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Speech by Dr. Hamburg to the Food and Drug Law Institute (FDLI) 2014 Annual Conference

Speech by Margaret A. Hamburg, MD
Commissioner of Food and Drugs
2014 Food and Drug Law Institute (FDLI) Annual Conference
April 23, 2014
Washington, DC

Good morning. Thank you Philip [Katz] for that kind introduction. It is a pleasure to be back with all of you again this year for your annual conference. It is hard to believe that it is my fifth time here.

But this is an important meeting and an important organization and we share so much, including efforts to ensure informed conversation and problem-solving in critical areas of food and drug law, policy and regulation.

This is not insignificant. Success today in the world of regulation cannot be accomplished alone. It requires developing new ways of thinking and working that involve unprecedented partnerships and collaborative ventures across disciplines, experiences, and sectors. And the relationship between FDLI and FDA is a prime example of the benefits of partnership and cooperation.

This morning I want to begin my remarks by paying tribute to someone who really has helped solidify and expand this long and vibrant relationship – while helping FDLI advance and broaden your own mission -- your fine executive director, Susan Winckler.

I’m sure there is great disappointment that Susan has announced she is leaving FDLI. Hers will be big and elegant shoes to fill. But she leaves behind a legacy of important and lasting contributions.

Working closely with FDLI’s excellent leadership and quality staff, Susan has helped to enhance the value of membership in FDLI, reinforcing it as a go-to place for information and substantive discussion on all sectors of food and drug law by a wide range of voices. 

Susan’s achievements to strengthen FDLI’s reputation include the unglamorous and often painstaking work of building from within -- revitalizing committees, engaging more members, and expanding the use of technology to enhance the availability and effectiveness of FDLI publications and conference materials so that they align even better with members’ interests and needs.

But she also has made sure that this organization continues to be an essential resource by ensuring that your work embraces the constant developments in food and drug law. She has integrated tobacco within the Institute’s work and reenergized and strengthened your work in the international sector to address the growing issue of globalization.

Needless to say, FDLI … and FDA … will miss Susan. It has been a pleasure working with her, which I have had the honor of doing, truly since I started at FDA. 

In fact, next month marks my five year anniversary as Commissioner of Food and Drugs – a length of time that sounds longer than it feels – at least on most days. And since our society tends to pay special attention to multiples of five, it seemed like today might be a good opportunity to talk about the some of the big picture goals I have embraced in this job, particularly our efforts to reposition FDA to be a stronger, smarter agency for the 21st century.
When I was first appointed, more than a few people – some of whom were quite well informed in the ways of Washington offered me the advice – unsolicited, of course -- that I should plan to limit the expectations of what I could accomplish in terms of change or reform.

The agency is just too big, the Washington bureaucracy too difficult and intractable, and I was further reminded that I would also have to deal with Congress.

They suggested that I pick a few issues or initiatives I thought were really important and where I might be able to get something accomplished in a couple years’ time. Now there was nothing especially wrong with that advice. But, as someone who had spent my professional life committed to strengthening structures and mechanisms to improve public health, it went against my basic nature and experience.

More significantly, as I took stock of the agency, its vast responsibilities, and its enormously talented and committed workforce -- I came to the conclusion that if FDA was truly to fulfill its mission in the modern era, this was a critical time to reposition in several fairly fundamental ways, addressing substantive issues as well as structural changes. And so, the priority areas I have focused on have involved underlying institutional change and have revolved around three main themes: engagement, science and globalization.

Perhaps most importantly, I felt it was critical that we open ourselves up as an agency. To ensure that our vital work at FDA is done with the best possible input from stakeholders, and with the trust and confidence of the public we serve, it was imperative that we increase transparency, enhance stakeholder engagement, and strengthen partnerships across sectors, disciplines and components of government. This is essential to ensuring that we make the best possible decisions, extend our reach and impact, and make the most efficient use of resources possible.

We’ve been approaching this in many ways. Through the transparency initiative launched soon after my arrival as Commissioner, we sought to bring new clarity and understanding to what we do, how we do it and why—for the general public, industry and other key stakeholders.  We have embarked on important new public-private partnerships in many domains of activity. And we have upgraded our communications including a far more accessible web site, with more timely and useful postings, and more recent ventures with social media, from blogs to Facebook to Twitter. 

We also have been undertaking regular listening sessions, hearing from various leaders, experts, and advocates, seeking ideas and feedback, as well as our recent efforts on patient-centered medicine, including holding dozens of public meetings with patient advocacy groups for specific diseases.

If you look across our many accomplishments in the past year, and there were many--they all build on this model. Our ongoing conversations with stakeholders informed the rollout of these important initiatives. With strong stakeholder engagement and being very clear and transparent about what we wanted to do and why, there was better information flow and fewer surprises. Just a few of the more recent initiatives include:

• Our final rule for a unique device identification system (UDI) that, once implemented, will provide a consistent way to identify medical devices.

• Our final guidance for mobile medical apps focusing FDA oversight on a narrow group of apps, provides developers with the needed clarity that they asked for.

• The proposed strategy and recommendations for a risk-based regulatory framework for health information technology that we released just a few weeks ago with HHS’s Office of the National Coordinator for Heath IT (ONC) and the Federal Communications Commission reflects the many comments we heard in public meetings about how to best promote innovation, protect patient safety and avoid regulatory duplication.

• Our work on food safety has involved enormous outreach and engagement. Since January 2013, we have proposed 7 major foundational proposed rules to implement the FDA Food Safety Modernization Act, or FSMA. This is a huge effort and will transform our food safety system in meaningful ways. To get these rules right, Mike Taylor and his staff have fanned out across the country to hear from farmers, producers and others in the community who are affected by these changes. The input and exchange continues to be an invaluable part of the policy making process.

• And in just the past couple of months a number of other efforts featured significant stakeholder engagement, including our proposal of a new nutrition facts label and our launch of groundbreaking education campaigns to prevent young people from starting to smoke. 

I’ve also sought to identify and modify Agency functions and processes in a way that will help us better achieve critical objectives. For example, we are working to strengthen and align programs and operation with more coherent policy and strategic development, and to better integrate the work of the centers with field programs so that instead of duplicating efforts we are strengthening the connections and the underlying work on these issues.

We have also worked to improve communications and collaboration within the Agency, with greater training to ensure the necessary levels of scientific and technical competence at a time when we are dealing with the reality of much more complex products in a more complex world.

While we still have much to do, I believe many of the changes we already have put in place, both in terms of strengthening our internal organization and the ways we work with industry and others, are making a difference, both in the quality of our work and in the support we are receiving for what we do.

One measure of that support comes from Congress. At a time of incredible partisan bickering, Congress has given FDA repeated votes of confidence, coming together to enact a series of landmark bills. The Food Safety and MOdernization ACt (FSMA) fundamentally shifted the paradigm for food safety from responding to contamination to preventing it. The Food and Drug Administration Safety and Innovation Act (FDASIA), not only reauthorized our important user fee programs but promoted innovation and enhanced the safety of the drug supply chain.

And last year Congress passed the Drug Quality and Security Act (DQSA), designed to improve the safety of drug compounding and further protect the drug supply by building an electronic, interoperable system to identify and trace certain prescription drugs as they are distributed in the United States. 

Now some degree of compromise is necessary for a bill to be enacted and we certainly don’t get everything we might want in these important pieces of legislation - as happened with the DQSA. But at a time of fundamental disagreement – even disagreement about FDA’s role - it is remarkable that Congress ultimately decided to put its trust in FDA to fix these public health issues...and the bill passed without objection in both the House and the Senate.

Congress has also voiced its confidence in FDA through its budget process.

Five years ago, FDA’s annual budget was about $2.7 billion. It’s currently about $4.4 billion dollars or 63 percent greater, which is remarkable at a time when our nation has been faced with huge fiscal challenges. Of course, our funding ballooned after the passage of the Tobacco Control Act, but nevertheless, FDA’s budget has actually increased every year for the last seven years.

I must underscore that this does not mean that we have all the funding that we need to fulfill the many responsibilities placed on us and what the public expects. Nor does it mean that we aren’t still working to recover from the sequester and other budgetary challenges of the last few years that hampered our work. But it does indicate that Congress understands that the responsibilities we have – including new ones that they have given us in recent years -- are critical to the health and safety of this nation and also that we are responsible stewards for the resources we do receive.

One way that I think we have built this trust and support for the Agency’s work is by focusing our attention on the central importance of science and evidence-based decision-making. And importantly, we have brought new attention to the role and value of regulatory science.

Regulatory science allows us to combine greater understanding of the underlying mechanisms of disease and human biology with new technologies and scientific advances to inform both more efficient product development and  new tools and strategies for smarter regulatory decision-making. This in turn helps us create and support meaningful and lasting innovation for the public good. 

As a field, regulatory science has been underappreciated, underdeveloped and underfunded, but it is, in fact, vital to our work at FDA and well beyond.

A robust field of regulatory science can help us leverage opportunities for innovation and more quickly bridge the gap between scientific discovery and real-world products and processes that make a meaningful difference in people’s lives.

And to be successful, regulatory science must bring together the best minds wherever they are found across industry, academia, government and elsewhere. It is through collaboration that we can truly advance this science and its applications.
FDA plays a critical role because we more fully understand what it takes to translate a good idea into a product with demonstrated safety, efficacy and quality and a product that can be scaled up and reliably manufactured.

FDA also is uniquely situated to examine important unmet medical and public health needs and how they match up with what is in the development pipeline. Part of my focus has been to support better alignment and incentives to address the gaps we see, and to foster the kinds of innovation that will make a real difference.

Simply waiting until we see what comes through our doors for review cannot be the going model. By advancing new and more efficient regulatory processes and bringing a higher level of collaboration, scientific engagement, oversight, and innovation to the table, we can encourage and enable new solutions and processes to bloom.

As an example, consider our collaboration with the Medical Device Innovation Consortium, which aims to advance regulatory science in the medical device industry. One of the consortium’s major projects is to develop and validate computational modeling and simulations which could be used in lieu of clinical studies in certain circumstances to generate data on a product’s safety and effectiveness, reducing the time and cost of development.

We have also established several Centers of  Excellence in Regulatory Science and Innovation – one at the University of Maryland, another at Georgetown University, and more are planned.

Another approach involves the many ways we have streamlined the drug review process and are using a range of strategies and pathways to help speed product development and review as appropriate. Most recently, we are implementing the new “Breakthrough Therapy Designation”  that was part of FDASIA. As of the end of March, we had received more than 166 requests for this designation; some 42 drugs have received the breakthrough designation and five have been approved.

I think we all have been surprised by the level of interest in this new pathway and its emerging value. But in fact, nearly half of the new molecular entities or NMEs approved by FDA last year took advantage of at least one of the available expedited drug review pathways. And some of these drugs were approved in as little as 4.5 months and three quarters of these NMEs were approved in the U.S. before anywhere else.

The success of these pathways has been possible because of our evolving approaches to both regulatory science and enhanced opportunities for product sponsors to engage with FDA reviewers and high-level FDA scientists and managers during earlier stages of drug development. We are finding that this kind of engagement can reduce the overall time for both development and review, as well as lead to better and more innovative products. I am pleased that we are now looking at creating a new expedited access program for devices that address unmet public health needs for patients with serious illnesses – modeled in part after these successful efforts in pharmaceuticals.

However, in keeping with the mantra that FDA has only two-speeds of approval—“too fast and too slow”--some have suggested that we are moving too quickly or sacrificing standards and consistency — for speed — in the review process.  Yet I think this misconstrues the mandate to add flexibility and tailor clinical trial requirements for each drug and the disease area for which it is being studied.

Certainly when Congress created the "breakthrough" designation, it recognized that the responsibility to help translate the newest discoveries and research into treatments for patients who are critically in need as the goal of drugs developed through the accelerated approval process.  And that means a rigid, "one size fits all" approach is not the answer.

It is important to understand that no matter what approval pathway is used, the Agency consistently applies the same statutory approval standards of safety and efficacy to all drugs in order to be marketed in the United States. Increased flexibility does not mean abandoning standards…and it certainly does not mean abandoning science. Just the opposite. We need to employ the best science in ways that will increase our efficiency, productivity, and our shared ability to find creative solutions to the challenges that confront us.

I’ve had a lot to say about the importance of regulatory science to medical products, but it also provides transformative opportunities to strengthen food safety. Cutting edge regulatory science is also critical to our efforts to combat food-borne illnesses.

For example, we continue to develop new technologies that are helping us strengthen our response to food-borne outbreaks. The Whole Genome sequencing program, being developed by FDA’s Center for Food Safety and Applied Nutrition (CFSAN), for instance, will allow us to quickly and accurately identify specific strains of pathogens in the foods that are making people ill and help us rapidly trace back foodborne illnesses to the source of contamination. These are developments that just a few decades ago might have seemed more like science fiction, than science.

And regulatory science applies to the historic work we are doing in the realm of tobacco. Our charge from Congress is an enormous one – to reduce the harm from tobacco products across the population.

One important aspect of this is to ensure that we have a thorough scientific research base that will allow us to make informed regulatory decisions.  That’s why we are funding groundbreaking research with NIH, CDC and partners in academia, on a range of critical concerns including patterns of behaviors and tobacco use, including e-cigarettes, as well as on the chemical components and possible toxins in tobacco products. And it is also why we are establishing 14 Tobacco Centers for Excellence at academic institutions throughout the country to focus on key research questions in this area.

Although FDA currently regulates cigarette tobacco, roll-your-own tobacco, and smokeless tobacco, as part of the Tobacco Control Act, however, Congress also gave FDA the authority to deem other tobacco products to be subject to the Food, Drug & Cosmetic Act (FD&C Act). As you probably know, we have been working on a “deeming” rule to potentially extend our authority to regulate additional products meeting the legal definition of a tobacco product.

This has been a long time in development but I can say today with some certainty that the proposed rule will be available for discussion and comment imminently.

We have a lot on our plate, but across all of our domains of activity, the challenges get ever more complex as we must also grapple with our dramatically changing global marketplace. This change, in which the foods we eat and the medicines and medical devices we use increasingly come – in whole or part - from countries other than our own, has enormous ramifications for the work FDA does, and the people we work to protect. 

And the numbers are astounding. Fifty percent of fresh fruits and nuts, 20 percent of vegetables, and over 80 percent of seafood consumed in America comes from abroad.  Similarly, 40 percent of finished drugs come from outside our borders, and 80 percent of active ingredients manufacturers are located outside the US.  Further, half of all medical devices are imported. And the volume of imports only continues to rise.

The fundamental shifts that are taking place as a result of globalization have huge implications for our ability to ensure the safety and quality of products manufactured elsewhere and the integrity of supply chains as those products move through an increasingly complex network of producers, manufacturers, packagers, distributors, exporters and importers. I would argue that this transformation also comes with huge economic and national security challenges as well.

As for FDA, we have already begun to reorganize our structure and transform from a domestic agency operating in a globalized world to a truly global agency fully prepared for a complex regulatory environment that takes into account the risks across a product’s life. This requires new reliance on enhanced intelligence, information and workload sharing with the help of regulatory partners, data-driven risk analytics, and allocation of resources achieved through public and private partnerships.

Most importantly, our job requires greater coordination of regulatory standards and practices across nations to ensure safety and quality, regardless of where a product is produced.  We are working through bilateral and multilateral agreements, as well as through international organizations, specialized partnerships and various coordinating bodies. And at the highest levels, we have embarked on creating a new model or framework for global governance.

But specifically with respect to our work at FDA, much has happened in recent years. We have greatly enhanced our ability to do targeted border screening as products come in, using a computer driven, risk-based program (called PREDICT) to identify imports that need fuller examination versus those commodities at low risk that can proceed through. But that alone is not enough. We must work to enhance safety and quality long before these products reach our shores. We have enhanced overseas inspections and now have FDA offices in many parts of the world. This is proving very beneficial, but again, we must do more. We cannot inspect our way to safety with some 300,000 facilities in about 150 countries making products for export to the United States that we must oversee.

That is why the collaboration with both industry and our regulatory counterparts is increasingly vital. The rapid and profound rise of global commerce and trade requires that continue to evolve and meet new demands. We can take comfort in the knowledge that we have significantly strengthened our ability to respond to these challenges and know where and how we must do more.

Similarly, the rapid advances in science and technology offer great opportunity but place new and evolving demands on us and the products we regulate.

Understanding the seriousness of these challenges – structural, scientific, economic, and global – is one reason why, when I took over as Commissioner, I could not settle for incremental changes in how FDA intends to conduct business for the 21st century. I am confident that the changes we are putting in place are sufficiently important and on track to survive well into the future. I believe that they are, in fact, absolutely essential.

And so we will continue to implement changes that fundamentally transform how we address these challenges before us, how our agency interacts with regulated industry and other stakeholders, and how the public understands and supports the mission of this important and unique agency.

It will require the work not just of the FDA, but of partners such as FDLI, to ensure the continuing alignment of our public health and medical care mission with the important regulatory and legal framework. I look forward to our ongoing work together, with continuity of purpose, renewed commitment to key principles, and a sense of the dynamic and changing world we live in that demands flexibility, resilience and an eye to the future.

Thank you.