in the coming year to commit to it. It was a good option for me to pursue."
Rhonda Johnson, a 41-year-old school bus driver from Charleston, SC, is participating in a clinical trial for a new drug intended to treat lupus nephritis. She views it as a way to improve treatment and quality of life for people like her. "It’s rewarding, because in the end I know I’ve helped others who are going through what I did," she says. "It’s something that might help me, but in the long run it will help others."
For many people with lupus, participating in a clinical trial is empowering, giving them a way to belong to a larger team focusing on their particular health concerns and assuring them of a thorough evaluation by a highly qualified team of health professionals with particular expertise in lupus.
"Regular clinical visits are covered by the clinical trial, and whereas a typical visit is limited to 20 minutes, I spend 45 minutes to an hour with a lupus trial participant," says Jennifer Grossman, M.D., assistant clinical professor of rheumatology at David Geffen School of Medicine at University of California, Los Angeles.
"Being enrolled in a clinical trial definitely provides an opportunity to have a lengthy, less pressured discussion with a doctor who is a specialist," says David Fiorentino, M.D., Ph.D., assistant professor of dermatology and medicine at Stanford University School of Medicine, who has conducted several clinical studies and is about to launch another to test a method of measuring the severity of cutaneous lupus.
"Participants may learn new ways to cope with their disease, and also may gain access to available resources, like support groups, they may not have known about," he says.
T.C. agrees. "Some people like taking part in clinical trials because they get even more attention from medical staff. For me, I feel like I’m being more active in my own health, which is always a good thing."
Trial participation also offers more immediate, practical benefits. Participants can receive experimental -- and expensive -- medications that relieve symptoms. These are medications that often aren’t covered by insurance carriers because they’re not specifically indicated for lupus. "Even if they are covered, co-pays can be as high as $600 a month," Grossman says.
Participation Is a Two-Way Street
During visits with trial participants, Grossman and her staff check lupus symptoms and conduct lab tests paid for by the trial sponsor. Her office shares test results with the participants’ other doctors, so that tests aren’t repeated. She also answers questions, educating participants about lupus, while they in turn educate her about how lupus affects them.
Fiorentino encourages people with lupus to participate in clinical trials because researchers cannot discover important new information or develop new and better drugs to treat the disease without the help of clinical trial volunteers.
"There’s no question that there are still important things to learn about lupus," says Fiorentino. "For example, we know there’s a genetic basis for lupus, but we still need to learn what environmental triggers precipitate lupus in a particular individual. We really don’t fully understand how lupus starts to involve the skin, or which elements are the first that go awry in creating the skin lesions."
"By participating in a study, people with lupus can take part in the research process," he adds. "That’s gratifying for many people. They’re contributing to knowledge."
Moreover, if people aren’t willing to participate in these trials, companies will spend their money developing medicines for other diseases, says Richard Furie, M.D., chief of the Division of Rheumatology and Allergy-Clinical Immunology at North Shore-Long Island Jewish Health System in New York. "You need two large trials that enroll hundreds, and in some cases thousands, of patients for the FDA to approve a drug," says Furie. "It’s costly for companies to set up and enroll patients in these trials. So it’s important for lupus patients to get involved. We have a narrow window of opportunity here."
However, it is important to remember that not everyone in the trial will get the medication being tested -- some people will get a placebo, or "sugar pill," that doesn’t contain any of the active drug.
That is why Fiorentino cautions people not to enter a clinical trial if the only reason is for the benefit that the investigational drug might offer. "You don’t know what you’ll receive -- whether the drug or the placebo. More importantly, a drug being studied in a clinical trial is developed to control disease symptoms, and is not expected to be a cure."
That said, some trials involve multiple medications, in which case even participants on placebo get symptom-relieving drugs they likely couldn’t otherwise afford even with insurance co-pays, says Grossman.
For example, in one recent lupus nephritis trial comparing the symptom relief of two medication combinations, one group of participants took mycophenolate mofetil (CellCept®), steroids, and placebo, and the other group took mycophenolate mofetil, steroids, and rituximab. The researchers sought to determine whether participants receive added benefits when rituximab joins the mix.
And for those concerned about being locked into a nontreatment group, Grossman offers reassurance: "Whether or not you’re on placebo, the researchers in charge make every effort to make sure you’re doing well. And if you’re not, you’ll be withdrawn from the study."
Understanding the Process
One of the first considerations in deciding to volunteer for a research study is finding out if you qualify to be a participant. All clinical trials have strict guidelines for the individuals who are needed for the research. Guidelines are based on factors such as age, type of disease, medical history, and current medical condition. Guidelines are there to protect the potential participant.
The factors that allow you to participate in a clinical trial are called "inclusion criteria" and the factors that would prevent you from participating are called "exclusion criteria." The criteria are used to identify appropriate participants and keep them safe, and to help ensure that researchers will be able to answer the questions they plan to study.
Yet, as personally beneficial as it can be to those enrolled, participating in a clinical trial often requires a commitment beyond what many people first consider. "Study participants may not realize that a clinical trial can be very different from seeing your regular doctor for basic health care," says Fiorentino. "Many trials involve office visits that can be lengthy, there are usually a lot of tests, like blood draws, and sometimes there can be other, more invasive tests, too. It’s important to understand the commitment."
He stresses that anyone who is considering taking part in a clinical trial should be sure to read everything about the study that the researchers provide. "Also, speak with your doctor about any questions or concerns you may have, and make sure you thoroughly understand the nature of the study, its purpose, and your role in it," he adds.
T.C. says she goes in for an office visit once a month. "The doctors running the clinical trials want to know about everything that happens to your health," she says. "Before each visit, I have to sit down and figure out what’s been going on with my health in the past month. It’s time and effort, but it’s not wasted."
In Johnson’s case, office visits are scheduled during the down time between her morning and afternoon school bus runs. "The doctors and their staff make it really convenient for me," she says. "They do a good job of arranging my office visits and lab tests around my schedule. They take excellent care of me."
Work or school conflicts and childcare issues are among the reasons given most often for withdrawing from a clinical trial, according to Fiorentino. Research coordinators can help resolve obstacles, and although researchers are typically prohibited from compensating people for their role in a clinical study, there can be support available to reimburse participants for travel and parking costs and other out-of-pocket expenses. Some trials also offer a modest stipend for their time.
Now in the 10th month of her participation in a 12-month clinical trial, T.C. says it has been a rewarding experience and that she would consider participating again in the future. "It’s very important to be part of this process that can help other people with lupus," she says. "I might do it again if it made sense for me and fit into my schedule. Overall, it’s been a very positive experience."
For More Information
For many people with lupus, participating in a clinical trial is empowering, giving them a way to belong to a larger team focusing on their particular health concerns.
Clinicaltrials.gov -- A website provided by the National Institutes of Health (NIH) with a searchable database of all NIH-sponsored clinical trials. Also has a list of frequently asked questions and other resources for people considering participation.
CISCRP.org -- The Center for Information and Study on Clinical Research Participation. A nonprofit organization dedicated to providing information about clinical research participation. Call toll-free 888-CISCRP3 (247-2773) or send an email to info@ciscrp.org.
CenterWatch.com -- A website that links researchers with people interested in participating in clinical studies and has information, resources, and a searchable database of trials.
"Taking Part in Research Studies: What Questions Should I Ask?" – A PDF Brochure from the National Prevention Information Network of the Centers for Disease Control and Prevention. Available at www.cdcnpin.org/Brochures/ResStudy.pdf or call toll-free at 800-458-5231.
To read the first article in this series on clinical trials, click on "Back Issues."
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