News & Events

Lessons Learned: How the FDA Matters

Remarks by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
National Press Club
Newsmakers Luncheon
March 27, 2015

(This text provides Dr. Hamburg's prepared remarks, and it should be used with the understanding that some material may have been added or deleted during presentation.)

Good afternoon. Thank you for that kind introduction. And thanks also to the members of the National Press Club for inviting me to speak to you today.

This is my last public address as FDA Commissioner, so I thought this was a perfect opportunity to reflect on what I have learned about this agency, which I firmly believe – now more than ever-- - is essential to the lives and health of every American, every day.

It is hard to overstate the unique and vital importance of this agency for all of us, but I’ll confess that I didn’t arrive at FDA with this fully formed perspective. I did not begin to appreciate the vast scope of FDA’s responsibility until I was ensconced in the job.

It still amazes me that the products we regulate account for between 20-25 cents of every dollar spent on consumer goods in this country. More specifically, FDA is responsible for promoting and protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, vaccines and biological products, medical devices, the safety of most of the food supply, the blood supply and other tissue products, cosmetics, and products that emit radiation. Most recently, FDA is responsible for regulating the manufacturing, marketing, and distribution of tobacco products.

And some things might surprise some of you, ranging from bionic eyes and replacement body parts made with 3-D printing, to the use of medicinal leeches. We oversee the safety of food provided to dignitaries at events like the State of the Union and I just learned this week that we regulate waste discarded from moving trains...Whatever it is, FDA is there for you.

But it’s not just the diversity of the products we oversee that some might find surprising – it is the way that we bring our enormous expertise to bear. In this age of skepticism about government, it is easy to imagine FDA regulators simply as bureaucrats focused on a narrow set of responsibilities.

Yet nothing could be further from the truth. What a remarkable group of physicians, scientists, lawyers, policy analysts, and other professionals and support staff, committed to helping people get the products they need and count on. Some of whom are here today.

I learned that FDA staff do far more than just use their knowledge and expertise to review applications or investigate safety concerns. They also undertake vital research to advance medical product innovation and improve food safety.

For example, scientists from our Center for Biologics made a crucial contribution to the development of the MenAfriVac meningitis vaccine by developing a needed conjugation technology. This vaccine has now protected more than 217 million people from what was a deadly killer across the so-called “meningitis belt” of sub-Saharan Africa.

FDA’s food scientists have helped develop sophisticated genome sequencing technologies to more rapidly identify– and stop – food borne outbreaks.

During the Gulf oil spill, I was surprised, but very grateful, when another team of FDA scientists developed a new laboratory technique that significantly accelerated the testing process for certain types of oil-related chemicals in seafood, enabling the FDA and States to reopen Gulf State waters more rapidly…and it got the very agitated Gulf Congressional delegation to stop calling! I was even further surprised when the White House dispatched me to New Orleans to eat seafood and demonstrate that the seafood supply was safe!

So there are all sorts of thing to talk about, but I want to focus on just a few key issues today—issues that I have focused on since I began and that I believe have made an important difference in strengthening and reaffirming FDA’s critical role in American society.

I think most would agree that I came to the FDA at a time when the Agency faced considerable difficulties and uncertainties. A series of visible foodborne outbreaks had resulted in disease, nationwide anxiety and economic disruption.  Several drug safety crises—Vioxx for one—had eroded public confidence.

At the same time, FDA was facing serious resource threats.  Budgets were tightening with the economic crisis, but chronic underfunding had already stretched the agency thin in many critical areas, and jeopardized our ability to keep up with inspectional demands, product reviews and with evolving science and cutting edge biomedical products at the very time that scientific and technological discoveries were revolutionizing medical products.

FDA’s challenges were exacerbated by the increasingly global marketplace for the products we regulate.  Imports of FDA regulated products were growing dramatically, and there was a series of serious episodes associated with adulterated products coming from oversees, most notably the imports from China of tainted heparin, and melamine contaminated dairy products and pet foods that caused deaths and serious illness.
These effects and constant negative drumbeat in the media, combined with Congressional criticism took a toll, public trust was flagging…yet it was clear that there was much good work going on.

Indeed, as I took stock of the agency--its vast responsibilities, and its enormously talented and committed workforce –it was clear that FDA was at crossroads…and decisions made then would matter in fundamental ways and for a very long time. If FDA was truly to fulfill its mission in the modern era, this was a critical time to reposition.

To do this I focused on three priority areas: increasing public engagement accountability and partnership; re-invigorating our scientific base by advancing regulatory science and underscoring the need for science-based decision-making—and scientific integrity-- as the foundation for all we do; and lastly, addressing the challenges of globalization and the huge implications that has for health, and for the safety and security of the products we regulate.

Six years later, thanks to an extraordinary leadership team and dedicated employees, we’ve seen enormous and important progress. We have renewed, expanded, and refined our mission and activities in important and powerful ways.

Notably as well, Congress has given us important new authorities to regulate cigarettes and other tobacco products, to transform our nation’s food safety system with a new focus on prevention, and to use more flexible and streamlined approaches to bring exciting new medical products to patients in record time.

To be effective, FDA must do its vital work with input from stakeholders, and with the trust and confidence of the public we serve. That is why it was imperative to increase transparency, enhance stakeholder engagement, and strengthen partnerships across sectors, disciplines and components of government – and we did.

Soon after I arrived at FDA, we launched an agency-wide effort to make useful, understandable information about the FDA more readily available to our stakeholders.

This Transparency Initiative brought greater clarity and understanding as to what we do, how we do it, and why —for the general public, industry, patient and consumer groups, and other key stakeholders. We also worked to increase collaborative efforts, including establishing important public-private partnerships.

We enhanced our communications, including listening sessions with leaders, experts, and advocates, seeking ideas and feedback. We focused as well on patient-centered medicine, which involved holding dozens of public meetings with patient advocacy groups for input on specific diseases. With stronger stakeholder engagement, there has been better information sharing, more predictability, and, ultimately, a better process and product.

My second priority—science—really builds on and reinforces the efforts just mentioned. As a science-based regulatory agency, our credibility and success depends on our ability to deliver on the promise of science, through smart, data-driven decisions that benefit patients and consumers. Smart regulation also requires the ability to respond to changing situations, new information and new challenges; we cannot have a “one size fits all” approach—but we must always bring the best possible science to bear.

And it requires that we advance regulatory science: both  the knowledge and tools necessary for the meaningful and timely review of products for safety, efficacy, quality and performance, and to inform more efficient product development.

Building on greater understanding of the underlying mechanisms of disease and human biology, 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 that will make a difference in people’s lives.

So advancing regulatory science is a huge priority, not just within the walls of FDA, but as an active, dynamic field of scientific research.  We are continually working to find new and better ways of doing things.  To seize new opportunities that exist in science and technology.  And to work with industry and our scientific partners in academia and government—in a collaborative way—to discover and apply new regulatory tools.
So what does this really mean?

In the foods area, using regulatory science we have taken critical actions that will improve the safety of the food Americans consume for years to come.  Importantly the development of science-based standards to create a food safety system focused on preventing foodborne illness, rather than responding after, thanks also to the Food Safety Modernization Act. We have also taken several significant steps to help Americans make more informed and healthful food choices.

These include working to reduce trans fats in processed foods; more clearly defining when baked goods, pastas and other foods can be labeled “gluten free;” updating the iconic Nutrition Facts label based on current nutrition science; and, most recently, finalizing the rules to make calorie information available on chain restaurant menus and vending machines.

In the medical product domain, we are pursuing such things as: enhancing the use of pharmacogenomics and qualified biomarkers; developing innovative clinical trial designs which enable clinical studies to be smaller, more efficient, and more adaptive; and developing new strategies using bioinformatics to more effectively mine large data bases to learn more about issues of both safety and efficacy, as well as such things as how we identify subpopulations of responders to a given treatment based on certain indicators.

These efforts matter in our ability to swiftly and surely review product applications that come before us…and they are also essential for reducing the time and cost—and increasing the likelihood of success—for product development itself.

In the ecosystem for biomedical product development, 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.
Simply waiting until we see what comes through our doors for review cannot be the going model.

FDA is uniquely situated to examine important unmet medical and public health needs and how they match up with what is in the development pipeline. Indeed a growing part of our focus in recent years has been to identify what is in the development pipeline, provide guidance and incentives to address gaps and accelerate progress, and foster the kinds of innovation that will make a real difference for patients.

Also, we have seen how early and continuing engagement between the FDA and researchers in the product development plan makes a huge difference in streamlining the process and making sure that the right questions get asked and answered from the get-go.

As you may know, we now have in place a number of expedited review programs that help to speed the development and availability of medical products that treat serious diseases. For prescription drugs, we have fast track, priority review, accelerated approval and now, thanks to recent legislation, FDASIA, we have the breakthrough therapy designation.

And we are seeing both development and review times decrease significantly, with exciting new therapies entering the marketplace sooner for the patients who need them.

Last year, we approved the most new drugs in almost 20 years and more orphan drugs than ever before. 41% of these new drugs were first-in-class products, resulting in a breathtaking array of truly innovative new therapies for patients. And the majority were approved using some kind of expedited regulatory pathway.

Today, FDA approves drugs faster on average than all other advanced nations, and the vast majority of the time, the U.S. is the first country in the world to approve important and novel medicines. Substantial improvements in the efficiency of medical device reviews have also been made.

Moreover, we have accomplished this while remaining the world’s gold standard for safety and effectiveness.

Yet we all recognize that despite these successes, too many diseases still await treatments and cures.  Serious public health needs, such as treatments for Alzheimer’s disease, are not being met.

In response, some have said that FDA regulation is the principal obstacle to the development of innovative treatments, and suggested that FDA’s authorities and procedures be fundamentally re-considered.

I strongly disagree. In actuality, regulation—when it’s done right—isn’t a roadblock—it’s the actual pathway to achieve meaningful and lasting innovation.  Smart, science-based regulation instills consumer confidence in products and treatments. It levels the playing field for businesses. It decreases the threat of litigation. It prevents recalls that threaten industry reputation and consumer trust—not to mention levying huge preventable costs on individual companies and entire industries. And it spurs industry to excellence.

The fact is, when done right, smart regulation allows us to deliver on the promise of science in the service of patients, consumers—and yes, even industry.

It is foolish—in fact dangerous-- to believe that reducing regulatory standards will make new treatment interventions appear if the science is not there.

Alzheimer’s is a good example. I have heard comments of late that something must be wrong with FDA because we are not approving as many drugs for Alzheimer’s as we are for cancer, and we are certainly not doing it as quickly.

Yet the reality is not the problem of unnecessary regulatory hurdles but rather the need for the medical research community to increase  understanding of the underlying disease process, natural history, and where are the best targets for therapeutic development. We are working closely with the Alzheimer’s research community and patient groups to do everything we can to help advance possible treatment strategies and product development approaches…and I hope we will soon see meaningful progress.

Of course there are sometimes tensions between moving new products swiftly out into the marketplace and making sure that they have been adequately studied.

As FDA commissioner, I have been surprised by how many people ask whether I favor safety or innovation. In fact at my confirmation hearing, when I guess I was a bit of an unknown in terms of the perspective I would bring to this position, someone supposedly went through and counted how many times I said safety vs how many times I said innovation.

They presumed this to be a measure of whether I was pro-industry or pro-consumer. I’m told it was about equal, but I never went back to check….and I certainly do not believe that the two are mutually exclusively. Why should we have to choose?  Protecting the public health—while encouraging, not discouraging innovation must be the goal…and it is.

After all, innovation is only meaningful if it makes a real and positive difference in the lives of patients and consumers.

That is why we must have standards—and science—to assess the benefits and risks. And when it comes to the treatment of disease, we must understand the broader context of use: the nature of that specific disease or condition, the other treatment options, and such.   We must also better understand the patient’s experience of the disease and its treatment, their perception of the risks and benefits, and of course their willingness to accept risks.

The balancing of risks and benefits is fundamental to FDA's regulatory role.  And it is always a challenge.  We joke that FDA is viewed as having only two approval speeds: too fast and too slow.

We are perceived as too quick to approve a drug or device when a significant safety issue is identified in the post-market context once the product is in widespread use.

On the other hand, we were too slow in approving when a drug that has undergone a lengthy development and review is finally approved and provides a real therapeutic benefit to patients.The task for FDA's scientist is to strike the right regulatory balance.

I also want to speak briefly about the importance of striking the right balance between fast access and good science.  In the race for the newest treatment, we must remember the point that innovation doesn’t matter if the product doesn’t work.

I cannot emphasize enough the critical need to maintain the standards of safety and effectiveness for medical products in this country.  It wasn’t long ago that companies were allowed to market drugs without proving that they were effective.

We have only to look back at that time to see the devastating consequences for patients and for medicine.

Drugs were marketed for thousands of unproven uses, most of them unsupported by adequate research. When in the mid-1960s Congress gave FDA authority to require evidence of effectiveness, almost 80% of the drug uses that companies were promoting turned out to be ineffective.

Many of them were also dangerous — for example, before companies had to show that their drugs worked, drug companies widely promoted powerful, toxic anti-psychotics like thorazine for low-level anxiety.

And there was little or no incentive to conduct the research necessary to find true medical advances.  Most promotion was based on unscientific studies, or no studies at all.

It’s important to understand that FDA supports responsible communication of scientific information.  But we do not support an approach that will harm patients or undercut the incentives for the necessary studies to be done to prove that that a specific use of a drug product is both safe and effective.

History has shown that patients have been harmed by physician reliance on preliminary or incomplete scientific information regarding unproven uses.

History has also shown the enormous patient benefits that result when a sponsor conducts rigorous clinical studies and demonstrates that a promising medical product is, in fact, safe and effective in treating a serious disease or condition.

FDA's objective is to strike the right balance between respecting the usefulness of communicating scientific data in certain circumstances on the one hand, and preventing harm to public health on the other.

We must not forget that the great leaps forward in evidence-based medicine of the last 50 years have come in large part as a result of the high standards for product approval that Congress put in place after a series of disasters involving unsafe and ineffective medical products.

Those standards have boosted the confidence that Americans place in medical products and that the world places in the American biomedical industry.  We must move forward, not backward, as our nation embraces the opportunities of cutting edge biomedical advances and the promises that they hold for the public health.

I want to turn now to the new responsibilities we are taking on to address the changing global realities that affect the health, safety, and security of Americans.

When FDA was established, our regulated industries were predominantly local and the volume of imported products was low. Today, however, other nations increasingly produce—at least in part-- the food and medical products America consumers and patients use in their daily lives.

Nearly 40 percent of finished drugs Americans consume today are made elsewhere, as are about 50 percent of all medical devices.  Approximately 80 percent of the manufacturers of active pharmaceutical ingredients used in the United States are located outside our borders
About 15 percent of our food supply is imported from other countries, but this includes some 85 percent of our seafood, nearly 50 percent of our fresh fruit, and 20 percent of our fresh vegetables.

The result of these changing dynamics is that at every step in our increasingly complex global supply chains there are potential risks to the consumer – through improper formulation or packaging, contamination, diversion, counterfeiting, or adulteration. This has huge implications for our health, safety and national security.

We no longer can rely on simple inspections at the border to ensure the safety of the products Americans consume. We have introduced new hi-tech, risk-based screening systems at the borders, but we must also work to prevent problems before they reach our borders. This requires greater coordination with other nations and international organizations to strengthen quality and safety oversight in the countries of origin.

And we have established FDA outposts around the world to serve as hubs for inspections as well as improved communication with stakeholders.

Most importantly, we increasingly collaborate and coordinate with our global regulatory partners, using enhanced intelligence, information and workload sharing to ensure safety and quality regardless of where a product is produced.

And though time is short, I also want to briefly mention the enormous progress we have made in taking on several other public health responsibilities. Shortly after I became Commissioner, Congress gave us the authority to regulate tobacco products. FDA’s scientists assigned to our new Center for Tobacco are proud to be at the forefront of the fight to reduce the risks of one of America’s biggest public health dangers. And here too, we are bringing science-based regulation to the manufacturing, marketing, and distribution of tobacco products.

We also fought successfully to change the law to strengthen FDA’s oversight ability to address a different serious health problem. When a lethal outbreak of meningitis was traced to a compounding pharmacy in Massachusetts, it became urgently clear that action was needed to address a set of compounding pharmacies that were becoming essentially drug manufacturers without oversight. We successfully engaged in the passage of new legislation that would close a gaping loophole for those producers – legislation that we’re now in the midst of implementing.

We’re also making progress against a major, growing public health problem…antimicrobial resistance.  A policy FDA instituted last year, after literally decades of trying--to phase out the use of medically important antimicrobials in food-producing animals for growth promotion purposes and to bring the remaining therapeutic uses of these drugs under the oversight of veterinarians – will go a long way toward protecting future antibiotics.  

All of these efforts—and many others—have played a role in restoring not just the ability of the FDA, but confidence in FDA by the people this Agency protects.

Recent opinion polls show that Americans support the work of the agency and value that its decisions are based on science not politics. One Gallup poll showed that between 2009 and 2013 FDA’s rating increased more than any of the other federal agencies covered in the survey. I hope they also appreciate that FDA is a real bargain, costing each American only about $8 a year for our many and essential services. Less, I suspect, than some of you may pay for a visit to Starbucks later today—and yes, their products are regulated by the FDA.

Standing here—not just as I conclude my remarks but also my tenure-- I’m proud to tell you that today’s FDA is a stronger, more engaged and more effective agency, better positioned for the challenges of the 21st century. We are an organization that embraces smart, science-driven regulation.

Our regulators – including highly trained and compassionate doctors and scientists -- play a unique and essential role in promoting and protecting the health of the public – from helping to speed the development and availability of new products that diagnose, treat or prevent diseases, to assuring a level of scientific rigor and a systematic approach that involves collaboration and leads to meaningful and lasting innovation and betterment of public health.

The role of the FDA as a public health agency is central to the health and safety of individuals, communities and our nation. We need to strengthen, rather than weaken or undermine it. To do so requires the support and partnership of all our stakeholders.  I look forward to watching that vital collaboration and to seeing the work of this relatively small in size --but mighty in purpose -- agency be both appreciated and supported.

Thank you

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