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Margaret A. Hamburg, M.D, Commissioner of Food and Drugs - Remarks at the National Press Club Speaker Luncheon

Remarks as Delivered of Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
National Press Club Speaker Luncheon
October 6, 2010

Thank you very much. It's a pleasure to be here. I enjoy looking out and seeing some friends and colleagues in the audience, and, of course, it's wonderful to have my parents with me, as well.

I have to confess that when I first agreed to come and give a talk at the Press Club today, I had a very clear idea of what I was going to talk about—and it was going to be about the passage of the new food safety legislation. However, that food safety legislation is still being debated in the halls of Congress. And so instead I decided to talk about another subject close to my heart. And that is regulatory science, which is an issue that more and more people are recognizing as critical to progress for patients.

We live in a time of huge opportunities to improve health—to translate breakthrough discoveries and innovation into benefits for people, enabling us to better diagnose, treat, and hopefully prevent and cure disease.

We are more poised than ever before to deliver the promise of science in the service of patients. But to do so, in my view, will require the advancement and more effective application of regulatory science—the discipline at the very heart of our mission at FDA.

If our efforts are to be successful, we will need the full engagement of the scientific community, and beyond. This must be a top priority for us at the FDA and for our nation.

This is something that I have become passionate about since becoming FDA Commissioner, so this afternoon I would like to offer some thoughts on this important issue…and share what FDA can and will do going forward to better serve the public’s health and realize the promise of biomedical research.

I am also pleased to announce that today we are releasing a White Paper entitled “Advancing Regulatory Science for Public Health: A Framework for FDA’s Regulatory Science Initiative.” This document provides a broad blueprint for progress in the field as well as our agency’s role in promoting that progress.

Let me begin by making sure we are all on the same page about what is meant by “regulatory science.” I am referring to the science and tools needed to assess and evaluate a product’s safety, efficacy, quality and performance.

Regulatory science involves the development of new methods, standards and models we can use to speed the development, review, approval and ongoing oversight of medical products.

Science underlies the very foundation of the Food and Drug Administration. And even today, a century after Teddy Roosevelt signed the Pure Food and Drug Act that led to the creation of our agency, our goal at the FDA remains to embrace our origins as a science-based agency that relies on data-driven decision making to promote and protect the health of the public.

But today, we sit at a critical juncture. 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. But do any of us believe that we have adequately delivered on the opportunities of science?

Though science and technology have progressed rapidly, we have not seen equivalent progress in treatments and solutions for many of the diseases and conditions that afflict our friends, neighbors, and families.

For example, we have been fighting a war on cancer since it was so declared in the National Cancer Act of 1971. Forty years later, many cancers are still difficult to treat and some are incurable. And if you are diagnosed with cancer today—especially one of the more complex, elusive forms—the standard, best-practice treatments you receive from your doctor will likely include toxic drugs that were approved for treatment more than 20 years ago.

In another realm, there is increasing alarm about the problem of antibiotic resistance. And we worry with good cause: today, antibiotic resistance mechanisms have been reported for virtually all known antibacterial drugs currently available for clinical use—which affects everything from global infectious diseases, to ear infections in school children, to staph infections in locker rooms.

People actually talk these days about a potential return to the “pre-antibiotic era” where we no longer have effective tools to treat serious infectious disease. Clearly we must encourage more judicious use of these important drugs through improved infection control, rational prescribing and better patient compliance. But even if we improve these practices, resistant bacteria will continue to develop.

No matter what, we need new and better drugs…and we need them now. Yet the R & D pipeline is distressingly low. The number of newly approved antibiotics—not just new formulations of previously existing drugs—has fallen steadily since the 1980s. And the range of new antibiotics is disturbingly limited in terms of the types or classes of antibiotics available and the diseases they can treat.

Overall, the reality is that billions of dollars have been invested in biomedical research—in 2008, more than $80 billion by the private sector and more than $30 billion in the NIH—an effort that is clearly imperative for medical progress.

And we have witnessed the resultant discoveries that hold the promise of major therapeutic advances—in fields as diverse as genomics, synthetic biology, stem cells and nanotechnology.

But, right now, we lack the ability to effectively translate many of these developments into vital products for those who need them. The number of new therapies is actually in the decline while the costs of bringing them to market have soared.

To put it simply, there is a troubling gap between advances in science and patient care. We need to build a bridge across this gap… and that bridge, in my view, is regulatory science.

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 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.

Regulatory science is an essential part of the overall scientific enterprise, yet it has been under-appreciated and under-funded. Because of this, we have been unable to apply the best possible science and technology to the tasks before us. And we are left relying on 20th century approaches for the review, approval and oversight of the treatments and cures of the 21st century.

Now is the time to move forward. A robust field of regulatory science will allow us to effectively and efficiently translate many of those breakthroughs into therapies and cures.

It can enable us to use our knowledge of biological pathways and gene variants to 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.

It will support efforts to optimize clinical trial design to speed the regulatory process and get products to patients faster.

Without advances in regulatory science, promising therapies may be discarded during development because we lack the tools to recognize their potential and because outdated, inefficient review methods unnecessarily delay the approval of critical treatments. On the other hand, both significant dollars and many years may be wasted assessing a novel therapy that with better tools might be shown to be unsafe or ineffective at an earlier stage.

I should emphasize that regulatory science comprises an array of disciplines and approaches. It takes place in laboratories, but it also involves clinical and epidemiologic research, bioimaging, 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.

It will take new investments, approaches, and partnerships for regulatory science to evolve the way that biomedical and life sciences research has in recent times. But we must pursue it.

For example, some 2.7 billion dollars and over a decade of time was spent sequencing the human genome which was successfully completed 10 years ago…and yet fewer than 50 therapies actually have genetic tests as part of their labeling for guiding their use. That's why we need regulatory science.

Even though we know that there are promising cell based therapies for treating a variety of now incurable diseases, we still don't know how to define the right number of cells to infuse or what happens to these cells after they are placed in the body. That is why we need regulatory science.

We don't have good models for predicting toxicity in humans, which leads to lots of time and money being spent by biotech and pharmaceutical companies each year developing new therapies with a failure rate of 90 percent. That is why we need regulatory science.

We increasingly call on the safety-focused science of pharmacovigilance to use data sets and reported information to monitor the post-market environment for safety signals and risk concerns. But we still need meaningful strategies to quickly evaluate concerns and assure the proper balance of risks and benefits for patients. That is why we need regulatory science.

We need regulatory science to place the emerging, promising areas of science and technology fully at the service of the public health.

So how do we move forward? We make investments in projects driven by regulatory science that really work. Let me give you a few examples.

For one, FDA is working to optimize delivery and dosing of drugs and therapies so that patients receive the most benefit with the lowest risk. A case in point is the updated labeling for warfarin, a widely prescribed anticoagulant, that two million new American consumers take each year to prevent blood clots, heart attacks, and stroke.

Warfarin’s optimal dose varies and was known to be influenced by several factors including a patient’s age, diet and use of concomitant medications. Then FDA validated the determination that a person’s genetic makeup also influences how they respond to the drug…which led us to approve changes to warfarin’s label.

This change provides healthcare providers with a genetic test that can improve their estimate of a reasonable warfarin dose for individual patients. This is an example of the personalization of medicine, or tailoring a particular therapy for a particular patient—or regulatory science at work.

Then there is TB—a world-wide scourge that has eluded us for centuries. I worked to fight TB in the early 1990s when I was Commissioner of Health in New York City, but despite the push I and countless others made during the resurging epidemic in the U.S. 20 years ago, we still lack effective diagnostic tests as well as the combination drug products that would make a real difference to treatment. But with regulatory science comes promise.

This past week, the FDA awarded $2.9 million dollars in grants to support regulatory science for TB, including projects to develop new biomarkers for vaccines, to create a TB specimen repository, to design new pre-clinical models for drug combinations, and to develop and validate new point of care tests.

In addition, the Reagan Udall Foundation has been working with the Gates Foundation and others to advance the Critical Path to TB Drug Regimens program, a collaborative initiative involving companies with TB drug candidates as well as multi-lateral stakeholders, to accelerate the development of new TB drug regimens.

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. FDA is helping to 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 is using new science to guide the assessment of subpopulations of responders and the evaluation and use of new diagnostic tests in that context.

Another important challenge is the development of medical countermeasures. In August, HHS Secretary Kathleen Sebelius announced an initiative to develop the countermeasures we need to strengthen and speed our nation’s response to bioterrorism and naturally occurring infectious disease threats—a top priority for this Administration and the nation.

Because FDA evaluation of product safety and efficacy so significantly impacts the course of product development, the review identified our agency as critical to the success of the overall enterprise and we were awarded $170 million to support our efforts.

Activities will include: enhanced review and novel manufacturing approaches for the highest priority medical countermeasures; assessment and optimization of the legal, policy, and regulatory framework for countermeasures; and advancing our regulatory science base and collaborations to improve regulatory evaluation, create viable regulatory pathways, and speed development.

This is the greatest infusion of regulatory science dollars in any FDA project to-date and shows recognition across government of the importance of regulatory science in fulfilling the mission at hand—and the key role of the FDA in making it happen.

FDA has also been working hard with NIH, Foundations and industry to develop an artificial pancreas for juvenile diabetes, designed to continuously monitor blood sugar levels and inject the right amount of insulin. Just think of the potentially enormous value to patients with Type I diabetes and their families.

This is a complex task and for patients to benefit, we must develop a scientifically solid testing path that ensures that the devices control blood sugar levels without risking hypoglycemia.

Next month we are holding a meeting on establishing standards for approval. This kind of innovation exemplifies the type of progress we hope to make across a wide range of diseases.

And this is just the beginning. Through FDA’s new Regulatory Science Initiative, I have made it our priority to work with our partners to lead the effort to advance the field. The bulk of our resources will be used to mobilize external collaborations and partnerships, particularly with academia, and support studies in major regulatory science research areas.

In 2011, we will continue to make strategic targeted investments in areas that support our regulatory science plan and to establish pilots and feasibility studies. We hope to expand these investments in the next few years to establish Centers of Excellence in Regulatory Science, funded by FDA and focused on collaborative, multidisciplinary and multi-sectoral research activities—most likely housed in academic settings. These Centers will be true collaborations, bringing academia, industry and FDA scientists together to conduct regulatory science research in targeted priority areas.

We are also working with the National Institutes of Health through a new NIH/FDA Regulatory Science Initiative to collaboratively encourage research in the field. Just last week, we jointly awarded more than $9 million to four projects.

The projects include research on nanoparticles and their characterization, a heart-lung model to test the safety and efficacy of drugs, innovative clinical trial design and a novel strategy to predict eye irritation.

Which reminds me… leadership from the FDA requires more than just vision.

We must also possess the tools and knowledge necessary to advance regulatory science from the inside-out. Which means we are accelerating efforts to recruit, train, and retain outstanding scientists and build the science base within the agency. After all, it seems fairly obvious that the agency charged with evaluating the safety and efficacy of essential products must possess a scientific capacity equal to, if not greater than, that task.

All of these issues—as well as the promise of regulatory science for seven critical public health areas—are outlined in the FDA White Paper I referred to earlier, “Advancing Regulatory Science for Public Health: A Framework for FDA’s Regulatory Science Initiative.” You can find a copy of the White Paper on the FDA website and we have copies for distribution today, as well.

Hopefully this paper will persuade you that regulatory science really matters. But more importantly, it will convince you that the time to act is now.

Regulatory science can deliver us better, more targeted therapies...and more swiftly. It can impact not just individual health but improve our health care system more broadly. And it can lead to significantly lower costs for the drugs and medical products we need.

And the power of regulatory science is greater still.

As a nation, a major priority is to focus on investments that benefit the entire nation—investments that will grow our economy… that will increase high-value jobs in key sectors…that will foster innovative products that will drive economic development…that will enhance the safety and value of our exports and elevate our global economic competitiveness and overall national prosperity.

Investing in regulatory science is uniquely positioned to do all that.

But what probably matters most to all of us on a day-to-day basis is that a robust field of regulatory science will truly enable us to reap the benefits of modern science.

We are living in the century of biology where scientific advances are ripe. With a collective effort, we can transform these advances into therapies to prevent and alleviate needless suffering and products to enhance our quality of our lives.

This will take time. This will take effort. But we can do it. And it will make a difference.

Thank you.