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FDA Commissioner Margaret Hamburg Details “Regulatory Science” for 21st Century Medicine

Universities, industry, and government must join to develop state-of-the-art “regulatory science” that can speed evaluation of new drugs and medical procedures and bring them more quickly to patients, Dr. Margaret Hamburg, commissioner of the U.S. Food and Drug Administration, told a AAAS audience.

Advanced research and technology are driving remarkable new discoveries, Hamburg said, but the United States’ current process of evaluating and approving them for use dates to an earlier era. The resulting gap between new discoveries and older treatments already on the market—some have termed it the “valley of death”—brings risk not only to patients, but to the nation’s economy and innovation culture, she said.

“We live in a time when science and technology are changing our world in dramatic ways,” Hamburg said. “We are seeing an explosion of knowledge and capabilities emerging from many domains of research and from around the globe. And with that comes a fundamental question of how do we make sure that we fully translate the potential and promise of that research into real-world products and programs that really matter.

Dr. Margaret Hamburg, commissioner of the U.S. Food and Drug Administration (left), and AAAS President Alice S. Huang

“A big part of the answer is by strengthening regulatory science. This is a component of the overall scientific enterprise that is really essential, and yet has been underappreciated and underdeveloped.”

Hamburg delivered a luncheon address at the 2010 AAAS Forum on Science and Technology Policy. The Forum celebrated its 35th year on 13-14 May with a selection of programs and events that included talks by John P. Holdren, assistant to the president for science and technology and director of the White House Office of Science and Technology Policy; U.S. Representative Vernon J. Ehlers (R-Michigan); Douglas W. Elmendorf, director of the Congressional Budget Office; Sabine Herlitschka, director of European and International Programmes for the Austrian Research Promotion Agency; and Patrick Gallagher, director of the National Institute of Standards and Technology.

Hamburg has had broad experience, and her influence has ranged from bioterrorism to infectious disease control and U.S. health policy. While New York City health commissioner from 1991 to 1997, she advanced innovative programs for controlling the spread of tuberculosis and AIDS. Between 1992 and 1997, the TB rate for New York City fell by 46%, and by 86% for the most resistant strains.

In 1997, she was selected by President Bill Clinton to be assistant secretary for policy and evaluation at the U.S. Department of Health and Human Services. Beginning in 2001, she served as vice president for biological programs at the Nuclear Threat Initiative, which is dedicated to reducing the threat from nuclear, chemical, and biological weapons. By unanimous voice vote in the U.S. Senate, she was confirmed as President Barack Obama’s FDA commissioner in May 2009.

She took charge as the FDA was under broad criticism for its handling of U.S. food safety oversight, its approval of drugs that later were found to pose dangers to some patients, and ties with the pharmaceutical industry that were, in the view of critics, too close.

While the FDA’s regulation extends over a quarter of the U.S. economy, regulatory science as discussed by Hamburg at the AAAS Forum is not widely-known. Where research scientists may take years to discover a new drug, regulatory science generally seeks to do its work more quickly; if research scientists develop a new cancer therapy, it is the goal of regulatory science to establish a mechanism that allows for effective evaluation of the therapy, and, if it is safe, allows its use by patients.

Generally, it is a tool used by government, and it is shaped by elected officials and other policy-makers. It is multi-disciplinary, often involving clinical research, epidemiology, statistics, and other fields. It closely overlaps with a growing field of initiatives in translational medicine, which seeks to translate cutting-edge discoveries into regular medical practice. [AAAS and Science last year launched a new weekly journal, Science Translational Medicine.]

Hamburg conceded that regulatory science lacks the allure of “discovery science.” But without the best possible regulatory science, she suggested, a too-lengthy time may pass before the benefits of discovery find practical use for patients.

“Americans cannot take full advantage of the breakneck speed of biomedical research unless we also emphasize innovation in regulatory science,” Hamburg said. “Just as biomedical research has evolved over the past few decades, regulatory science must also evolve in important and powerful ways.”

She cited several fields of medical research where regulatory science will be critical for supporting clinical research—in studies of emerging stem cell and cancer treatments, for example, or in the effort to more tightly control tuberculosis, or TB.

But, she said, TB is a complex challenge. A cure requires multiple drugs administered over many months, and if a TB infection is not eradicated, the disease can evolve so that it’s resistant to drugs.

“Fighting TB effectively requires combination products,” Hamburg explained. “It will take forever if we have to approve each new drug independently and then look at how they work in combination. But that is how the process would traditionally work. Instead, we have embarked on a new initiative to work with researchers and companies interested in developing combination products which will be reviewed as such.”

That’s “much more complicated for FDA scientifically,” she added, “but it is what patients need and what public health demands.”

The effort to develop a more robust regulatory science requires the FDA to build its in-house science capacity, Hamburg said. And, she added, it will seek close collaboration with allied medical and regulatory agencies, such as the National Institutes of Health (NIH). Already, she said, the FDA and NIH recently launched “an important and broad collaboration” in translational and regulatory science.

She said that FDA will continue and expand relationships with academic groups, patient advocacy groups, and industry. International collaboration also could play a critical role in advancing regulatory science.

“There are enormous opportunities to collaborate with scientific colleagues around the world on matters of regulatory science, and also to engage with international sister regulatory agencies to address issues of common concern, share information, and to harmonize standards and approaches,” Hamburg said.

“Outreach and collaboration are central to regulatory science efforts. When successful, these collaborative efforts will help predict more swiftly and effectively which discoveries will succeed or fail as actual products, thereby reducing product-development costs and getting better products to patients faster.”

President Obama has backed an overhaul of the agency, and Hamburg said that the FDA already is benefiting from renewed attention and new resources. But given the precarious state of the U.S. economy, she predicted that the agency faces a significant challenge in meeting its regulatory science goals in the near future.

“A robust, state-of-the-art regulatory science is essential to the work of the FDA,” she insisted in her conclusion. “But more than that, it represents an important driver of our nation’s health, the health of our health care industry, and the health of our economy. … It’s vital to our nation’s global competitiveness. It is a field of endeavor that must be fully embraced by academia, industry, and government.”


Read the full text from Margaret Hamburg’s address to the AAAS Forum on Science and Technology Policy.

See more news from the 2010 Forum.

Get details about the program and speakers at this year’s Forum.