Policy: The Art of What's Practical

That strong affirmation of singular leadership was good news for pharma. Hamburg's background as an AIDS researcher and in tuberculosis treatment exposed her to the importance of biomedical innovation and drug development in providing doctors with tools to treat serious diseases. After attending medical school in the 1970s, the emerging AIDS epidemic lured her away from academia. During those early days of clinical trials for AIDS treatments, "I decided I wanted to work at the intersection of medicine with broader social, legal, economic and service issues." She saw how important it was to support those discoveries and translate them into "real-world care for the people who need them."

Hamburg also understands the nuances in assessing risk in bringing new drugs to market. An approval process that is too fast might fail to detect safety problems, while too slow a process can delay treatments for patients with life-threatening diseases. Hamburg considers it a "false dichotomy" to separate safety from innovation, and says that promoting public health involves finding the right balance. FDA can do more in the area of drug safety, she suggests, but not at the expense of delaying innovation. As she and Sharfstein wrote in the NEJM article, "Some benefits are not worth the risk; some risks are worth taking."

FDA may be willing to take more risks, Hamburg says, due to additional authorities provided by the FDA Amendments Act (FDAAA). The ability to restrict distribution and to better detect safety problems of newly approved drugs may support earlier approval of important medications. And more collaboration with other government agencies, industry, and patients could help detect safety problems and identify fruitful pathways for research that can lead to the approval of treatments.

To assess drug risks and benefits appropriately, FDA needs to fully understand the science behind emerging research and products. Hamburg is concerned that the nation's huge investment in biomedical research has not been balanced by comparable support for regulatory science.

"[FDA is at] the nexus of translating genomics into targeted therapies and new diagnostics," she noted at a symposium on the "Future of Personalized Medicine" in October. This requires moving beyond randomized, controlled clinical trials to new test methods that can to distinguish responses and effects on smaller patient groups.

Hamburg promises a more consistent, more integrated approach to evaluating medical products related to personalized medicine, citing plans to issue draft guidance on biomarker qualification and on co-development of drugs and diagnostics. FDA also hopes to spur development of new biomarkers that can identify patient subpopulations most likely to respond to a drug, or to have an adverse reaction.

Better Infrastructure for Good Science

For FDA to support the translation of discoveries into medical products, Hamburg believes it's important to define more effective and efficient regulatory pathways for emerging products. This involves recruiting and retaining top scientists and giving them the facilities and opportunities they need to do their jobs.

It also means stronger collaboration with the scientific community on ways to streamline the drug review process. One idea is to bring together academia, industry, and government at centers for regulatory excellence in order to assess biomarkers and develop new strategies for clinical trial analytics. The Commissioner envies Europe's Innovative Medicine Initiative, which has 2 billion Euros to spend on developing biomarkers, diagnostics, and translational research. Hamburg would like to start a similar interconnected program in the US.

One vehicle for a replenished regulatory science base could be the Reagan-Udall Foundation, which so far has been undermined by a lack of Congressional funding. Hamburg supports it, recognizing that the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention have benefitted from their ability to tap outside resources to support important projects. As a regulatory agency, FDA is "a little bit different," according to Hamburg, and needs clearly defined standards and guidelines to avoid conflicts of interest. But Reagan-Udall, she believes, could be valuable in providing access to new research.

Credible, well-funded regulatory science is equally critical to improving readiness for the next pandemic flu crisis. FDA has taken some of the blame for delays in providing an effective H1N1 vaccine this fall, and Hamburg admits to being frustrated that the production process was not smoother. Still, she prefers to regard the response from FDA and industry as "quite remarkable in terms of how much has been mobilized in response to a previously unrecognized strain of flu virus."