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Margaret A. Hamburg, M.D., Commissioner of Food and Drugs - Remarks at the AAAS Forum on Science and Technology Policy

Remarks as Delivered for Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
at the 
AAAS Forum on Science and Technology Policy
May 14, 2010


Good afternoon and thank you so much Dr. [Alice] Huang for that generous introduction. It’s a pleasure to be here today at what I understand is the meeting on science and technology policy issues. I know you’ve all already had a busy day and a half, so thank you for being here now.

I have been looking forward to speaking at this meeting … a forum on science and technology policy. It gives me the opportunity to speak about an issue that I’ve become passionate about as FDA Commissioner … one that I view as the link between cutting-edge science and technology—some of which I know you’ve been hearing about over the past two days—and progress in the form of safe and effective new medicines and therapies for the American people.

That field is regulatory science—the science and tools we need to assess and evaluate a product’s safety, efficacy, quality and performance. We need it to develop new methods, standards and models we can use to speed the development, review and approval of medical products.

This afternoon, I would like to discuss with you the importance of regulatory science in our changing world and share some specific FDA policy initiatives to strengthen the field inside and outside the agency … and hopefully also convince you that regulatory science represents an essential and dynamic component of our scientific enterprise—though it is hard to compete with the much more sexy realm of discovery science.

As you all know, we live in a time when science and technology are changing our world in dramatic ways. We are seeing an explosion of knowledge and capabilities emerging from many domains of research and from around the globe. And with that comes a fundamental question of how do we make sure that we fully translate the potential and promise of that research into real-world products and programs that really matter.

I believe that a big part of the answer is by strengthening regulatory science. This is a component of the overall scientific enterprise that is really essential, and yet has been underappreciated and underdeveloped.

We know that the constant developments in science and technology—in fields as diverse as genomics and nanotechnology—hold the promise of major therapeutic advances. But, right now, we lack the ability to effectively translate many of those developments into vital products for those who need them. A gap—some call it “the valley of death”—has formed between biomedical research and the development of new medical products.

We need to close that gap. And we can. There are many factors, but one important aspect is to streamline and modernize the regulatory pathway.

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.

In other words, regulatory science is the critical bridge between biomedical research and new medical products. Americans cannot take full advantage of the breakneck speed of biomedical research unless we also emphasize innovation in regulatory science. Just as biomedical research has evolved over the past few decades, regulatory science must also evolve in important and powerful ways.

We must harness advances in science and technology to ensure that we have the most effective and efficient regulatory pathways to address the opportunities before us. And to do so, we must work together—academia, industry and government.

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

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

Let me give you a couple of examples.

Promising research is underway using stem cells to restore brain function lost in patients with Parkinson’s disease, and to treat various other medical conditions, as well. But before these treatments can reach patients, we must develop scientifically valid standards and manufacturing processes for stem cell therapies so they can be produced reliably and safely. Without these, the technology’s promise cannot be realized.

Basic research studies are identifying potential tumor markers that can indicate whether a patient’s cancer will respond to a specific therapy or combinations of therapies. But for these markers to be applied in clinical practice, ushering in an era of “personalized medicine,” the agency must use new science to guide the assessment of subpopulations of responders and non-responders and the evaluation and use of new diagnostic tests in that context.

NIH, industry, and foundations are working together on an artificial pancreas for juvenile diabetes—which would continuously monitor a patient’s blood sugar and automatically inject the right amount of insulin. But for patients to benefit, we must develop a scientifically solid testing path that ensures that the devices control blood sugar levels without risking hypoglycemia.

And we can defeat TB, a disease that has eluded us for centuries. NIH and others are supporting research into treatments for drug-resistant TB . But TB is complicated to treat. It requires multiple drugs over many months to cure … and if you partially treat, drug resistance develops.

Fighting TB effectively requires combination products. But it will take forever if we have to approve each new drug independently and then look at how they work in combination. But that is how the process would traditionally work. Instead we have embarked on a new initiative to work with researchers and companies interested in developing combination products which will be reviewed as such … much more complicated for FDA scientifically, but it is what patients need and what public health demands.

We want FDA to serve as a gateway—not a barrier—for the products that people need and count on everyday … A strong, robust field of regulatory science is essential to our work. It also means that we must strengthen overall scientific capacity within the FDA.

When I became Commissioner about a year ago, I was well aware of the pressing need to address issues of science at FDA. A number of reports and advisory boards had warned about the dire state of science funding at FDA and how the scientific and regulatory demands were dangerously outstripping FDA’s capacity to respond.

It seems obvious: the agency charged with judging the safety and efficacy of drugs and medical products—and monitoring the safety of those products as long as they are on the market—must possess a scientific capability equal to that task.

Our regulatory scientists must be able to understand therapies that are being developed using the most recent scientific advances. They must have the right tools to evaluate these therapies. And they must be a partner to the greater scientific community in their work to bring these therapies to patients.

Bu the sad truth is that for for many years, we have been poorly supported in these efforts. I am happy to report that we are making progress, with new attention and resources focused on strengthening scientific capabilities within FDA.

We are seeking to support and enhance our workforce, from clinical and scientific reviewers, to lab and expert manufacturing scientists and inspectors. I must say, that FDA does have an outstanding cadre of professionals and support staff. I am truly impressed by their quality and their dedication.

But we must do more to recruit and retain the best possible scientists, through quality training and work, meaningful career ladders, fellowship programs, scientific collaborations, exchanges and other professional development activities.

These programs and approaches will also help support the staff and scientific expertise needed for early and continuing scientific engagement by FDA with researchers and product developers, throughout the product development and evaluation process.

As we build our scientific capacities, it is essential that we work to identify where the greatest scientific gaps are, and augment our ability to perform in this rapidly evolving scientific environment.

But not only is more scientific knowledge required, but also a culture in which many scientific perspectives and opinions are sought and brought to bear on complex regulatory science problems.

No matter what, FDA must be a science based, science-driven agency in all we do.

Our success will depend on outreach and collaboration. We must be active participants in research and development through partnerships with academia, industry and other government agencies.

Toward this goal, we recently launched an important and broad collaboration in regulatory science with the National Institutes of Health—including the first-ever joint funding announcement for regulatory science.

We want to use this collaboration to propel discoveries in science from microscope to marketplace.

This model of inter-agency partnership will also be explored in other ways, such as with the National Institute of Standards and Technologies (NIST) around areas of standards development and qualification, and with the Defense Advanced Research Products Agency (DARPA) to help provide regulatory input on novel products under early development in their programs.

There are additional strategies to enhance collaboration as well. I am excited about the possibility that through a competitive application process—resources willing—FDA may be able to develop Centers of Excellence in Regulatory Science … which would support collaborative, targeted regulatory science research by academics and/or industry scientists to address research priorities defined jointly with the FDA.

These centers would be based at academic research institutions and could make a long-lasting difference, solving problems across entire product classes, enabling analyses of complex data and identifying new strategies for manufacturing of cell based therapies to name a few. And importantly, the collaborative nature of these Centers will provide new opportunities for learning and training for both scientists within and outside of the FDA.

And with our Critical Path Initiative, FDA will continue to partner with academic groups, patient advocacy groups, and industry to bring innovation to fields such as genomics, imaging and informatics so they can be applied to gaps in drug and diagnostic development.

Through these partnerships and the engagement of other outside advisory groups, such as our Science Board, we will gather information to help define regulatory science priority areas…and foster and support the work necessary to address them.

In our work, we must also look internationally. After all, science is a global enterprise. There are enormous opportunities to collaborate with scientific colleagues around the world on matters of regulatory science, and also to engage with international sister regulatory agencies to address issues of common concern, share information, and to harmonize standards and approaches.

Outreach and collaboration are central to regulatory science efforts. When successful, these collaborative efforts will help predict more swiftly and effectively which discoveries will succeed or fail as actual products, thereby reducing product development costs and getting better products to patients faster.

Rather than simply waiting at the end of the pipeline to approve or reject a product, FDA can help make trials more efficient by qualifying biomarkers that accurately predict outcomes and by encouraging innovative trial designs that are equally effective but less burdensome and time-consuming.

Rather than accepting that the only way to test for drug safety is to expose many thousands of patients to new products, FDA can help create innovative assays for safety that can better predict liver and kidney toxicity.

Rather than conducting its work in secret, FDA can become more transparent and share key issues publicly, so that insights can be shared and the field of drug discovery can move forward more quickly.

After all, these regulatory science challenges are not just for FDA alone. I urge you all to think about the science involved in the assessment of safety and effectiveness, and to engage academia in helping think through the next phase of scientific progress to support innovation in medicine.

In the coming days, FDA will be releasing a White Paper on regulatory science. The paper includes an overview of the field and its progress for patients, as well as ideas for harnessing regulatory science tools to protect our food supply, modernize toxicology and hazard assessment, regulate tobacco and for developing products for emerging infectious disease threats and bioterrorism. It also offers a regulatory science implementation framework for our agency.

The White Paper will be made available on the FDA’s website soon, so please look out for it.

I have no illusions about how difficult it will be too achieve all of our goals in regulatory science. These are difficult times—as a nation we face staggering budgets constraints, and an array of compelling yet competing priorities for attention and resources.

But regulatory science matters. Certainly, a robust, state-of-the-art regulatory science is essential to the work of the FDA. But more than that, it represents an important driver of our nation's health, the health of our health care industry, and the health of our economy … it’s vital to our nation’s global competitive. It is a field of endeavor that must be fully embraced by academia, industry and government.

I hope that you will help us in our efforts to advance the cause.

Thank you.