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U.S. Food and
Drug Administration
FDA Consumer magazine
January-February 2002
Table
of Contents
By Michelle Meadows
When Jim Anderson of La Plata, Md., became an FDA patient representative in 1997, he wasn't sure how much help he could be. In preparation for his first advisory committee meeting, the Food and Drug Administration sent him a new drug application (NDA) briefing package, which he describes as "10 pounds of paper."
"I couldn't pronounce half the words," he says, "and I was scared to speak ..."
But the fear didn't last long, says Anderson, who was diagnosed with prostate cancer in 1993. He recalls reviewing a drug that gave "a small chance to provide pain relief and a significant risk of heart failure," in his opinion. He was glad he spoke up about it because his concern prompted a serious discussion among the scientists and doctors.
Anderson was one of about 23 patient representatives who gathered in Rockville, Md. for an FDA training workshop in September. Since 1991, patient representatives have served on FDA advisory committees to help review products. Advisory committees provide a forum through which the FDA seeks advice from outside experts and consumers.
Patient representatives give the FDA and its advisory committees insight on issues and questions important for patients and family members living with serious and life-threatening illnesses. The patient representative program began after Congress passed legislation requiring consumer representation on advisory committees and HIV/AIDS advocates demanded to be included on advisory committees that review products affecting their lives. Over the years, the FDA saw the significant contribution HIV/AIDS patients made to the regulatory review process, and the distinct category of patient representatives was expanded to include other committees.
FDA experts in the areas of drugs, radiological health, medical devices, and biologics briefed those at the workshop on the agency's product review process. Among the workshop agenda topics was how to sift through the hefty NDA applications, which contain results from animal studies, clinical tests, and other drug information. Patient representatives should expect to spend at least 10 hours reviewing the material before an advisory committee meeting.
Patient representatives typically have experience with and are knowledgeable about a specific serious or life-threatening illness. They also typically have a formal affiliation with a patient advocacy organization. About 60 patient representatives now serve on the FDA's 32 advisory committees.
Most patient representatives serve on committees that review products related to HIV/AIDS and cancer. But additional diseases, such as arthritis, diabetes, lupus, and Parkinson's disease, are also included. Patient representatives are reimbursed for travel, lodging, and daily expenses. A modest honorarium is provided for each day of service at the meeting.
During the fall workshop, Steven Hirschfeld, M.D., Ph.D., a medical officer in the FDA's division of oncology drug products, described the challenge of pinpointing the indicators that reasonably predict whether a drug will have a benefit. "When it comes to approving a drug's use, you want to prolong someone's life, make it better, or both," Hirschfeld says. "The hard part is figuring out what and how you can determine what 'better' means. A change in the size of tumor nodules may or may not have a benefit."
Hirschfeld advises patient representatives to make a list of the questions they think are critical and then work to find the answers in the NDA briefing documents. Typical questions include: What are the claimed benefits? How durable is the benefit? What are the risks? Are the patients in these studies representative of the typical patient? Does the data prove what the company claims it does or is it just interesting data that indicates the need for further research? Then, he says, make a list of the answers to these questions, with both the company analysis and the FDA analysis side by side. Such a list helps patient representatives focus on what questions and points to bring up during the meeting.
"Ask the hard questions" of the pharmaceutical companies and of the FDA analysis, Hirschfeld says. Some common problems with NDA submissions are incomplete data and multiple terms for the same type of adverse event. An event may be described as "liver toxicity" in one section and "right upper quadrant pain" in another. Another thing to watch for, he says, is a claim after the fact of an unexpected benefit. For example: "It didn't shrink tumors after all and it did not prolong life. But the people in the study felt their memory improved." This may be an important observation for the design of a future study, Hirschfeld says, "but it should not be construed as the basis for approval of the application under review."
Anderson says, "Over the last five years, I have become more sensitive to what a big job FDA has." He has also been selected for the FDA's cancer drug development patient consultation program, a recent extension of the patient representative program. The consultation program is a joint effort between the FDA's office of special health issues and division of oncology drug products. Its goal is to bring patients into the drug development process earlier than at the advisory committee level, which comes later.
Hirschfeld says, "No one else will have the experience and perspective of a patient familiar with a disease to determine if a benefit is meaningful or if the burden is severe." According to Hirschfeld, patient representatives don't need to be statisticians. "The idea is that you pick out key points, ask whether there is a benefit that justifies the risk, and make your point of view known," he says. "And don't be seduced by an eloquent speaker with color slides--from either the pharmaceutical company or the FDA."
For more information on the patient representative program, write to the FDA's Office of Special Health Issues (HF-12), Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 20857, 301-827-4460. Or, visit www.fda.gov/oashi/patrep/patientrep.html on this Web site.
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2001-DEC-26.