News & Events
Margaret A. Hamburg, M.D., Commissioner of Food and Drugs - Remarks at the PhRMA 52nd Annual Meeting
Remarks as Delivered of Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
PhRMA 52nd Annual Meeting
March 18, 2010
Good Afternoon. Thank you Mr. [Marty] Carroll for the generous introduction. It’s a pleasure to be here at this important meeting. You have had a very full day. You’ve heard from many important—and influential—leaders and thinkers…people in powerful roles in government and people that are responsible for key aspects of our health care system, and for promoting and delivering new ideas and new products.
I will try to be brief, as I know I am the last thing standing between you and the closing reception. I appreciate all of you staying until the bitter end today, especially as there are a few other things going on in the Washington DC area these days…and some of them are pretty important for health.
But I am not going to take my time today to talk with you about health care reform…or even the health care challenges per se that we face in this country. Rather I want to focus on our shared challenge to deliver the promise of science for health.
You all know as well as I that we live in a time when science and technology are changing our world in dramatic ways, with an explosion of knowledge and capabilities emerging from many domains of research.
There are huge opportunities to improve health—to translate breakthrough discoveries and innovation into benefits for people, enabling us to better diagnose, treat, and hopefully cure or prevent disease. As you’ve heard today from earlier speakers, we are more poised than ever before to develop products that will really make a meaningful difference for 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 and fundamental to our work together. This must be a top priority for us at the FDA and for our nation.
So as today’s meeting comes to a close, I’d like to leave with you some thoughts on this important subject…and what FDA can and will do going forward to better serve the public’s health and realize the promise of biomedical research.
According to our mission statement, the FDA is “responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation.” I should note that the regulation of tobacco products was also recently added to that list.
In addition we are to help “speed innovations that make medicines and foods more effective, safer and more affordable.” And we should “help the public get the accurate, science-based information they need to use medicines and foods to improve their health.”
We have a big set of tasks before us. All aspects of our mission are crucial to the public health and well-being of our families….and science is the key factor that cuts across and unites all of our varied activities and responsibilities. And so it should be.
Importantly, science undergirds the very foundation on which the Food and Drug Administration has been built. Today’s FDA has its origins in the passage of the Pure Food and Drug Act, which was signed into law by Teddy Roosevelt over 100 years ago.
My earliest predecessor, the very first Commissioner of the FDA, was Harvey Wiley, a chemist from Bureau of Chemistry at the Department of Agriculture…and, in fact, at that time, the Act creating the FDA was referred to as the Wiley Act, in large part because Dr. Wiley caused a huge sensation during those years with his “scientific” studies of adulterated food and medicines. Though I am not sure that his work would meet the rigors of scientific research today, he was dubbed “the crusading chemist”… and he lefty his mark on the agency and the country.
Dr Wiley conducted human trials…but without the benefits of IRBs. He recruited a boarding house full of eager young male volunteers, to whom he feed meals with various contaminants such as borax or other preservative additives. He then observed and documented the results.
This so-called “poison squad” of Dr. Wiley’s became quite a media phenomenon and fueled increased opposition to adulterated foods and cure-all patent medicines filled with toxins and alcohol. All of this combined with Upton Sinclair’s turn-of-the-century exposé of the meat-packing industry, The Jungle, to lead President Roosevelt to create the FDA, vowing to protect the public against what the Act called: “misbranded, or poisonous or deleterious foods, drugs, medicines, and liquors.”
For better or for worse, the passage of the Pure Food and Drug Act emerged from the media attention that surrounded Wiley’s experiments and the gory details of The Jungle. And while Upton Sinclair later said of his book that he had “aimed at the public’s heart, and by accident…hit in the stomach,” there can be no doubt that Dr. Wiley, and his team from the Bureau of Chemistry conceived of the new FDA as an agency grounded in and driven by the latest in science.
Today’s world is very different from Teddy Roosevelt’s and Harvey Wiley’s. But even a century later our goal at the FDA is to draw on their founding principle of science in the service of public health as we learn to adapt to the powerful new forces shaping our world. As the late Senator Kennedy, one of our nation’s great champions of health, once put it, the FDA has become the most important public health agency in America.
Indeed, as we enter this new decade, it is more important than ever that we recognize three things:
First, that the explosive pace of scientific innovation demands that we assure a regulatory process that is adequate and appropriate to harness these opportunities to improve health and prevent disease;
Second, that ensuring the safety of food is not a “snapshot-in-time” task, but an ongoing effort that requires a life-cycle approach to FDA regulated products, as well as sustained commitment;
And, finally, that we live in a globalized world and that public health protection must be a global endeavor.
Before taking this job, I never appreciated how crucial—and how unique—the FDA really is. It is a science-based agency with the mission of promoting and protecting the public health. And as such it commands a special role in addressing threats to health—and as the gateway for products that people need.
Our responsibility and reach are enormous. Consider that the FDA regulates products that account for between 20- 25% of every dollar of consumer spending in this country.
Moreover, even if we only looked at products in the PHARMA arena, it is resoundingly clear that we are responsible for the oversight of products people really need, products they care about and products that matter in a most fundamental way to their health, safety and well-being.
The bottom line is that if FDA does not do its job, there is no backstop. Ours is a unique role, and it is critical that we do it well.
Since taking on my role as FDA Commissioner, I have been surprised by how many people are watching and asking whether the agency is leaning pro-industry or pro-consumer. Sometimes I feel like this is almost a cottage industry.
But this is the wrong way to think about FDA’s role. Both consumers and industry groups have a tremendous stake in a strong, fully-functional FDA that takes science-based action on behalf of public health. And at its core—as Senator Kennedy observed—the FDA is a public health agency.
And that’s why we have pushed forward with a public health agenda for the agency.
A lot of people wonder what does this mean? The Institute of Medicine has defined the mission of public health as "fulfilling society's interest in assuring the conditions in which people can be healthy." To be healthy, people need access to a safe and nutritious food supply and to the safest, most effective medical products possible. The FDA's role is to support this access and, in doing so, to promote health, prevent illness and prolong life.
For us, a public health approach means that we focus on carefully trying to balance risks and benefits, that we focus on health outcomes of people and populations, that we focus on transparency, openness and communication, that we emphasize prevention of problems rather than scrambling to respond to them once they have become entrenched, and that in all our actions, science and evidence must propel our work.
I want to turn now to the issue of science….and take a moment to look forward toward a promising pathway for innovation—both for FDA and for industry.
When I was nominated to be FDA Commissioner—now just about 1 year ago—I knew that an important task would be to strengthen science within FDA. It was not lost on me that a number of reports and advisory boards had warned about the dire state of science funding at FDA and how the scientific and regulatory demands were outstripping FDA’s capacity to respond.
It may seem obvious that the agency charged with the responsibility of 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—needs to possess a scientific capability equal to that task.
Our regulatory scientists must be able to understand therapies that are being developed using the most recent scientific advances. They must have the right tools to evaluate these therapies. And they must be a partner to the greater scientific community as they work to bring these therapies to patients.
All of this is absolutely essential to the ability of FDA to do its job—and I am happy to report that we are making progress, with new attention and resources focused on strengthening scientific capabilities within FDA. But I have come to realize that the task is much, much bigger.
Billions of dollars have been invested in biomedical research--an effort that is indispensible for medical progress-- but this research will not translate into real-life therapies for those who need them unless we make an appropriate investment in regulatory science-- -- the science and tools we need to assess and evaluate a product’s safety, efficacy, potency, quality and performance.
I am quite certain that all of you recognize how the rapid pace of change in science and technology presents tremendous opportunities. As we speak, many of you are investing in new types of products to help Americans in new, often life-changing ways.
But at the same time, I suspect you all recognize that the development of new therapies has, in fact, been in decline, and the costs of bringing them to market have soared.
Results of this unfortunate trend include lost opportunities to improve the effectiveness of U.S. medicine and growing threats to the economic health of the innovative U.S. pharma and biotech industry. I believe that new approaches and partnerships in the emerging field of regulatory science are urgently needed to bridge the gap between drug discovery and patient care.
Just as biomedical and life sciences research has evolved in the past decades, regulatory science must also evolve. We must harness advances in science and technology to ensure that we have the most effective and efficient regulatory pathways to address the opportunities before us.
We cannot rely on the techniques and approaches of the 20th century for the prompt review and approval of the prevention, treatment and cures of the 21st century
I now often use the metaphor of a rower on the Potomac with one powerful arm and one scrawny arm. Rowing with all his or her might, the boat cannot travel in a straight line. So it goes with U.S. biomedical advances over the past several decades.
Basic science, spearheaded by NIH, is pulling hard. But it also takes muscle to create innovative tools, standards, and approaches for the efficient assessment of medical product safety, efficacy, and quality.
Without these complementary advances in Regulatory Science, promising therapies may be discarded during development for two simple reasons: we lack the tools to recognize their potential and 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.
Strengthening Regulatory Science is an urgent challenge for the collective minds of academia, industry, and government. Advances in this field will help modernize product development and provide better tools, standards, assays, disease models, and science-based pathways to improve the speed, efficiency, predictability, capacity, and quality of product development, evaluation, and manufacturing.
Together, we must ensure that our scientific enterprise includes a robust, state-of-the-art field of regulatory science. This is essential to FDA's work for sure, but at least as significantly, it represents an important driver of our nation's health, the health of our health care system, the health of our economy and our global competitiveness. It is a goal that we must all embrace.
If we do so, I am confident we'll be able to speed the movement of new discoveries—of innovations—to practical applications. We have seen examples, in the recent past, where concerted scientific investment led to the development and approval of new drugs in remarkably little time. But we can and must do better.
This has a very personal resonance with me. While doing my residency in internal medicine in New York City during the mid 1980s, I was taking care of a lot of patients with AIDS. It was a very hard time. The patients had this devastating illness and yet we had virtually nothing to offer for treatment …only supportive care.
Shortly afterwards I went to work with Tony Fauci at the National Institute of Allergy and Infectious Diseases, first as his special assistant and then as Assistant Director of the Institute. By that point in the AIDS epidemic, in the late 1980s, there were a few emerging potential drugs and a huge push was being made to get as many people as possible into clinical trials of the new anti-retrovirals. It was the only way patients could get access to drugs. Every day, we were dealing with desperate people who wanted to live and had little hope.
Within a decade, FDA had provided a science-based regulatory pathway for companies to follow ... and then approved an entire new class of drugs to treat HIV infection--protease inhibitors. We had drug cocktails that were keeping hundreds of thousands of AIDS patients alive.
The early years of the AIDS epidemic were my early years in medicine, and they gave me a deep appreciation of the fundamental importance of scientific insights and innovative research, but also what scientists, pharmaceutical companies, advocates and FDA can do working together.
I am excited by the opportunity that I believe we now share to really advance and develop the field of regulatory science…and in doing so, make a real and enduring difference in people’s lives.
Several partnerships with industry and universities to advance regulatory science are starting to bear fruit, but there is so much more to be done.
I am encouraged that the President’s FY 2011 budget includes the first ever initiative for regulatory science. And just several weeks ago, I sat on stage beside HHS Secretary Kathleen Sebelius and NIH Director Francis Collins, who you heard from earlier today, to announce an important collaboration between FDA and NIH to help bring the fruits of science to patients—or, in the words of Secretary Sebelius, to bring them “from the microscope to the marketplace.”
The goal is to place the emerging, very promising areas in science and technology—such as genomics and personalized medicine, the development of stem cell therapies and therapies that harness the power of nanotechnology—fully at the service of the public health.
We must support such things as the identification and application of novel biomarkers of disease, innovative clinical trial designs, standards for the characterization of stem cells, valid safety assays, and other regulatory advances.
And before I close, I want to focus briefly on one additional aspect of regulatory science that I think will be increasingly important and that we must work on together…and that is safety science.
We have long known that even the best pre-market clinical trials can track the impact of a drug on only a relatively small, and carefully selected sample of patients, for a limited period of time. The data we glean from these trials stops far short of giving a full picture of what really happens when hundreds of thousands of patients actually are taking the drug.
We are entering a new era for drug safety where protecting public health means that the FDA’s responsibility doesn’t end when we grant a product pre-market approval; that is merely the first check point in assuring safety.
The fuller, and more accurate, picture emerges when a drug enters the real world of the mass market. And the FDA’s responsibility to monitor and act on safety risks that emerge is one that extends for the entire time those products are on the market.
We know that there are enormous opportunities to improve both innovation and safety if we can effectively leverage science and research to address medical product needs throughout the lifecycle of a product.
At the heart of it is the safety-focused science of pharmacovigilance, where using various data sets and reported information, we can effectively monitor the post-market environment for safety signals and other risk concerns. And as we do more to look for emerging safety signals in the marketplace, we will find them and must have meaningful strategies to rapidly evaluate those concerns and assure the proper balance of risks and benefits for patients.
Looking forward, I believe that we must deepen our thinking—and enhance our activities—to better address the scientific, technical and ethical issues involved in evaluating post-market signals about drug safety.
There is much to talk about, but I think it is time to close. These are challenging and busy time for us all. We have different pressures and responsibilities….but I do believe that we have a shared—and rather special-- opportunity to work together to drive innovation and to deliver its fruits to the people who rely on us.
I look forward to continued engagement with all of you as we define and implement the scientific agenda that can help us truly translate discoveries, innovation and promise into real world products for those who need them.