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The FDA and the Next Generation of Drug Development

Remarks at the Consensus Science Conference as Delivered by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Washington, DC
November 30, 2011

Thank you all—from industry, academia, and government—for joining us for this important discussion on “Consensus Science: New Tools and Tactics for Next-Gen Drug Development.”  Ultimately, this is a discussion aimed at helping translate new drugs from discovery to delivery…and into the hands of those who need them.

I also want to recognize the many scientists—250 who are with us today—who are working to establish common data standards for clinical research…and sharing their clinical trial data so that it can be pooled and made public.  Over 60 new biomarkers, PROs, and other new tools are now being evaluated.

Finally, I want to thank our conference co-sponsors, the Critical Path Institute and the Clinical Data Interchange Standards Consortium.  The Institute is a global leader in creating collaborations that advance scientific innovations that will improve health by opening new windows on prevention and new doors to treatment.  And the CDISC has done outstanding work in establishing standards to support the acquisition, exchange, submission, and archive of clinical research data and metadata.

You efforts—especially over the past five years—are paying off at a truly critical time.  You are producing the regulatory science we need to better evaluate new drugs. And your vital public-private partnerships are helping to enhance and accelerate the development of safe and effective drugs for everything from TB to drugs for orphan diseases.  Your work is important…its value has been proven…and it matters to both industry and science.
For all your organizations have accomplished, I am grateful…you colleagues at the FDA are grateful…and the American people are grateful.  Today I’d like to speak to you about two areas of concern for the FDA that are also essential for the creation of new tools and tactics for next-generation drug development.  They are strengthening regulatory science…and supporting innovation.  Both are extremely important to me, and this morning I’d like to share a little of my thinking on these topics.

All of us here understand the extraordinary importance of regulatory science.  A bench scientist may develop a new approach to a disease. A clinician may be able to show that it works. But regulatory scientists develop the knowledge and tools to translate discovery and innovation into hope—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—as a scientific community—we are not effectively translating these scientific discoveries into therapies, prevention, or cures.

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.

A big 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.

A robust field of regulatory science 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 a robust field of regulatory science would save significant dollars and many years by ensuring that we have the tools to detect unsafe or ineffective therapies at an early stage.

Additionally, 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.

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 in 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 strategic plan, Advancing Regulatory Science at the FDA, we’re devoting time and resources to help lead the effort to ensure that the necessary investments are made.

The plan’s core strategies are strengthening the science base at the FDA—including mission critical applied research; the scientific and professional development of FDA staff; and—perhaps most important for our discussions today— scientific collaboration.

We’re building partnerships—across government and with academia, industry, and the non-profit community—to help fill the critical gap between promising discoveries and approved products, while accelerating the development and reducing the costs of innovative products.

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’ve 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’ve 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.

The two pilot programs will support targeted research in FDA’s strategic priority areas and strengthen cutting edge training in areas important to drug, biologic, and medical device review—as well as scientific collaboration. The Centers are expected to enrich the scientific capabilities of the staffs of both FDA and the participating universities—and to support excellence in FDA’s innovation, product reviews, and science.

The Centers for Scientific Excellence approach builds on and supports FDA’s regulatory science strategic plan—as well as our Critical Path Initiative.

As many of you are aware, the Critical Path Initiative was a product of the extraordinary vision and leadership of FDA’s Dr. Janet Woodcock…and Dr. Vicki Seyfert-Margolis.  It’s created a nationwide network of scientists who search for ways to accelerate drug development, testing, and review.  And it’s helping the FDA to drive innovation in scientific processes through which medical products are developed, evaluated, and manufactured.

The Initiative was launched in March 2004, with the release of FDA's landmark report “Innovation/Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products.” The publication diagnosed the reasons for the widening gap between scientific discoveries that have unlocked the potential to prevent and cure some of today's biggest killers, such as diabetes, cancer, and Alzheimer's, and their translation into innovative medical treatments. 

The report concluded that collective action was needed to modernize scientific and technical tools as well as to harness information technology to evaluate and predict the safety, effectiveness, and manufacturability of medical products.

Collective action to advance the field of regulatory science—to give us the knowledge, tools and strategies we so need—is at the heart of energizing momentum for innovation 

Ultimately, though, 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.  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.

That brings me to my second are for discussion:  the FDA as catalyst for innovation.

Supporting innovation 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.

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 FDA 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…to create a rapid drug development pathway for targeted therapies…to harness the potential of data mining and information gathering…and to streamline FDA regulations.

I’d like to take a closer look at what we’re doing in just one of these areas, one which is of particular interest to all of us here:  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 outlined in our “Innovative Drug Report” which we released last month, 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.   All are compounds that have never been marketed before in the United States.

Sixteen of these new drugs were reviewed within six months. And we also evaluated nearly 70% of these 35 drugs for quality, safety and effectiveness – and approved their use for patients—before they were available in any other nation in the world.   But this is not a unique finding.  For example, an FDA review of drug approvals found that of fifty-seven novel drugs approved from 2006 to 2010 by both the FDA and the European Medicines Agency, three quarters were approved first in the United States.

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. 

In another example, in 2006 we approved a treatment for Pompe Disease—a rare genetic disorder that causes debilitating muscle weakness—on the basis of a single pivotal study of 18 patients.

Of course, as we continually strive to accelerate innovation and speed up development, we must also continually work to strengthen our ability to identify and resolve drug safety issues.  Innovation for innovation sake is not in anyone’s interest.   For patients and consumers, newer does not necessarily mean better—if drugs are not safe and effective.

Over the next two days, as you undertake discussion and dialogue on the areas I’ve just outlined: innovation and regulatory science…and as you explore innovative tools for drug development such as data standards, biomarkers, and clinical images…I hope that you also keep three challenges in mind. These are challenges that must be met if we want to further consensus science and spur the next generation of drug development.  And they are three challenges which will require your input and ideas.

First:  How to we better coordinate our efforts? 

Already, all of you are providing a wonderful example of how we can collaborate across organizational and other boundaries to develop new pathways for drug development.  These are the pathways that are also the bridge to getting new discoveries into action.  But what more do we need to do?  How can we continue to break down the boundaries and borders between our efforts?

Second:  How do we prioritize?  Which do we leverage?  This is especially important during difficult economic times when we must use resources wisely…and when all of us have unlimited needs, and limited resources.

And third:   How do we best engage the academic community?  We need their insights...we need their contribution to strengthen regulatory science…and we need their continued, and growing, partnership.

Meeting these challenges will not be easy.  But they are necessary if we want to further our common cause:   Generating innovative tools to develop efficacious medicines with optimal risk profiles.

How we meet these challenges—and how we further our work—was probably best stated by President Abraham Lincoln.

The history of the FDA actually begins with Lincoln, who created the Division of Chemistry that would evolve decades later into the Food and Drug Administration. 

Lincoln once advised that “…we must rise—with the occasion.  As our case is new, so we must think anew, and act anew.”  

I can think of no better advice for all of us here as we confront new challenges…but also new opportunities.   As we strive to generate innovative tools—and treatments. And as we engineer new, and better ways of doing things.

Thank you, and I wish you all a very successful conference.