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The FDA at the Crossroads

Remarks at the Consensus Science Conference as Delivered by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Washington, D.C.
November 15, 2011

Thank you Dr. Goldman and Dr. Rest for that warm and gracious introduction.  And thank you to the Union of Concerned Scientists and George Washington University for hosting this important forum. I wish that I could stay for all of the sessions today, but I will be very interested to hear more about the rest of the day’s panels and discussions.

As the theme of your conference acknowledges, this is a critical moment for the Food and Drug Administration. In many ways we are at the crossroads…and decisions made now will matter in fundamental ways and for a very long time.

As a science-based, regulatory agency with a public health mission, the FDA has a very special role in addressing threats to health and as the gateway for products that people need. I think most would agree that we serve a unique and essential government function. Certainly many of our mandates and responsibilities are ours alone. If we can’t do our job and do it well, there is no one to backstop behind us.

This is a challenging time—a time when many different, often competing, factors affect the way we do our jobs and the opportunities before us. Over the past several years, we have dealt with expanding responsibilities, a tightening economy and the pressures of globalization.

This is also a time when science and technology are changing our world in dramatic ways, with an explosion of knowledge and capabilities emerging from many domains of research.

Since I became Commissioner two-and-a-half years ago, we have grown, evolved, renewed, and refined our mission and activities in important and powerful ways.

We took on new responsibilities and new authorities to support our public health mission, most recently with the passage of the Food Safety Modernization Act, as well as the implementation of the historic new tobacco control legislation.

We mobilized and partnered with agencies and organizations to address emerging crises like the earthquake and tsunami in Japan, the Gulf oil spill and pandemic flu.  We have also confronted major outbreaks of foodborne illness.

And we have worked hard to strengthen the science base at the FDA while advancing the field of regulatory science. At the same time we have also worked to extend our reach in new ways around the world as oversight of FDA regulated products has become a global endeavor.

As I have come to appreciate—more and more each day—our agency is charged with an enormous and significant task: to promote and protect the health of the American people, and, increasingly, people all over the world. This includes efforts to ensure the safety, effectiveness, and quality of drugs, vaccines, medical devices, as well as the safety and wholesomeness of the vast majority of our nation’s food supply, and more—all-in-all products that account for between 20 and 25 percent of every consumer dollar spent on products in the U.S….. and products that the American people rely on in fundamental, sometimes lifesaving, ways.

And it also includes working proactively to foster the scientific innovation that will lead to tomorrow’s new breakthrough products. Both roles are essential to delivering progress for the American people—making sure that consumers and patients have the products that need and count on. And both roles affect not just human health and well-being, but also the health of our economy by encouraging consumer confidence, growing key industries, and creating jobs.

It has been said that, every day, we are in every American home, at every table and in every medicine chest. But it is more than that. We are in every hospital, pharmacy, grocery store, doctor’s office, school cafeteria, restaurant, and every distribution chain along the way from all across the globe. 

Which means, as you can imagine, there are many issues I could talk about today—and dozens of specific programs and initiatives that have helped us reshape and reposition the FDA for the 21st century. But for today, I’m going to focus on the bigger picture—on some key themes and priorities that drive all of our work and keep us focused amid competing priorities: the importance of sound science and smart regulation in all that we do, our role as a catalyst for innovation, and the challenges of our globalized economy and the oversight of products with global pedigrees.

But important to all of those themes is even something bigger and that is the integrity of the FDA. Building and maintaining trust and confidence in the FDA is the linchpin of everything that we do. I believe that, fundamentally, the American people understand that we all benefit when we have a strong, fully functional FDA.

History has shown that both consumers and industry have a tremendous stake in an agency that takes science-based action on behalf of public health…an agency that not only takes action to keep Americans safe and healthy—but also promotes the science, and standards, that keep the engines of innovation running.

With this power, of course, comes great responsibility. And we have a responsibility to take action that is transparent and can inspire trust in the public whose health we are trying to promote and protect.

Unfortunately, public confidence in my agency has taken hits in the last decade. And one thing that I learned early on is that when people do not understand FDA or its actions, they often assume the worst. Only by really explaining what we do, and why, can we foster that trust, even in times of conflict or controversy.

So I have been working with my colleagues to promote a culture of even greater openness at the FDA. We are creating an agency that takes its own leadership role seriously while also trying to listen to all of our relevant stakeholders.

And we have taken a number of important steps in this regard. Soon after I arrived at FDA, we launched our Transparency Initiative—an agency-wide effort to make useful, understandable information about the FDA more readily available to our stakeholders. We wanted to overcome the perception that our agency is—as it has often been called—a “black box” where decision making takes place behind closed doors and out of the public eye. And I believe we are on our way there.

But earning and maintaining the trust and confidence of the American people is about more than any one initiative. It’s about recognizing the importance of strong, smart regulation—and real integrity in the science.

We owe it to the public to be transparent and to articulate reasons for making decisions—but we also owe it to them to make smart, data-driven, scientifically-based decisions that benefit patients and consumers.

This is extremely important to industry, as well. We often hear from our colleagues in industry about regulatory uncertainty—and a lack of clarity in the process for getting medical products approved by the FDA. We are working hard to clarify and modernize our regulatory pathways, making them more transparent, consistent and predictable. But the truth is that a lot of so-called regulatory uncertainty that is, in fact, grounded in scientific uncertainty.

And this is largely because advances in science are changing the product-development landscape. Major breakthroughs have brought us new types of products and new opportunities for progress—but they have also posed new challenges for industry and the FDA as we seek to fully capture the promise of this new science.

Which brings me to one of the main points that I want to underscore:  the importance of sound science and smart regulation.

There are many reasons for why we are grappling with this scientific uncertainty, including increasingly complex medical products and an incomplete understanding of biological systems, disease pathogenesis and human biology.

But in my view, it also stems from a lack of attention and investment in regulatory science—the science and tools needed to assess and evaluate a product’s safety, efficacy, quality and performance. It involves the development of new methods, standards and models we can use to speed the development, review, approval and ongoing oversight of medical products.

A bench scientist may develop a new approach to a disease. A clinician may be able to show it can work. But regulatory scientists must help develop the knowledge and tools to translate discovery and innovation, into those products that hold so much promise.

We are at an incredible moment for discovery—and as we look ahead at the scientific landscape, there are so many areas that hold tremendous promise for progress, including genomics, synthetic biology, systems biology, advanced therapies like stem cells, and emerging technologies, like nanotechnology.
But we all recognize that we—as a scientific community—are not effectively translating these scientific discoveries into therapies, prevention, or cures, or fully applying our knowledge to better support the safety of food and medical products—or the health of the public.

Despite unprecedented spending on basic research and development by government as well as the biotechnology and pharmaceutical industries—to the tune of more than $80 billion this past year—the pipeline of new drugs is disturbingly dry.

There are numerous reasons for this--scientific, economic, and regulatory—but among other factors, there must be a shift in how we think about drug candidates and discover new ones…and how we evaluate emerging therapies when it comes to their benefit/risk profile.

Part of the solution is having the right regulatory and product development tools—which is why regulatory science is such an essential component of the scientific enterprise.

A robust field of regulatory science would enable us to use our knowledge of biological pathways and gene variants to help identify promising new drug candidates and new potential targets for treatment.

It would help prevent promising therapies from being discarded during development because we lack the tools to recognize their potential or because outdated review methods delay their access.

And it would save significant dollars and many years by ensuring that we have the tools to detect unsafe or ineffective therapies at an early stage.

Unlike work performed by specific sponsors for a specific product, regulatory science is important for multiple products and stakeholders. The knowledge generated from such studies informs a whole body of innovation—and entire classes of drugs—rather than single products.

I should emphasize that regulatory science comprises an array of disciplines and approaches. It takes place in laboratories, but it also involves clinical, epidemiologic, and statistical tools and information-gathering systems.

With more advanced regulatory science, we could usher in an era of personalized medicine, by linking advanced genetic data and biomarkers with targeted therapies.

We could make significant strides the science of safety, including predictive toxicology.

We could develop and optimize innovative clinical trial designs and analytics that facilitate the possibilities of targeted therapy and that require smaller patient populations, shorter timeframes and lower costs.
And we could find better ways of mining and applying the information and knowledge that resides in the vast quantities of data housed at the FDA and other agencies around the world.

For all of these reasons, I have made advancing regulatory science on behalf of the public health a top priority since I became Commissioner. Through our Advancing Regulatory Science Initiative, FDA is devoting time and resources to help lead the effort to ensure these investments are made.

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 and the delivery of better, safer products to the American people and to people around the world.

For example, last year, the FDA and the National Institutes of Health launched a new NIH-FDA Council and a Regulatory Science Initiative to encourage research in this field. We have already awarded our first set of grants in regulatory science and hope to do the next round soon. 

Although dollars have been tighter than hoped, we have also begun to establish Centers for Excellence in Regulatory Science, focused on important research in the field, conducted in a multidisciplinary and cross-sectoral manner. 

In August, we established a Center in Arkansas focused on nanotechnology, involving FDA’s National Center for Toxicology Research, several academic institutions in the state, and the Governor’s office. And last month we announced two new regional Centers for regulatory science and innovation, one at Georgetown University, and the other at the University of Maryland.

Advancing this field of regulatory science, to give us the knowledge, tools and strategies we so need, and in turn reducing scientific uncertainty is, I believe, at the heart of energizing momentum for innovation in medical product development.

And that brings me to the second topic issue facing FDA at this juncture:  our role as a catalyst for innovation.

As I’ve suggested, we’re dedicated to protecting and promoting the health of the American people through scientifically sound, smart regulation.  But, as part of our public health mission, we also must promote the innovation it takes to ensure that Americans have timely access to life-enhancing and life-saving products.

Supporting innovation—helping to open new windows on prevention and new doors to treatment—is profoundly personal for me. When I was a first year medical student, I remember being told that in the era of modern medicine, infectious diseases would soon be relegated to the history books.

Then, as my medical school training unfolded, we watched the emergence of a mysterious new illness:  a devastating and unfamiliar condition that came to be known as HIV/AIDS.  Initially no one knew what caused it or what to call it. A few years later, we knew what it was, but for those who contracted HIV, we had no effective treatments…no medicine or miracle on the horizon.

As a physician, I’ll never forget how impotent and ineffective I felt—powerless to combat the virus…or extend life…or even do much to ease suffering.    Thankfully, today AIDS is no longer a death sentence, but a chronic, yet manageable, disease.  That development is a testament to the power of innovation… and determination.

We know that if we want to continue to help foster the kind of innovation that we saw with the AIDS epidemic, we must continue to find new and better ways of doing things—while always maintaining a gold standard of safety and effectiveness.

Just last month we outlined some of our plans and progress in a new report:  “Driving Biomedical Innovation:  Initiatives for improving Products for Patients.  Building on a series of discussions with stakeholders, the report outlines how FDA is focused on implementing major reforms in a number of areas.  Among our initiatives, we’re working to build the infrastructure to support personalized medicine; create a rapid drug development pathway for targeted therapies; harness the potential of data mining and information gathering; and streamline FDA regulations.

I’d like to take a closer look at what we’re doing in just one of these areas:  Developing tools and strategies to accelerate drug review and approval, while consistently ensuring drug safety and effectiveness. 

Our expedited drug approval pathways now include Fast Track…Accelerated Approval…Priority Review…and Expanded Access programs.  All are designed to speed the testing, availability, and approval of drugs in different ways—while never neglecting safety—and they’ve made a real difference. 

For example, as I mentioned earlier, in Fiscal Year 2011 we approved 35 novel medicines.  These new drugs are targeting diseases such as late-stage lung cancer, metastatic breast cancer and hepatitis C.  They also include the first new drug to treat lupus in 50 years…the first to treat Hodgkin’s lymphoma in 30 years…and the very first drug ever shown to be effective in extending the lives of patients with metastatic melanoma.  

Almost half of these 35 new drugs were approved under “priority review”—where the FDA has a six month goal to complete its review for safety and effectiveness. We also evaluated nearly 70% of these 35 drugs for quality, safety and effectiveness—and approved their use in patients—before they were available in any other nation in the world.

We’ve also demonstrated considerable regulatory flexibility.  In reviewing and approving the 35 new drugs mentioned in the report, we streamlined clinical requirements to permit smaller, shorter or fewer studies wherever possible. 

Ultimately, harnessing science on behalf of the public health is about more than adequate tools. It’s about applying a new way of thinking about how we do research and development from the get-go—and not just having good ideas about emerging, exciting areas of science, but finding truly innovative ways of making them useful, applicable, and understood in terms of benefits and risks for patients and consumers.

Before closing, I want to touch on one more issue—and that is meeting the unique, and complex, demands of globalization. Today, what were once considered domestic products are actually global commodities.

Since 2000, the imports of FDA-regulated products have more than quadrupled. …and the supply chain from manufacturer to consumer has become more and more complex … involving a web of re-packagers and redistributors… and making oversight significantly more difficult;

The numbers are staggering.  In the food realm, about 35-40 percent of fresh fruit and produce and nearly 80 percent of seafood come from other countries. And for medical products, a stunning 80 percent of active pharmaceutical ingredients in drugs consumed in the U.S. come from outside our borders and approximately 40 percent of the drugs available in this country are actually manufactured abroad.

Lapses in global supply chain safety—deliberate and unintended—can lead to the distribution of unsafe or ineffective products.

Unfortunately, we have already seen what happens with the system fails. Contaminated heparin and counterfeit glucose monitor strips are two examples of many that have carried serious implications for health and safety.

The realities of our transforming, 21st century world requires us to think and act in new ways. That’s why the FDA is engaging –bilaterally and multilaterally—with countries and companies throughout the world.  We’re sharing scientific and technical expertise.  We’re working with our counterparts in other countries to harmonize international standards for safe food, drugs and medical products.  And we’re helping countries with less mature economies and regulatory systems build capacity so that they can produce food and commodities that meet international food safety standards.

Additionally, we’ve established a permanent presence—in places such as China, India and Latin America—that produce a high volume of regulated goods for our market. In addition, we have implemented more sophisticated strategies for intercepting problems at the border and we are increasing the number of foreign inspections.

This new global outlook was outlined in FDA’s new strategy, “Pathway to Global Product Safety and Quality”  that we issued last June. It outlines four major pillars of activity.

In close partnership with our foreign counterparts, FDA will assemble global coalitions of regulators dedicated to building and strengthening the product safety net around the world.  With these coalitions, we intend to develop global data information systems and networks in which regulators, worldwide, can regularly and proactively share real-time information and resources across markets. 

We’ll also continue to expand our capabilities in intelligence gathering and use—with an increased focus on risk analytics and thoroughly modernized IT capabilities. And we’ll effectively allocate agency resources based on risk.

I know I’ve covered considerable ground with you today.  I’ve discussed these topics at some length because I know of your keen interest in FDA’s mission and the role of science in supporting that mission.

At this pivotal moment, you are invested in these issues not only as dedicated scientists but as engaged citizens of the world. 

Let me close by simply saying that your organization and your members can play a significant role in assuring continued progress in all of these fronts:  advancing regulatory science, fostering innovation, working in a globalized environment.

And—most important—will help ensure that we continue to fulfill our mission, our promise, to protect and promote public health in America.

Thank you for your time, and I wish you all a successful and productive conference.