FDA Innovates

Remarks given at the NEHI Conference on Bridging the Innovation Gap
By Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Boston, Massachusetts
April 26, 2012

Thank you, Beverly for the generous introduction.

I am honored to be invited here today.

It is a privilege to address one of the largest, most diverse health care organizations in the United States.  But I am especially pleased to be asked to address a topic that is very close to my heart—and occupies much of the thought, creativity and work of everyone at FDA. 

I’ll get to innovation at FDA in a moment.  But I want to start by commenting on the NEIH Innovation Barometer survey that Glen and Chrystine presented an hour ago.  I thought it was very interesting. Working on the inside of a large complex agency, it is always valuable to gain the insights of health care leaders, and I don’t ever want to lose sight of how what we do affects health care and the public.  Yes, we innovate.  And, yes, we regulate.  But we also need to make sure we’re listening and working together.  That is why I was very pleased to learn from your survey that the great majority of the healthcare opinion leaders appreciate FDA’s work.

My colleagues and I take that appreciation to heart, and we will continue to work hard to earn the trust and support that Glen and Chrystine talked about.

I am particularly encouraged by the support indicated for increased funding for FDA. 

This is a vital concern. Money alone does not guarantee a success, but FDA’s responsibilities have grown dramatically in recent years. It is hard to imagine, but FDA is actually responsible for the regulation of products that account for somewhere between 20 and 25 cents of every dollar of consumer product spending in this country. We regulate drugs, medical devices, biologics and vaccines, the safety of our nation’s blood supply and the vast majority of our food supply; cosmetics, dietary supplements, products that emit radiation…and most recently, tobacco products. The scope of our responsibilities is vast, and they concern things that really matter, to all of us, each and every day.

Increased funding would certainly help us address the many mandates given to us by Congress—and to fulfill our unique and essential mission to promote and protect the health of the public. I believe strongly that both consumers and industry groups have a tremendous stake in a strong FDA that takes science-based actions on behalf of public health.

And—without doubt—that includes promoting innovative products and advancing regulatory science.

So let me turn now to the topic of innovation. It is an issue that is important to us all, whether you come from industry, from academia, from clinical practice, or from government. Of course we each bring our own set of questions and concerns to the table. But we all recognize that we are at a critical juncture.

For the people here from the medical products side—those who develop new devices, diagnostics, and drugs—these have not been easy times. The trend lines for innovative products relative to investments in research and development are not what any of us would like. The costs of bringing new drugs to market have skyrocketed and the pipeline of promising new drug candidates is distressingly low.

Many companies are facing the so-called "patent cliff."  Worsening the situation is the unstable economy, changing rules for reimbursement, and, I know, concerns with the FDA…fears that a lack of predictability, consistency, and transparency in our decision-making processes and actions will lead to less investment in innovative technologies and companies, as well as increased competition from foreign companies.

These are challenges that I understand, appreciate, and we are working hard to address.

And for the people here from the healthcare side—those responsible for delivering care to patients—many of you are frustrated that we, as a scientific and medical community, have not been able to adequately realize all of the seeming opportunities in science today.  And that is true. Patients are urgently waiting for new therapies—and often not getting them.  And we are not adequately addressing burgeoning public health needs.  Disease conditions such as obesity and Alzheimer’s, are taking a growing toll, profoundly affecting American families, and measurably adding to our health care costs and our national debt.

How is it that we are in a veritable golden age for biomedical discovery…the sequencing of the human genome has revealed new potential drug targets; combinatorial chemistry, high throughput screening, and biosynthesis has led to thousands of candidate drugs; cutting-edge electronics, nanotechnology, and materials science has the power to transform medical devices...yet despite this, we are not effectively translating these scientific discoveries into therapies, prevention or cures.

There is nothing I would like better than to find a single solution to all of these challenges.  But there isn’t one.  Instead, we need a comprehensive, integrated strategy that engages the full "eco-system" to advance biomedical product innovation. This includes new and strategic investments in research, driven by American ingenuity and science with input from our global partners, and more thoughtful design and implementation of policies on everything from patents and intellectual property to economic incentives for innovative medicines, reimbursement issues, and, of course, sound regulation.  Meaningful and enduring solutions will require true collaboration by stakeholders – and strong leadership from business, academia and government. 

And that is why a meeting like this is so important.

History tells us that full engagement and leadership can work.

When I was in my first year of medical school, my classmates and I were told that we were on the threshold of consigning infectious diseases to the history books. We might as well have been told that the sun revolves around the earth because over the next few years, we watched in dismay the emergence of a mysterious new illness.

No one knew what caused it.  No one knew what to call it.  And certainly no one knew how to care for it. The AIDS epidemic had begun.  For those who contracted HIV, we had no effective treatments—and no medicine on the horizon. We could offer neither cure, nor hope.  

As a physician, I’ll never forget how impotent and ineffective I felt…powerless to defeat the virus…or extend life…or even to ease my patient’s suffering much.  But scientists and pharmaceutical companies creatively applied themselves to the task, patients mobilized, and communities got involved.  At the same time, healthcare practitioners worked tirelessly, and the FDA responded by accelerating review and approvals.

Once recognized as a national public health priority, leaders from across the medical research and healthcare system responded by working together.  They produced tangible, meaningful, and lasting results for patients afflicted with HIV/AIDS.

Thankfully, today HIV/AIDS is no longer a death sentence.  It is a chronic, but manageable, disease.  This one of the great scientific and technological achievements of the last 30 years, and it would not have happened without an all-out effort by the entire scientific and health care community.  What we have accomplished in the fight against HIV/AIDS is surely a testament to the need for, and the power of, innovation.

Innovation is a core element of the FDA’s mission.  But innovation is not just about basic research and new discoveries.  It is about taking those new good ideas and translating them into products that are stable and reliable, that are safe and effective for their intended purpose, and that will provide real clinical benefit for the patient. Innovation is also about finding new and better ways to do things to meet the needs and challenges before us. 

This is why regulatory science is so important. When I started talking about the importance of regulatory science—which I’ve been doing relentlessly since I became Commissioner—I got a lot of raised eyebrows. A common response was:  "Regulatory, what?"  Frankly, these days, at least in Washington, you are far more likely to hear the word "regulatory" linked to the phrase "job-killing" than to "science." But believe me, this is a short-sighted view.

I suspect that many of you who work in the medical product development area are very familiar with what I am getting at when I talk about regulatory science, but some of you probably are not. So perhaps I should take just a few minutes to briefly describe what I mean.

Regulatory science is the science 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.

It is, quite plainly, an essential component of the scientific enterprise. Yet it has been under-appreciated, under-developed and under-funded for many years. And, as a result, we have been unable to apply the best possible science and technology to the tasks before us.

We are clearly at a crossroads. And we face some major challenges. But I also view this as a leadership moment for the FDA. We have an opportunity to build momentum and deliver progress on behalf of patients in America and around the world.

To do so, we must look inward. Certainly 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. So we are working hard to support, strengthen, and enhance FDA’s already outstanding workforce, from clinical and scientific reviewers, to lab and expert manufacturing scientists and inspectors.

But equally important, we must look outward. We are currently engaged in an agency-wide effort to advance the field of regulatory science and engage our partners across government, industry and academia. In my view, regulatory science is the critical link—the bridge—between cutting-edge discoveries and real-world diagnostics, treatments, and cures.

We cannot take full advantage of the breakneck speed of  biomedical research unless we also emphasize innovation in regulatory science.
A robust field of regulatory science can 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 can 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 can 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 can usher in an era of personalized medicine, by linking advanced genetic data and biomarkers with targeted therapies.

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

We can 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 can 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.

As we work to strengthen the field and apply the new knowledge to what we do, regulatory science can have outsized impact in other areas, as well.

Importantly—and uniquely—investments in regulatory science can help address growing threats to the economic health of the innovative U.S. pharma and biotech industry and help to grow more broadly our national economy. These investments would increase high-value jobs in key sectors, foster innovative products that will drive economic development, and enhance the safety and value of our exports, elevating our global economic competitiveness and overall national prosperity.

The FDA has developed a strategic plan for regulatory science. But advancing regulatory science will require the full engagement of the scientific community and beyond. It will take new investments, approaches, and partnerships for regulatory science to evolve in the ways necessary to keep up with and capture the opportunities ahead of us.

Let me give you a few examples of steps we’ve already taken:

We’ve formed a Joint Leadership Council with the National Institutes of Health to help promote the translation of basic and clinical research into medical products and therapies. This effort includes some joint grant-making for extramural projects as well as collaborative intramural activities.

We’ve established three Centers of Excellence in Regulatory Science—one involving a set of research universities in Arkansas, one at Georgetown University and one at the University of Maryland.  These Centers will conduct targeted research, strengthen the training of FDA’s medical products reviewers, and bolster scientific exchanges and collaboration.

And we are working with the Reagan-Udall Foundation, a private and independent nonprofit research organization, on several exciting regulatory science projects.

FDA will continue to take a leadership role in driving regulatory science forward.  But we have another distinct but related innovation task.  As we advance the scientific underpinnings of our regulatory framework, we must also be sure that our regulatory pathways are as streamlined and modern in other ways as well.

In plain English, FDA must not be a roadblock.  Just the opposite:  Our job is to enable innovation – but without sacrificing our high standards for ensuring safe, effective and high quality products.

This is a difficult balancing act…and one of the hardest aspects of my job. But it has to be something that we are aggressively focused on.  And as an agency, we’ve had measureable successes. 

I won’t indulge in a long list.  But let me give you a few highlights.  In the Device arena, last year, after taking a systematic and clear-eyed look at ourselves and a lot of outreach to industry, patients and the health care community, we identified 25 specific steps to help address unmet medical needs and reduce the time and costs of medical device development…and we have acted on almost all of them.

We’re giving manufacturers clear information about how FDA judges the benefits and risks of medical devices during their pre-market review.  And we’re providing much better guidance to companies developing similar versions of already licensed biological products.  

When you look at recent drug approvals, some important trends emerge. Last year, we approved 35 new, groundbreaking medicines, the second highest number in the last 10 years. They include drugs targeting 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. Two of the drugs—one for melanoma and one for lung cancer—were approved with a diagnostic test that helps identify patients that will most likely benefit from the drugs. These two products exemplify the power that personalized medicine can hold. 

Sixteen of these new drugs were reviewed within six months. FDA’s average approval time for a drug has dropped from two years in 1992 to just over one year.

And of the 35 new drugs, nearly 70% were reviewed by FDA for quality, safety and effectiveness—and approved for use in patients—before they were available in any other country in the world.  But this is not a unique event.  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, we streamlined clinical requirements to permit smaller, shorter or fewer studies wherever possible.

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.

But I believe this record shows our ongoing commitment to helping companies navigate the FDA’s regulatory pathways—and bring their safe and effective medical products to patients as quickly as possible.

The goal is to encourage product innovation.

That’s why our Center for Devices and Radiological Health created Innovation 2.0 pathway, a novel approach for working collaboratively to expedite approval of groundbreaking new devices.   This new pathway was designed with the help of outside "entrepreneurs-in-residence" who developed the framework and operating procedures together with an FDA team.

Another way to encourage product innovation is by providing clarity about our expedited drug reviews, in particular accelerated approvals, a mechanism that has been highly effective. Since its introduction in 1992, it has brought more than 80 new products to market, including 29 cancer drugs and 32 HIV drugs. We believe this mechanism can be increasingly utilized for rapid approval of a wide variety of diseases and conditions, including rare diseases.

In addition, for drugs that demonstrate a dramatic therapeutic response early in clinical trials, we have been exploring a possible new regulatory pathway to expedite development of "breakthrough" therapies that would facilitate the use of shorter and smaller trials, and more intensive engagement of FDA with the sponsor to reduce duration of the development process.

I have only mentioned a few examples of some of the new thinking that is going on throughout our agency. We’re looking at old problems in new ways while confronting new challenges.

I’m proud of the changes FDA has made over the years to accelerate drug and device approval—while never neglecting our duty to protect the public.

But we cannot work in isolation.  Finding new and more efficient ways to get products to people—while keeping those products safe and effective—is a responsibility we share with our private sector partners, with academia and with patients and consumers. 

And it is just that kind of strategy that the White House is unveiling today in its national Bioeconomy Blueprint, which outlines a comprehensive approach to harnessing innovations in biological research to address national challenges in health, food, energy, and the environment.  The blueprint will guide Federal agencies—in coordination with one another and in partnership with private-sector entities—to enhance economic growth and job creation, improve the health of all Americans, and move toward a clean-energy future through scientific discovery and technological innovation.

I would call your attention in particular to the plan’s support for investments in research and development, to its focus on translational and regulatory science to help move inventions from the lab to the market, and to its advocacy concerning public-private partnerships and pre-competitive collaborations. 

Later today, at a White House event, Secretary of Health and Human Services Kathleen Sebelius will mention two significant FDA initiatives as part of the Bioeconomy Blueprint.  I hope that as you learn more about this ambitious program, you will lend it your expertise and your support.

I look forward to continuing to work with you…for it is our collective efforts that will drive innovation in medical products forward…and it is those collective efforts that will allow us to meet the needs of the patients we serve.

Thank you