Table of Contents
FDA Consumer magazine
September-October 2000
by Carol Lewis
Should the latest drug to treat AIDS be allowed on the market or will it put patients at risk? One member of the committee evaluating products for the Food and Drug Administration has difficulty casting her vote. She is not a doctor, not a scientist, not even a health-care provider, but her opinion counts. And she faces a complicated choice. On the one hand, not enough multicultural studies have been done to her liking to determine the drug's overall safety and effectiveness. On the other, the desperate patient whose life the medication may save sits silently pleading beside her.
"There were nights I didn't sleep because I wondered if I'd made the right decision," says Susan Cohen, one of many individuals grappling with the complex, science-based issues related to FDA's public health responsibilities. Cohen serves on a committee of outside experts and consultants who advise FDA in its evaluation of regulated products and help the agency make sound decisions based on the reasonable application of good science. It may seem unnecessary to seek outside advice, given the full complement of scientific specialists the agency employs, but as scientific advances in the many areas that FDA regulates become more specialized, the agency sometimes looks to the critical expertise that exists at major research institutions for a more balanced evaluation. Consumer representatives like Cohen contribute to the scientific debate by ensuring that the health-care concerns of specialized groups, such as the elderly, the minority, and the disabled, are adequately represented on FDA's 32 advisory committees.
Although Cohen, who recently served on FDA's Antiviral Drugs Advisory Committee, eventually voted to recommend approval of the AIDS drug, these decisions, she says, are never made without lingering doubt.
"The United States is made up of so many different cultures and you need to include data on all these groups when you're considering marketing a drug that's going to help a lot of people," she says. At the same time, while trying to stay objective about the criteria she uses for making a judgment, the consumer advocate is also aware that sometimes there's a personal side to the issue. "When you're voting, you are very conscious of the person sitting next to you because he represents that immediate segment of society that needs the help."
But do these outside opinions really make a difference? FDA thinks so.
"Advisory committees are among the most important FDA institutions where representatives of the public have a place at the table," says Sharon Smith Holston, FDA's Deputy Commissioner for International and Constituent Relations. "And because of them, various public sectors have gained a better understanding of the frequently complex reasons why new products are--or are not--allowed on the market."
Historically, FDA made regulatory decisions concerning new and investigational products with only occasional assistance from the outside. Independent advice was limited to difficult issues on an as-needed basis and only to compensate for a lack of specific expertise within the agency. But over the last 50 years, FDA found that it could best protect the public health if the people it served better understood its strengths and limitations, and if they partnered with the agency in a common enterprise. In short, the traditional relationship between FDA and the public had to change.
One telling example occurred shortly after World War II, when the agency, through a series of public meetings, consulted the lay public in its development of standards of identity for certain food staples. To find out what ingredients Americans wanted--or didn't want--in white bread, FDA asked for the views of not only nutritionists and manufacturers of bread, but also of consumer organizations representing the nation's proverbial man in the street. Based on consumers' concerns, FDA made sure the adopted standards reflected the public's wishes and recommendations.
Encouraged by this experience, the agency took the next step by hiring part-time consumer consultants to work on other food standards and similar issues. These earlier successes eventually evolved into a full-scale public-participation program that would later include patient and consumer representatives like Cohen.
In 1972, Congress passed the Federal Advisory Committee Act to promote the formal use of advisory committees in all of the federal government, just as FDA's public participation system was evolving into more extensive use. FDA's advisory committees provide independent, expert scientific and medical advice to the agency on the safety, effectiveness, and appropriate use of products under its jurisdiction. The committees consist of individuals with recognized expertise and judgment in a specific field, and who have the training and experience necessary to evaluate information objectively, often under controversial circumstances. The advisory committee process encourages public interaction with the agency in arriving at decisions, and members of the public are encouraged to appear before the committee during the open part of each meeting.
But the big advance came in 1976 when Congress amended the Federal Food, Drug, and Cosmetic Act and made FDA responsible for evaluating and approving medical devices. At the same time, the law mandated that the interests of the general public would now be represented on medical device advisory committees by non-voting consumer members. President Carter acknowledged the growing importance of public representation in 1978, when he formally called on all federal agencies to "provide adequate opportunity for consumer participation in the decision-making process."
Today, the charter of each advisory committee provides for at least one voting or non-voting member to represent the consumer perspective. Consumer representatives raise concerns that might not otherwise be addressed before products come to the marketplace, while other committee members focus on these issues from the scientists' and clinicians' perspectives.
Typically, and depending on the focus of the particular committee, consumer representatives who have served in the past haven't exactly been lay people. They have included consumer advocates, college professors, consumer lawyers, nurses, physicians, microbiologists, engineers and veterinarians. Most have ties to consumer- and community-based organizations. Although they are not necessarily experts in their, like their counterparts, consumer representatives must be able to analyze data, understand research design, discuss benefits and risks, and evaluate the safety and effectiveness of the products under committee review--but from the consumer perspective.
"A consumer member on one of the FDA panels needs to recognize that she or he is always in the minority, thus placing her or him at a disadvantage," says Diony Young, consumer representative for FDA's Obstetrics/Gynecology Devices Panel. But even though she doesn't have the expertise that other panel members have, Young emphasizes that consumer representatives must be strong advocates on behalf of the public, and that they "must not be intimidated or coerced by physicians and others with higher professional credentials."
Although consumer representatives must be technically qualified to serve on advisory committees, FDA's Acting Associate Commissioner for Consumer Affairs, Patricia M. Kuntze, says their role isn't always understood by other committee members.
"We provide information and training, and actively participate in orientation sessions so that other committee members are aware of the role of the consumer representative on any given committee," Kuntze says. "However, sometimes it takes the experience--and I can guarantee that after one or two meetings, no one on the committee has any questions or reservations about the added value of the consumer representative."
Consumer representative Abbey Meyers adds, "I only know that when I read the background materials, and when I listen to the scientific presentations, I must never forget the patients." President of the National Organization for Rare Disorders in Connecticut and consumer representative for the Biological Response Modifiers Advisory Committee, Meyers feels, "If my allegiance is to them, and if I always focus on their welfare, I will have served them well even if the rest of the committee disagrees with me."
Barbara Loe Fisher, on the other hand, was surprised when FDA wanted to appoint her to the Vaccines and Related Biological Products Advisory Committee, which is responsible for evaluating the quality of scientific data presented by manufacturers applying for licenses for new vaccines. A nationally known critic of the mass vaccination system, Fisher has spent the last 18 years publicly calling for the institution of vaccine safety and informed consent reforms.
"This was not a passive appointment where I could sit quietly and just listen," Fisher says. "It became clear to me early in that first meeting that the contribution I uniquely could make, and had to make, was to be the voice that had rarely been heard there before." But Kuntze says that's just what the agency is looking for--those who represent the different points of view that balance the information committee members have to weigh.
"There's synergy in the panel as a result of the type of people who are on it," says committee member and former chairman of the Clinical Chemistry/Clinical Toxicology Devices Panel, Henry C. Nipper, Ph.D. "We all help each other out, we all learn from each other, and I believe the panel is stronger because of this good mix of backgrounds and interests."
FDA formalized a selection process, called the Consumer Consortium, that involves consumers in the recruitment, nomination, and recommendation of consumer representatives to serve on FDA committees. The consortium may involve 10 to 15 members representing consumer and patient advocacy groups, such as the National Women's Health Network (NWHN) in Washington, D.C., who serve a diverse range of constituencies. FDA's Office of Consumer Affairs (OCA) is responsible for coordinating the nomination and selection process, and facilitates consortium discussions so that consumers can evaluate candidates for consumer representative vacancies on advisory committees. At least nine months in advance of a vacancy, OCA consults with the agency's manager of the committee (the Executive Secretary) to determine the scientific or technical expertise that is required.
"It makes certain seats hard to fill," says Cynthia Pearson, Executive Director of NWHN, a current consortium member. "Having a consumer representative at the table is essential, yet consumers don't always have enough science at hand to fill the role." Others, she says, are masters at their craft. "Candidly, it's a mixed bag, but we've seen that consumer representatives can and do sway opinion."
Additionally, OCA conducts extensive recruitment efforts through a host of other channels including self-nomination, current and former consumer representatives, and outreach efforts to key consumers.
In order to learn the views of people who were intimately familiar with the devastating effects of such raging epidemics as HIV infection and AIDS, the agency created a new category of public participation--patient representatives--and added them to the advisory panels that dealt with various aspects of the diseases. Prompted by both the valuable contributions that consumers were making on regulatory issues not readily apparent to the legal experts and scientists, and the shock of the 1980s AIDS epidemic, FDA believed that the process of including consumer representatives could serve as a model.
In the fall of 1988, activists of an organization called ACT UP (AIDS Coalition to Unleash Power), consisting mostly of young men with AIDS and their friends and relatives, stormed FDA headquarters, demanding immediate access to unapproved therapies to fight AIDS and the HIV virus. Protestors accused the agency of being too slow in reviewing AIDS drugs and of conducting business "as usual" while they were dying. Some of the demonstrators were dressed like corpses in a morgue and carried signs that said, "I died for the sins of FDA," "Time isn't the only thing the FDA is killing," and "Killed by the System."
"The entire demonstration, genuine as it was in its intent, was predicated on wrong assumptions," says Deputy Commissioner Holston, who was trapped inside the building behind police protection during the rally. She says that FDA was not barring access to promising new antiviral drugs, and that the agency, in fact, was far from slow in processing AIDS applications for therapies, to the extent that there were any to process.
Zidovudine, or AZT--the only drug against AIDS then in existence--had been approved by the agency in March 1987 in three-and-a-half months, she says. Prior to that, it had been given to 4,000 people with AIDS in a study that FDA authorized in just four days. According to Holston, the ACT UP protest turned out to be a distortion of truth, but an experience that actually inspired the agency to examine public participation with a new sense of urgency.
The later appointment of patient representatives enabled FDA to conduct far-reaching and thorough consultations on how to confront the AIDS epidemic and mitigate its enormous suffering, while tackling the overwhelming scientific and medical problems associated with the disease. Although there was some initial resistance by committee members to include patient representatives on the panels, FDA found that their opinions paid significant benefits. For example, some of the activists understood the scientific problems faced by the agency in reviewing new antiviral drugs, and this was a very important added benefit--they were very successful not only in presenting the AIDS community's views to FDA, but also in explaining FDA's positions to people with AIDS. In addition, AIDS patients could judge better than anyone what drug side effects were acceptable in return for possible health benefits.
"We see that more and more patients with serious and life-threatening diseases are actively involved in their own treatment," says Theresa Toigo, Associate Commissioner for Special Health Issues. "Our informal evaluation tells us that this initiative has not only been accepted but is endorsed by scientists, FDA staff, and the pharmaceutical industry."
In fact, the success of AIDS patient representatives stimulated a similar initiative for cancer patients and their advocates. In the early 1990s, following discussions with representatives of the cancer patient community, FDA established a cancer liaison program within its Office of Special Health Issues to work closely with these patients. And because there are too many types of cancers for a single patient to represent the entire field, public participation took another step forward in March 1996 when President Clinton announced a new policy that provides for voting patient representatives on all FDA advisory committees dealing with cancer-related issues. This program has included over 26 patient representatives on 16 advisory committees to review 40 cancer-related therapies. One patient representative has served five times.
Patient representatives often have a history with the disease for which a new treatment will be discussed. In some cases, a caregiver or representative of a patient group might be chosen to serve. As with consumer representatives, a background as a scientist or researcher is not necessary, as long as the representative can comprehend the scientific data presented and effectively communicate patient concerns. More importantly, their experience as a patient enables them to bring a unique perspective to FDA advisory committees.
"Patients can offer a point of view that no one else on the committee can provide because we've had the experience to share what they haven't," says patient representative Martha Solonche, a five-year survivor of both uterine and ovarian cancer. Solonche believes that the "emotionally charged" nature of some of the issues is "all the more reason for someone with the disease to argue a point."
"One of the biggest hurdles at a committee meeting," says Sallie Forman, a patient representative who suffers from colon cancer, "is convincing the rest of the panel that the approval of a drug that may buy me three or four more years may also take away my quality of life. In other words, a drug that is so toxic to the system, but gives one more week of life, isn't worth it."
Solonche agrees. "No one on the panel could understand this. Only someone who's lived through constant side effects, every time they take a certain drug, can know what that's like."
Public participation at FDA has become a two-way process through which the agency communicates priority health information to the public and the public in turn expresses its views, attitudes, reactions, and knowledge to FDA.
"Together, this team of advisors delivers a valuable external viewpoint about difficult issues that face the agency," says FDA Commissioner Jane E. Henney, M.D. And its growing emphasis, she says, clearly demonstrates that consumer and patient contributions to the advisory committees are significant. As a result, communications have improved between FDA officials and committee experts, and the public has begun to feel more involved in the agency's decision-making process.
Carol Lewis is a staff writer for FDA Consumer.
To find out more about becoming a patient or consumer representative on an FDA advisory committee, send a resume to one of the following offices:
For Consumer Representatives:
Food and Drug Administration
Office of Consumer Affairs (HFE-40)
Advisory Committees Desk
5600 Fishers Lane
Rockville, MD 20857
For additional information on consumer representation, call 301-827-5006.
For Patient Representatives:
Food and Drug Administration
Patient Representative Program
Office of Special Health Issues (HF-12)
5600 Fishers Lane
Rockville, MD 20857
For additional information on the patient representation program, call 301-827-4460.
--C.L.
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