Michael A. Friedman, MD

Annual Speech to

The Food and Drug Law Institute

Washington, DC

Dec. 9, 1997

Good morning.

As many of you know, last February, the Secretary called central casting for another physician with a beard and glasses to act as commissioner. That's why I get to address you today. And I do so as much from a personal as from an institutional perspective.

In the past, this presentation has provided the venue to review and highlight FDA performance. That 1997 has been a perfectly splendid year of unprecedented achievement, and that the Agency remains vigorous, healthy and dynamic, is a testimony to the excellent FDA staff. I say this with neither arrogance or complacency -- but to rest on past achievement would invite such criticisms.

Looking forward, I want to use this time to share with you my vision of the challenges and opportunities that the Agency faces.

Let me tell you what this talk will not be: It will not be too long. It will not be highly statistical. It will not be a detailed look at FDA reform, nor the food safety or tobacco initiatives. These are weighty, rich, important subjects that deserve the fuller attention afforded them at separate sessions throughout this meeting.

I do intend to be constructively provocative, to challenge you to envision ways you can help FDA achieve still more as we move into the next century. Our citizenry, this audience and the Agency's personnel have so much vested in an optimally functioning FDA. Because of what you do and what you know, you have a special responsibility in this effort.

Today, I'd like to consider four sets of issues:

1. Our increased work load and current performance even an era of finite resources;

2. The rising expectations that all Americans have for the Food and Drug Administration fueled by our improved performance;

3. The revitalization and enhancement of science and scientific methodology and the need for a permanent chief science officer;

4. And lastly, information.

FDA not only regulates products but it also manages the flow of timely, accurate, useful information.

In the last few years, there has been a significant increase in our overall workload. (See Slide 2.) Just in applications for new drugs and biologics, the number has increased 50 percent since 1992.

The same trend is apparent throughout the agency. The quantity of regulated imports (See Slide 3.) has nearly doubled since the beginning of the decade. Yet the number of FDA inspectors has increased only slightly.

As medicines go off patent, the number of applications for generic products has skyrocketed. In fiscal year 97, (See Slide 4.) FDA approved a record 416 generic drugs.

Taken together all (See Slide 5.) the different types of product applications and submissions -- for drugs and biologics and devices and food colors and additives and veterinary therapeutics -- our workload has increased about 12 percent annually for the last several years.

At the same time, important, new public health assignments were added to the Agency's already prodigious list of responsibilities. While some of them came with additional resources, others did not. The first provisions of the tobacco regulation went into effect in February. The President launched his Food Safety Initiative early in the year. Just two months ago, President Clinton added still more' food safety obligations when he announced the initiative to make fresh fruits and vegetables safer, both those imported and those produced in this country.

Impressively, through improved management and sheer hard work, FDA has been able to perform at a record-setting pace.

As noted in the fifth report (See Slide 6.) to Congress under the Prescription Drug Users Fee Act of 1992, the agency's review of new drugs and biologics continued at the remarkable level of 96 percent on-time performance.

And, at the same time, (See Slide 7.) the median time to approval for drugs and biologics has been dropping steadily and will be at 12 months for the 96 cohort. And that's for everything: priority drugs and standard drugs.

Please remember that this is not an academic exercise in speeding up the review process. There are real patients waiting for these medications. We estimate that more than 11 million Americans received a newly marketed drug this year that would not have been available until 1998 without PDUFA. For many, these drugs provide significant -- sometimes life-saving -- benefits. And the financial cost for this acceleration in review times? Eight dollars per patient.

The PDUFA report contains abundant statistics documenting our continuing progress. No matter how performance is measured, FDA's accomplishments have been exceptional.

The Center for Devices and Radiological Health also had a noteworthy year, approving a record number of pre-market applications, eliminating backlogs and improving review times in all product categories.

Forty-eight PMAs were approved in FY 97, five more than the previous year. Nearly all were for new products and 18 of them represented important diagnostic or therapeutic advances. (See Slide 8.) The average time to approval decreased from 26 months in FY 96 to only 17 months in FY 97. At the same time, CDRH eliminated the backlog of PMAs and PMA supplements.

The center also made substantial improvement (See Slide 9.)in its 510(k) application review times, cutting almost in half the peak average review time of 194 days in FY 94.

I want to point out that the mere recitation of the CDRH's performance statistics, as impressive as they are, does not really do justice to the management accomplishments -- and without user fees. Our challenge, however, is how to maintain and continue to improve this program with limited resources in the years ahead.

Another example of what focused management can accomplish is provided by the Center for Food Safety and Applied Nutrition. For years, the center's staff has struggled -- with inadequate resources -- to deal with the volume of petitions for food and color additives. (See Slide 10.) In the late 1980s and early 1990s, the center acted on fewer applications than it received, creating a backlog. Attention was focused on the problem in the summer of 1995, and since then, CFSAN has made steady progress.

The number of pending petitions was 295 that year. Today, with minimal additional resources, they have cut the number of pending applications in half -- to 145 -- and the center continues to make decisions on more petitions than it receives. CFSAN has fashioned pragmatic solutions without compromising scientific rigor.

These important achievements are replicated in all of our centers, including veterinary medicine and biologics, as well as our field operations and special areas of interest such as orphan products, oncology, and women's health.

Just as an earlier slide (See Slide 11.)displayed the overall increased workload, this graph shows that the Agency's overall performance -- as measured by the number of products approved -- has risen an average of 17 percent year after year for the last several years for all the different kinds of products that we review.

The question that FDA faces--and something that should be of concern to everyone in this room--is how to sustain this performance with constrained resources. Can we achieve further management efficiencies without compromising FDA's traditionally high public health standards?

How can the Agency continue to manage a 12 percent annual average increase in the total number of all types of applications it receives and continue to produce performance gains of 17 percent a year (See Slide 12.) if FDA's budget grows at an annual rate of 1.3 percent in constant dollars?

Industry has come to appreciate the efficiency and predictability that FDA has demonstrated under the Prescription Drug User's Fee Act and understandably expects more. That is why industry so strongly supported reauthorization of PDUFA, including these advertisements (See Slide 13.)in The Washington Post.

I am pleased that PDUFA was reauthorized this year. It was a successful collaboration of the Agency, the industry and the Congress. This program was recognized just recently by the Ford Foundation, Harvard University's John F. Kennedy School of Government and the Council in Excellence in Government with the prestigious Innovations in American Government Award. This intensive effort demonstrates how much we can accomplish when we work together.

Given this impressive overall performance, it is obvious why the Congress, the Administration and the American people expect more and more from the Food and Drug Administration. Like industry, the public has become accustomed to the agency's superior level of performance; they expect that we will, even more effectively, promote and protect the public health.

Not surprisingly, the people who work at FDA, I have discovered, harbor the greatest expectations of all. To take the best, most accurate, most prompt, most appropriate actions. I have seen them push themselves -- and me -- to get the right answer in the shortest time. I applaud them for that.

Taken together, these expectations for speed, for accuracy, for safety of products increase the challenge, especially as our workload outstrips our resources. We certainly subscribe to the President's balanced budget program and recognize general government resource constraints. What's more, the pressures are not likely to be eased anytime soon because the steady rise in the number of new medical therapies is a direct consequence of a robust science environment.

Over the last several decades, federal funding has poured into biomedical research through the National Institutes of Health, (See Slide 14.)fostering the revolution in molecular biology and genetics as well as fundamental new understandings in cellular biology and technologies to manipulate them.

The result is an intellectual tsunami that threatens to overwhelm us.

And as the industry's R$D investment (See Slide 15.)has grown, the payoff has been a drug development pipeline full of promising new therapeutics, some of which are strikingly novel. Yet, we recognize that we cannot have available all conceivable scientific skills within the Agency. We must strategically plan for the future to accommodate these new insights.

How should we do this? First, we need to more effectively incorporate the evolving science into our regulatory framework. This year, scientists at FDA have begun a rigorous self-examination. An outside review by a panel of experts pointed to some of the ways that the agency needs to improve its current scientific skills. But it, and we, recognized that no single agency will ever have all the technical expertise required to evaluate every novel product pouring out of the nation's laboratories.

That means FDA must find new ways to leverage the expertise at our sister agencies, such as the National Institutes of Health and the Centers for Disease Control and Prevention. FDA recently signed an innovative agreement with the National Institute of Dental Research to work together to exchange insights and expertise and cross staff each other's advisory boards. I believe the agreement will foster better evaluation of products used in dentistry. It should also serve as a model for collaborations with other agencies and institutions.

FDA has created other types of collaborations, such as food safety policy development with the U.S. Department of Agriculture and the new, close ties between the Agency and the University of Maryland. It will be increasingly important to forge more links to academic and even industrial scientists.

If the agency is to advance, it also needs strong scientific leadership. Dr. Bern Schwetz is doing an excellent job as our interim science officer, but he will want to return to his family and his own research in due time.

To replace him, we need an outstanding chief scientist, a nationally recognized, distinguished, capable leader who can help lead the agency into the next century. Since it is in the industry's interest, and in the interest of the general public, for FDA to remain on the scientific cutting edge, you could help us identify superior candidates.

I have a second suggestion I would like you to consider. If we are to be prepared to deal with the novel and innovative products, we need to know what's coming. One possible solution would be for companies to confidentially share with us their long-range research plan. That way, the Agency could develop access to the right science base and be ready when products come in for market approval. Several CEOs I have approached have been enthusiastic, believing as I do, that this kind of cooperation should improve the quality and efficiency of the total development process.

As we surf the scientific cutting edge, FDA must continue evolving. We will always be a chimeric scientific regulatory agency. And our success in this mission depends on high-grade information.

But we must also share our information. Although we currently provide large amounts of useful data to the public, we will have to find more and better ways to get facts to the physicians, patients and consumers making complex choices about the therapies they need and the products they buy.

Important steps have already begun. The Medguide program, for example, will provide patients with useful details every time they fill a prescription.

For over-the-counter drugs, labels will carry more complete information. Useful information. That is the goal.

But we face formidable challenges. For one thing, scientific information evolves as new discoveries are made. We develop important new insights into products long approved ñ or in some cases even previously rejected.

How ironic that we declared that phenolphthalein, the primary ingredient in some popular laxatives, needed to be remove from the market at nearly the same time that we issued an approvable letter indicating thalidomide was safe and effective enough for careful use in limited situations.

The agency assumes an ongoing obligation for every product it approves and every decision that we make. Clinical studies can never include sufficient volunteers or long enough follow-up to detect all possible problems, so if an adverse effect arises after FDA approval, the Agency must remain vigilant and act when the data warrant. That's what led, for example, to the call for the removal of the diet drugs fenfluramine and dexfenfluramine because of cardiac valvulopathy. As more products are approved, more interactions are possible, making FDA's post-marketing scrutiny increasingly important.

In addition, the agency is rapidly advancing into the world of electronic information management. This transition places a great burden on our review centers but it also offers tremendous efficiencies. Whether it is total electronic filing or FOI or adverse events reporting or clinical trial databases, the new approaches to managing information will change how we do business in ways we cannot yet fully imagine. We all believe that our performance will be better, probably faster, but certainly the startup will stretch our resources to the limits.

Throughout this talk, I have described our achievements of the last year and laid out some of the issues I see ahead. I have purposely avoided specific solutions, though I certainly have ideas about how to approach these problems. Instead of laying out my conclusions, however, I want to challenge you to think about this agency and its future Ö our collective future.

The public has a vested interest in FDA's health and vitality, and has a role in helping prioritize which of the Agency's diverse and competing missions should get funding and which can be reduced or deferred.

I have talked about change, about process improvement, innovation, pragmatism, collaboration and scientific/administrative enhancements. But do not misunderstand me. While FDA is evolving, our commitment to traditional public health values is unchanged. Our standards, our passionate dedication is immutable. Many things will change in the future, but our abiding commitment to promoting and protecting the public health will not.

Thank you.