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FDA and Innovation for the 21st Century

Remarks as Prepared for Delivery of Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
FDLI Annual Meeting
Tuesday, April 5, 2011

Good morning—and thank you for that generous introduction and warm welcome. It is a pleasure to be here again to speak to you. This is an organization with very close ties to the FDA, and by now I feel that I can really consider so many of you valued colleagues and old friends.

And as friends of our agency, you know that we have faced significant challenges and accomplished a great deal in the past year.

Since I saw you last, we have grown, evolved, renewed, and refined our commitment to serving the American people and promoting and protecting their health and well being. And we focused on some key priority areas—integrity, the importance of science, and responding to the challenges of a globalized world.

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 new tobacco legislation.

We worked to regain the trust and confidence of the American people by putting in place a rigorous initiative to promote transparency for the public and accountability within the agency.

We recommitted ourselves to strong and fair enforcement policies.

We strengthened science at the FDA and promoted, more broadly, the field of regulatory science to support the development and advancement of the knowledge and tools needed for the effective and efficient review of product safety, efficacy, quality, and performance.

And we worked to shift the paradigm of how we interact and partner with the international community as we strive to ensure the safety of imports and meet the demands of our modern, globalized world…strengthening systems, building new capacities and, importantly, collaborating in new ways with our international counterparts to build a public health safety net for consumers around the world that is supported and maintained by a global alliance of regulators.

Of course, this is just a sample of the work we’ve done this year. Our scope and responsibility are enormous, and we have a full plate for 2011. Our increased responsibilities will, frankly, be a challenge, especially in light of the current budget situation—and almost certainly, we will be asked to do more with less…easy to say, hard to do.

But from the beginning, I have pledged to be a strong advocate for what our agency needs. Rising to this challenge is particularly critical at this time, because history has shown that everyone—consumers and industry alike—benefits from a strong, effective FDA that is fully equipped to fulfill our public health mission…which is a point I have made to this group several times before. And I know you take it to heart.

In fact, each time I’ve spoken with you, I have taken the opportunity to outline a vision for action—and described in some detail the ever-evolving framework that we use to do our job as regulators but, more importantly, as public health professionals. And I’d like to continue that tradition today.

This morning, I would like to talk to you about innovation. Now, I realize that in many quarters, innovation has become something of a buzzword. It has taken on different meanings for different people, but the fundamental idea is consistently the same. As we as a nation look ahead into the 21st century, we are realizing how important it is for us to stimulate our industries, encourage entrepreneurship and creativity, and prepare our citizens for the future. And for all of that, we need innovation.

But at the FDA, innovation has always been a central focus. Even though we aren’t always fully recognized for it—we have a long history of innovation at the FDA. In fact, it is part of our mission. We are charged with both protecting and promoting the health and well being of the American people. This means that our job is both to ensure the safety and efficacy of FDA-regulated products and to take real steps to foster the scientific innovation that will lead to tomorrow’s new breakthrough products.

For FDA, innovation is not just about new ideas, but it is about making sure that those new ideas truly translate into the products and opportunities that people need and count on. Moreover, it’s about changing systems…replacing outmoded or insufficient patterns with new, better, more effective ones.

Let me tell you how.

A century ago, Burton J. Howard, chief of the Bureau of Chemistry's microchemical laboratory, needed a way to prove his suspicion that the tomato ketchup industry was using preservatives and spices to conceal worm-eaten refuse in their cans from consumers. Using a standard blood-counting cell and a compound microscope, he developed the Howard Mold Count. And with a simple, scientifically-rigorous review process in place, he was able to prevent countless illnesses for many years to come.

In 1911, that was called innovation at the FDA. Today, innovation looks very different.

It looks like a "transcatheter" pulmonary heart valve that can be implanted through a catheter, eliminating the need for open-heart surgery in some patients.  In 2010, the FDA approved this product under a "humanitarian device exemption," moving it through the system in fewer than five months of review.

It looks like using whole genome sequencing technology to help trace the source of a recent Salmonella outbreak in eggs back to the contaminated product and from there to the contaminated food facility.

It looks like Halaven, one of 18 FDA-approved treatments for advanced breast cancer. Halaven was approved after a seven-and-a-half month review on the basis of a single study in 762 women who had received at least two previous regimens of chemotherapy. On average, patients receiving Halaven in the study lived three months longer than those receiving an alternate therapy.

And, amazingly, it looks like two truly revolutionary prosthetic arms being developed by the Defense Advanced Research Projects Agency, or DARPA. One arm, being designed and tested by DEKA Research, is an advanced prosthetic arm and hand with a neural interface. It is an incredible innovation and getting very close to market.

The other, being developed by the Applied Physics Laboratory at Johns Hopkins, is a neurally controlled arm and hand prosthesis. The plan is for it to actually be controlled directly by human thought through an implanted sensor on the brain surface. That would mean that it could be used in cases of paralysis—and would not be limited to those who have had an amputation. This neural control is the aspect that truly pushes the envelope past anything else near the market—and is truly astounding.

The fact is we are in a golden age for biomedical discovery. Complex chemistry and biosynthesis has promised new drug candidates; the sequencing of the human genome has revealed opportunities for advanced, targeted therapies; and cutting-edge electronics, nanotechnology, and materials science is revolutionizing medical devices. And that barely scratches the surface.

The potential of these discoveries is great, and the stakes—for patients and for medical progress—have never been higher…and we have much to deliver. In the next decade, powerhouses in Europe and Asia will test America’s role as the leader in global medical product industries, forcing us to make choices about how to move forward.

And as we do so, the FDA is prepared—and eager—to help lead the way.

As you know, we are an agency whose impact is felt far outside the walls of industry, the scientific community, or even our homes. In the U.S., FDA-regulated products account for more than 20 percent of consumer spending and total more than a trillion dollars annually.

The industries we regulate currently lead the world in innovation, provide millions of high-paying jobs, and several are among a handful of vital industries with a positive trade balance with other countries. At the same time, we help save billions of dollars in unnecessary spending by preventing foodborne illness and getting products to market that lower healthcare costs and save lives.

Our mandate is enormous—and what we do really matters to the lives of Americans every day. Which is why it is so important that it is clear: our role is to facilitate innovation, not to impede it.

But we are facing some major challenges.

As you know, the FDA plays an essential role in the development of new products and therapies for patients and consumers. But that pipeline is at risk: despite unprecedented spending on basic research and development by the government as well as the biotechnology and pharmaceutical industries—to the tune of more than $80 billion this past year—the number of submissions of new molecular entities to the FDA for review is on the decline.

In addition, the rate of failures in drug development remains high: recent reports suggest that any given drug studied in early human clinical trials has less than a 10 percent chance of making it successfully all the way through to approval and marketing, with most of the attrition occurring during clinical trials, well before the actual FDA review process begins. And as medical devices and other products become increasingly complex, the standards and tools we use to evaluate their safety and efficacy are not keeping pace.

In other words, the return on investment for biomedical products continues to drop.

Unfortunately, FDA has been identified in many analyses as a key factor in this decline—largely because rising product development times and costs are attributed to delays and uncertainty in the regulatory approval process.

We certainly get a lot of criticism—some of it deserved—but in reality the situation is a lot more complex. There are many factors involved and, looking to the future, we must, as a nation, develop meaningful strategies to address what is a real and serious problem. Looking at FDA, it is important to recognize the proactive role we need to play, and also to address some myths and misunderstandings. In fact, our rate of approvals has not slowed for many years, even as products become more complex.

And despite what you may be hearing, we have a strong track record for meeting and often exceeding tough deadlines for review times established by industry in conjunction with FDA as part of the user fee process. We have also made progress in speeding products to market. In the past few years, 90 percent of the 3,000-plus 510(k) medical device submissions we receive each year were reviewed in 90 days or less.

In addition, there are a lot of criticisms out there that we are slower than Europe in approving new products—and are therefore driving companies away.

In fact, FDA consistently approves new drugs and devices as fast or faster than other countries, while maintaining the safety standards that Americans count on. For example, of the 57 novel drugs approved by both FDA and the EMA, the FDA’s European counterpart, from 2006 through 2010, 43 were approved first in the United States.  Of the 27 drugs in this group that were given priority review because of their special therapeutic potential, 24 were approved first by the FDA…and the FDA’s median time to approve these priority drugs was 183 days, some 220 days faster than the EMA.

Similarly, a study recently commissioned by the device industry showed that FDA approves low risk medical devices faster than Europe, and higher risk as fast as Europe.

Regardless, we certainly don't view our agency as "in competition" with the EMA or any other sister regulatory agency—our scientists work closely with theirs and share information on a regular basis. And as we strive to meet the challenges of globalization, as I mentioned before, this is more important than ever.

The truth is that on the world stage, the FDA is still regarded as the premier regulatory agency, the one that other nations look to as a standard... and one that opens up international markets for FDA-approved products.

And we continue to have a unique perspective as a regulator, but also as a science-based public health agency. So, at this critical moment of need for patients and consumers in our country and around the world, we have a real responsibility to take advantage of this unique position and deliver new, innovative products to the people who need them most. And this is an opportunity for us to strengthen and modernize our scientific capacity to support the review and approval of products for the 21st century.

Which brings me to the obvious question: what does all this mean for the work we do at the FDA?

Well, as you know, innovative products that are truly transformative create unique scientific and regulatory challenges. So we are rolling up our sleeves, developing FDA’s regulatory science and innovation initiatives and strategies, and doing everything we can to rise to those challenges—and to ensure that FDA is a consistently powerful catalyst for innovation.

Moving forward, we will focus our efforts on several distinct yet interrelated areas of activity—each of which must be addressed if we are to make a meaningful difference in supporting innovation.

First, regulatory reform. We will continue to work hard to streamline and modernize regulatory pathways, and make them more predictable and transparent.

As part of this effort, we recently announced plans to reform our 510(k) process, which allows for expedited review of around 90 percent of FDA-regulated medical devices. Under these reforms, we will streamline 510(k) submissions for eligible devices, clarify criteria for eligibility, and provide a more efficient review process.

Earlier this year, we announced 25 specific actions to improve the predictability, consistency, and transparency of our premarket review programs. We also announced a novel priority review program for pioneering technologies, aimed at encouraging and fostering the development of cutting-edge medical device technologies, strengthening the nation’s research infrastructure, and advancing quality regulatory science for all medical devices. Notably, it promotes the exchange of ideas among external and internal innovators, device experts, and FDA staff—building a framework for recognizing innovative ideas early and often and incentivizing innovation for years to come.

And on the drug side, FDA has developed a number of partnerships with industry, academia, other government agencies and various other stakeholders, to strengthen the scientific underpinnings of our regulatory work and better harness advances in science and technology to make the review process more effective and efficient. We have implemented a "21st Century Review Initiative" which is designed to further standardize, modernize and streamline the drug review process.

And for drug manufacturing, we instituted the quality by design approach, a system through which, working with companies on manufacturing systems that build in quality and consistency, we have been able to see significant reductions in failure rates.

In each of these cases, regulatory reform involves changing practices but also developing and applying better scientific understandings and tools to the review process.

Which brings me to the second area: strengthening science and reducing scientific uncertainty.

Much of regulatory uncertainty is rooted in scientific uncertainty: a lack of clear, reliable data that supports decision making for product safety and efficacy. There are many reasons for this scientific uncertainty, including increasingly complex medical products and an incomplete understanding of human biology. But most importantly, it stems from a lack of attention and investment in regulatory science—the science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of FDA-regulated products.

This, in my view, is unacceptable. Investments in regulatory science can drive sound, smart regulatory policies that lead to safer, more innovative products for people who need them, while at the same time contributing to the economic growth and health of the device and pharmaceutical industries.

Which is why advancing regulatory science on behalf of the public health has been a top priority since I became Commissioner. Through our Advancing Regulatory Science Initiative, we are devoting time and resources to leading 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.

For example, last year, the FDA and the National Institutes of Health launched a new NIH-FDA Regulatory Science Initiative to encourage research in this field. Last year, we awarded our first set of grants in regulatory science—$9.4 million over three years to support four research projects—and we hope to be able to expand this program soon.

Another productive partnership has been with the Foundation for the NIH and PhRMA to create The Biomarkers Consortium, an innovative public-private partnership that is searching for and validating new biological markers to accelerate the delivery of successful new technologies for the prevention, early diagnosis, and treatment of disease.

As we move forward with the initiative, and resources willing, we hope to establish Centers for Excellence in Regulatory Science, housed at academic institutions, funded by FDA and focused on important research in the field.

Regulatory uncertainty also is rooted in our inability to fully share our knowledge. We house all of the data for every medical product ever submitted and approved in the US, yet no one has mined this information to learn how to better predict failure…or design drugs and studies better the next time. Sharing data and enhancing communications with companies without compromising their trade secrets is another priority for our agency.

This is what I meant by changing systems. We need to replace what is outmoded or not working with stronger, more effective patterns and practices. If we focus on building the science base that drives all aspects of our work at FDA—both the protection and promotion aspects of our public health mission—and strengthen the field of regulatory science more broadly, we can promote the kind of innovation we need to deliver real progress for patients and consumers all over the world.

Finally, as a nation we must focus on economic policy—including incentives, tax credits, how to deal with market uncertainty, access to capital, reimbursement and many other issues. This is not directly in FDA's domain, but it's extremely important. And, of course, we sit in a unique position to see how different approaches are working. Orphan drugs is one good example where we have been able to witness the impact of certain incentive approaches on product development in this important area. And success has also depended on our ability to wed important incentives with innovative scientific approaches to meaningfully study candidate drugs in novel ways.

The work of the FDA is increasingly being recognized as essential to all aspects of the public health—including innovating it for the future.

We have always been an agency committed to innovation and to bringing the best, most creative, most effective ideas to bear for the American people. But now, we are taking all of our efforts to the next level. In this challenging time, the FDA can—and will—be part of the solution. And our focus on smart, forward-thinking, science-based action will help get us there.

All of you probably know a bit about our roots—and the two President Roosevelts—Teddy and Franklin—that made us into the agency that we are today. But our history actually stretches back even before that to our 16th president, Abraham Lincoln. It was Lincoln who created the Division of Chemistry that would evolve decades later into the Food and Drug Administration.

Under very different circumstances and at a very different time, the Great Emancipator himself offered us advice that rings as true today as it did then: "The occasion is piled high with difficulty," he said, "and we must rise—with the occasion. As our case is new, so we must think anew, and act anew. We must disenthrall ourselves, and then we shall save our country."

Like everything we do at FDA, our vision for innovation is a vision for the future of public health. So we owe it to ourselves—and to the American people—to think and act anew. Only then can we truly deliver what the people need and what they deserve.

Thank you.