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FDA
Sharon Smith Holston
Deputy Commissioner for External Affairs
10th IMS International Symposium
Brussels, November 3, 1997
Good afternoon, and thank you for this opportunity to discuss the relationship between the United States Food and Drug Administration -- FDA -- which I have the honor to represent, and patients.
This is an important subject for FDA. Let me show you one reason, why. [start the projector] You know the saying that a picture is worth a thousand words. This is the scene outside our agency's headquarters one morning in the fall nine years ago. These are activists of an organization called ACT UP -- "AIDS Coalition to Unleash Power" -- mostly young men with AIDS and their friends and relatives. They came to accuse FDA 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," as this slide shows: "Time isn't the only thing FDA is killing," and "Killed by the System." We, at FDA, were "the System." The demonstrators were demanding immediate access to unapproved therapies to fight AIDS and the HIV virus. The entire demonstration, genuine as it was in its intent, was predicated on wrong assumptions.
In fact, FDA was not barring access to promising new anti-viral drugs, and the agency had been 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 three-and-a-half months, and prior to that, had been given to 4,000 people with AIDS for a study that FDA authorized in just four days. Treatment IND, a new FDA mechanism that expanded drug availability and greatly speeded up agency review and approval for critical drugs, had been introduced two years prior to this demonstration; another accelerated process -- so-called Subpart E regulations -- was ready for publishing; and FDA's division of antiviral drug products and a retrovirology lab -- both of which had been established to fight against AIDS -- were staffed with dedicated people and functioning.
Objectively, the demonstration was a furious outburst aimed at the wrong target. But subjectively -- for myself and many of my colleagues who were trapped inside the building as demonstrators tried to break through police lines to get us -- the ACT UP protest rally was a wrenching experience that inspired much introspection. We could not understand why all of our efforts were so badly misunderstood, and frankly, some of us were angry at being cast as the "bad guys."
The demonstration became a watershed event in our changing perception of how to best accomplish FDA's traditional mission of protecting consumers and promoting the public health. Starting with the original Food and Drug Act of 1906 and continuing until the mid 1980s, the directives we received from Congress and the White House were, in essence, to accept more responsibilities; and above all, to be thorough in protecting the public.
In 1938, the Federal Food, Drug and Cosmetic Act greatly expanded FDA's powers and directed the agency to make sure that drugs are safe. In 1962, an FDA reviewer who refused to approve a drug until she had more evidence of its safety was honored by President Kennedy with our country's highest award for distinguished federal civilian service. It was Dr. Frances Kelsey, whose refusal to be hurried saved the United States from the thalidomide tragedy.
Clearly, the message was that it is better to take more time and be certain, than to move quickly and possibly make a tragic mistake. In the same year, Congress passed a law that made our review process slower but still more thorough, by requiring that FDA screen all new therapies not only for safety, but also for effectiveness. In 1976, another law directed the agency to ensure also the safety and effectiveness of medical devices.
And all along, the American public seemed to be perfectly comfortable in the cocoon of safety provided by the laws and the agency's regulations. As late as the mid 1980s, in public opinion polls FDA ranked as the second most appreciated government agency, right after the National Parks Service. We felt honored to be number two after the national parks, where millions of Americans and others spend their vacation. We must have been doing something right. We were the good guys.
The contrast between these decades of approval and the message we received from the ACT UP demonstrators was like the difference between day and night. In their eyes, we were not an agency that protected their health, but an aloof, impersonal, uncaring and merciless "System" -- a bureaucracy that was killing them.
This indictment, uninformed and terribly unfair as we believe it was, taught us a lesson. We realized that no matter how hard we tried, we could not do our best to protect the public health unless the people we served understood better our strengths and limitations, unless they cooperated with us, and unless we became partners in a common enterprise. The traditional relationship between FDA, the active protector, and the passive, protected patient, had to change.
I cannot go into all of the repercussions of this new attitude at our agency, but I can give you a few examples of the policies that reflect our increased appreciation of patients' right to be empowered, and their capacity to contribute to our mission.
The most telling example of the importance we place on the role that patients can play in health care decisions is the agency's policy of including them in FDA's most important and difficult process -- the approval of new medicines and medical devices. The origins of this policy go back to the years following World War II, when FDA sought out consumer opinions on what standards should be established for certain food staples.
The agency found that the consumers provided valuable perspectives on regulatory issues that were not readily apparent to the legal experts and scientists. We therefore began including consumer representatives on the scientific committees of outside specialists who advise the agency on whether or not to approve new products under FDA review. In the 1970s these consultations with members of the public were adopted also by other government bodies, and subsequently, the policy that FDA initiated became law for all U.S. government agencies. Later, we formalized our selection process by creating a consortium of consumer organizations which meets a few times each year to review the qualification of consumer candidates, and to nominate some from among whom lay members for the advisory panels are selected.
I think, by any measure, that by the early 1980s the process of including consumer-nominated representatives was working well, but the shock of the AIDS epidemic moved us to make it still better. We set out to bridge the communications gap between our agency and the AIDS patients. In order to learn the views of lay persons who were intimately familiar with the devastating effects of the HIV infection and AIDS, we created a new category of public representative -- the representatives of patients -- and added them to the advisory panels that dealt with various aspects of the disease.
Although there was some resistance to this by some of the committee members, we have concluded that the decision has paid significant dividends. The AIDS patient representatives on our advisory panels have proven to he highly responsible and extremely effective at articulating issues of major importance to AIDS patients, such as the need for supplemental therapies and for data on surrogate endpoints.
They helped us focus attention on difficult risk and benefit questions, and could judge better than anyone what drug side effects would be accepted by AIDS patients in return for possible health benefits. Some of these activists displayed remarkable understanding of the scientific problems faced by the agency in reviewing new antiviral drugs. And -- and this was a very important added benefit -- they were very successful not only in presenting the views of their community to FDA, but also explaining FDA's positions to people with AIDS.
In fact, over time the views we heard in that patient community changed from angry accusations that we're moving too slowly to concerns whether we are taking enough time to review therapies before permitting access. There is now in the AIDS community a much greater understanding and appreciation for the complexity of the drug-approval process.
We've been also developing close relations with cancer patients and their advocates. In the early 1990s, following discussions with representatives of the cancer patient community, our agency established a permanent staff to work closely with cancer patients, and about four years ago a cancer liaison program was established in FDA's Office of Special Health Issues. Most cancer patients who call the agency want more and better information about cancer clinical trials, and our staff provides them with whatever information is available about treatments under investigation. In addition, our agency is collaborating with the National Cancer Institute and the National Action Plan on Breast Cancer to increase the number of cancer clinical trials that are registered in a publicly accessible database.
We're also including cancer survivors as voting and non-voting members on cancer-related advisory panels. Thus far, we have been informally identifying survivors who have experienced, or are living with, the type of cancer for which the particular therapy under review is intended. We are, however, developing a more structured process to recruit, assess, select and orient them so that they can use their special knowledge most effectively. And to further strengthen patient participation in FDA processes, our agency has implemented an oncology patient residency program to train potential patient representatives and enhance their understanding of the drug review process.
The oncology fellows spend time in the agency, learning from the agency staff that's involved directly in the review of cancer therapies. Two oncology fellows are currently enrolled, and two others will soon enter the program. We were extremely gratified that one of our fiercest critics -- who later died from breast cancer --eventually became our first oncology fellow.
I'd like to move briefly to another example of how we have tried to give patients a greater role in their own health care -- by providing them with more drug information about the drugs they use. The need to provide patients with more drug information than what they receive from their doctor or from the sticker label on their medicine was recognized already in the 1970s. To meet this need, FDA initiated -- and the pharmaceutical industry and pharmacists agreed to voluntarily carry out -- a program in which each new filled prescription was to be accompanied by a leaflet that would explain in lay language the drug's purpose, how it should -- or should not -- be used, what were the potential side effects, and include all warnings which the patient needed to know about and heed.
For many reasons, the early efforts did not catch on. In the late 1980s, however, three developments invested the program with a new sense of urgency. One of them was surveys showing that patients who received written drug information were more likely to follow their drug regimen and thereby avoided mismedications that caused many injuries and deaths, and added more that $70 billion to our annual health care bill.
The second new development was the widespread use of computers in pharmacies, and the availability of ready-to-print-out, patient-oriented drug information on diskettes produced by several companies. And the third, and most compelling reason for the agency's resolve to act, was our changed perspective, strengthened by what we were hearing from patients themselves, of the patients' right to know. Patients felt, and we agreed, that they should not be merely passive recipients of advice and care by the health care professionals, but had the right to information that would enable them to participate in decisions affecting their health. They wanted to be empowered with information that would put them in a better position to take charge of their own lives.
The confluence of these three factors resulted in a major appeal by FDA to the pharmacists to commit themselves to two goals established by a government program called Healthy People 2000. The first of these goals is to provide, by the year 2000, three out of every four patients who have a new prescription filled with written and useful drug information of the type I described a minute ago. The second goal, to be reached by the year 2006, is to make this distribution almost universal, reaching 95% of all patients. At the direction of our Congress, a coalition of patients and consumers, health professionals, pharmaceutical companies and others developed a plan for achieving the goals that had been adopted by our agency.
Today, I am happy to report, 67 percent of patients who have their prescription filled receive a drug information leaflet, printed on the spot just for them. We are confident that the goal of 75% by the year 2000 will be met.
We are also working closely with manufacturers to redesign the labeling for over-the-counter drugs. The manufacturers are cooperating with us in designing texts that are more legible, more readily understood, and have fairly uniform headings, subheadings, and type faces. The other recent step to improve drug information for patients is a proposed FDA guidance that allows radio and television advertisements with truthful and not misleading drug information, provided each ad also warns about the medication's main health risks, and describes how consumers can get full details about the advertised product.
Before closing, I want to add one more reason why the pictures I showed you have a special place in the annuals of our agency. FDA regulates the entire national food supply with the exception of poultry and meat, all drugs and biologics, all cosmetics, and all medical and radiological devices. Our workload adds up to one trillion dollars worth of products and represents one-quarter of all domestic consumer spending by more than 260 million Americans.
With fewer than 9,000 employees, and a budget of less than one billion dollars a year, we are under constant pressure to streamline our processes and increase our efficiency -- and we're doing all of that. But none of the scores of innovations, reforms and efficiencies we've introduced in recent years has changed our agency's traditional outlook and culture so thoroughly as our recognition that FDA cannot go on as the lone enforcer of public health laws -- that public health protection is a collective job that included regulators, the public and industry -- and not just at home, but all over the world.
This is a crucial insight which stems from our encounter with ACT UP demonstrators nine years ago. Its merit can be judged in part by our changed relations with the AIDS community. Over the last three years, as Congress debated legislative reforms for our agency, FDA had few supporters as ardent as the spokespersons of patients with AIDS. Together with the other patient and consumer groups, they argued strongly for maintaining the high levels of protection that FDA -- with their full participation -- continues to provide. They're very much involved in the regulatory process, which is where they and we believe they belong. For Keeps.