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HIV/AIDS and the FDA: Learning from Past, Looking to Future

Remarks as Prepared for Delivery of Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
amfAR Conference – Making AIDS History: Ending the Epidemic
July 26, 2011

Good afternoon—and thank you so much for inviting me to be part of this special day. It’s really a pleasure to be here among so many members of the HIV/AIDS community: the activists, scientists, policymakers, and patients who have spent the past three decades building a movement that saved millions of lives—and changed the world. Despite all the work we have ahead of us, and the real crisis that still exists in many parts of the world, we should be proud of how far we have come.

Today’s conference is an appropriate recognition of the extraordinary progress made…but also an important call to action that the most profound challenge still remains before us: to end this devastating epidemic. I am humbled and honored to be here…and astounded that I am on the same agenda as Whoopi Goldberg!

I work on a very broad range of issues these days, but, like so many of the people you’ve heard from today, this was the disease that redefined my career and committed me to public service. I was just a medical student during the emergence of HIV/AIDS. I remember seeing these strange cases of immune deficiency, mainly in previously healthy young men; nobody knew then what the cause was or what to call it. But by the time I did my medical residency in New York City, I was taking care of lots of AIDS patients. But there was so little we could offer. Patients were wasting away before our eyes, but there were no drugs available…only supportive care. It was a difficult time.

Soon after, I went to work for Dr. Tony Fauci at the National Institute of Allergy and Infectious Diseases. At that point in the AIDS epidemic—back in the late 1980s—candidate drugs were just emerging. The great push was getting as many people as possible into clinical trials, so they’d have at least some chance for treatment. Some questioned why these clinical trials had to happen at all. They reasoned that patients facing death should have the chance to take anything that might bring them a benefit. Not surprisingly, it turned out that some of those medications worked and others did not, and, since then, knowing which medications are effective has extended the lives of millions of patients in the United States and around the world.

This was also the time when I first started paying attention to drug development…and to the FDA. NIAID, of course, was very involved on the basic research side and in the design and implementation of clinical trials. But we worked closely with FDA staff and leadership on questions of science and policy…though I can tell you that I certainly never imagined then that I would end up as FDA Commissioner today.

Back then, for me, it was about being part of the effort to find treatments—and someday, we hoped, a cure—for HIV/AIDS. And what was so inspiring—and ultimately so effective—was how all the various stakeholders come together…warily at first, but ultimately with a shared sense of both respect and purpose. Since then, we’ve seen extraordinary advances—and there is so much new and exciting science ahead of us.

Today we have 33 HIV/AIDS drugs in seven different classes that are developed and distributed by the 10 largest pharmaceutical companies in the world. After decades of research, we have proof of concept for next-generation ARV-based microbicides. We have new strategies for prevention. We have more hope than ever that we might find the path to a cure. And we have seen incredible examples of systemic change: patient-driven clinical trials, a more collaborative regulatory paradigm for drug approval, innovative models for HIV education, a new brand of advocacy and political investment—the list could go on.

We’ve come a long way. And the reason we were able to is actually pretty simple: we worked together.

Since the earliest days of the epidemic, for victories large and small, one thing remained constant: the need to bring key stakeholders, including patients and patient advocates, together around the table to define critical unmet needs, delineate the research agenda, brainstorm innovative approaches, address issues of how to balance risk and benefit—and inspire each other to action… and a sense of urgency.

The approval of the first antiretrovirals, after all, could not have happened without early coordination among researchers, companies, advocates—and the FDA. And so many people with HIV/AIDS are alive today because of those collaborations and advances. As we move forward, the issues and needs will continue to evolve. But our commitment to working together—to strengthening this incredible community—must remain constant.

Because, as you know, we have so much left to do, in this country and around the world. We still need a vaccine. We need better preventive strategies. We need Phase II trials and beyond for the new microbicides. We need to deepen our knowledge of the pathogenesis of disease and the human response. We need continuing education and awareness, so that we can bring down rates of new infection. We need to better understand and address the impact of prolonged treatment—such as antibiotic resistance and adverse side effects. And, of course, we need a cure.

My agency, the FDA, plays a huge role in making a lot of this happen—but we are only one piece of a large medical product ecosystem that includes researchers, government, small businesses, large biotechnology and pharmaceutical companies, insurance providers or payers, physicians, and most importantly patients and the American public. It is up to all of us now to learn from the lessons of the past and cast a vision for the future.

And from my perspective as the head of the FDA—this unique and essential, though not always beloved, public health agency—this means not just capitalizing on advances in science and addressing current and unmet public health needs, but making fundamental investments in the field of regulatory science…which more and more people have begun to see as critical for progress for patients.

The fact is we are spending billions and billions as a nation on scientific research that could, if harnessed, transform public health. But we are not even close to fully translating new discoveries and emerging opportunities into products for people who need them. Which is why we need regulatory science. For us, regulatory science is the science of developing the knowledge tools, standards and approaches needed to assess the safety, efficacy, quality, and performance of  products we regulate. It involves the development of new methods, standards, and models to modernize and streamline the review, approval, and ongoing oversight of medical products.

In other words, the field is an essential component of the scientific enterprise. But it has, for years, been under-appreciated, under-developed, and under-funded. And so we are left relying on 20th century approaches for the development, review, approval, and oversight of the treatments and cures of the 21st century. Without advances in regulatory science, promising therapies may be discarded during development or because we lack the tools to recognize their potential and because outdated, inefficient review methods unnecessarily delay the approval of critical treatments.

A strong field of regulatory science can help us use our knowledge of biological pathways and gene variants to design better, more effective therapies and help eliminate ineffective drug candidates earlier in the pipeline.

It will foster the use of advanced genetic data and biomarkers to find faster paths to disease targets, and support efforts to use more innovative and adaptive clinical trial designs; designs that enable us to get robust, scientific answers, but in a more timely and cost-effective way, as well as to potentially identify subpopulations of responders to different therapies.

And it can enable us to find better ways of mining and applying knowledge gleaned from the vast quantities of data at the FDA and other agencies around the world to better inform us about safety, efficacy, differential subpopulation effects and much more.

And at the most fundamental level, it will help place the emerging, promising areas of science and technology fully at the service of people—now and in the future.

The bottom line is that we need new and better tools to do this—and we need them now. But this will not be possible if we as a nation do not invest—both resources and manpower—in the field of regulatory science. It was early investments like this, after all, that enabled FDA to say that surrogate markers could be uses in clinical trials in the first place—and led to the approval of some of the earliest ARVs.

But surprisingly, we have not developed and extended these kinds of opportunities in many critical ways. Which is why I have made regulatory science a top priority since I became Commissioner. In addition to strengthening the science base at the FDA, we are building the partnerships—across government and with academia, industry, and the non-profit community—that will drive the development of innovative medical products for HIV and countless other diseases and conditions.

One concrete example of why this matters: A scientific priority for FDA is the review and approval of combination products—which is critical for the development of new AIDS therapies. Historically, approving combinations of drugs has taken many years: FDA had to first approve each drug individually before we could even test them together.

But a few months ago—in an effort to adapt the review process to the unique importance and characteristics of these therapies—we put out a new guidance encouraging companies to test investigational drugs in combination when doing so can offer a significant advantage to patients. This scenario has unique relevance to targeted therapies for HIV, which may need to be combined to enhance effectiveness and/or prevent resistance, as well as for problems like TB, which remains an enormous disease threat around the world with disproportionate burden for people living with HIV/AIDS.

This is a small step, but it is part of an effort that will change the landscape of the drug development process. It is much more challenging scientifically for the FDA, and some studies will involve products initially developed by different companies, but it is what patients need and what public health demands. And it is clearly moving us into the future—into the age of smart, targeted therapies that build on advances in science and that offer the promise of major clinical benefit with more specificity and far less toxicity.

I also want to note before I wrap up that FDA also plays a significant role internationally, specifically by working with PEPFAR. A tentative approval program enabled us to approve more than 100 life-saving anti-retroviral drugs, which have been made available to more than 2 million people throughout Sub-Saharan Africa, Asia, and the Caribbean under PEPFAR. Again, there is so much more to do here, but progress we can be proud of.

It is important for us to recognize that this is a dynamic movement—a continuing process—and that we must keep building on the scientific and advocacy leads that have been cultivated over the past three decades. And of course we must forever remember that this movement, though not a movement of choice, has been one of passion—and one of community. It was the result of a unique moment in which people from many different communities came together in crisis to form a new one—a stronger one…capable of achieving what seemed then like the impossible.

I’m going to leave you words from my former boss—a man who has grown into one of the most powerful AIDS activists there is: President Bill Clinton. "The leaders here assembled can rewrite human history in the new millennium,” he said. “But we must believe the simple things — that everywhere in every land, people in every station matter. Everyone counts, everyone has a role to play, and we all do better when we help each other." Indeed, together, we can continue to rewrite the history of this disease forever.

Thank you.