US Food and Drug Administration Commissioner Dr. Margaret Hamburg reflected on her challenging job at a May 8 meeting of the Alliance for a Stronger FDA. “We have to earn the confidence and trust of the public every day,” she said, citing the agency’s need to improve processes – streamline and modernize – while maintaining standards of safety and efficacy for the products FDA regulates. She said the FDA wants to be viewed as an “honest broker” and a responsible steward of the resources entrusted to it.
FDA is, and must continue to be, a science-based, data-driven agency. “The quality of our work and our ability to navigate the complex landscape we are responsible for depends on this,” Hamburg said. Responding to Alliance President (and FasterCures Executive Director) Margaret Anderson, who asked about common misconceptions about FDA, Hamburg expressed surprise that other entities, including government agencies, may not see FDA as a science-based agency. FDA is conducting cutting-edge research in-house that is helping people, according to Hamburg.
Hamburg said that the FDA will continue to focus on its regulatory science initiatives as part of its efforts to streamline and modernize the product approval process. Regulatory science is the science of developing new tools, standards, and approaches to assess safety, efficacy, quality, and performance of regulated products. She called it a highly collaborative effort and said that initiatives will be embedded in program elements throughout the agency – “in the texture of the work we do.”
The “Big Data” phenomenon is becoming part of the conversation at the FDA, according to Hamburg. FDA owns huge stores of data and has identified a need to develop the science of how to do meta-analysis to mine this information. The next frontier is data sharing, she said. Anderson pointed out that currently FDA cannot disclose specifics around new drug applications, so information that might provide valuable insight is unavailable. “We need to be able to find a way to open data about failed trials, subpopulations of responders, side-effects, and so on, but it has to be done thoughtfully,” Hamburg said.
FDA also has to be a global agency, she said. The world has changed since FDA’s original authorizations were drafted, and this requires some changes in the way FDA conducts business. The ability to partner with sister regulatory agencies globally is critical, particularly in the area of legal and regulatory harmonization. “We have an opportunity to provide leadership worldwide,” she said. Raising standards in the international community – particularly in developing countries – benefits everyone.
Anderson asked Hamburg to describe how FDA is aided by interacting with the patient advocacy community. “Patients bring focus on the real world in which products [regulated by FDA] are used,” Hamburg said, “and we get critical input from patients and stakeholders on the risk-benefit calculus.” The value of benefit balanced by risk is not an absolute. It has a societal valuation, and the same judgments about risk-benefit cannot be applied across all situations.
FDA has also learned valuable lessons from the HIV/AIDS story, Hamburg said. For example, FDA is thinking about how to expand the accelerated approval mechanism to broader domains, and make it a stronger tool. FDA also recognizes the importance of patient involvement in clinical studies and is looking for ways to push clinical research closer to patients in their own communities.
With FDA’s vast and diverse set of responsibilities, one of the most challenging aspects of Hamburg’s job is coping with an increasingly tense budget situation. Identifying core responsibilities and looking at them in the context of the current budgetary reality is key. “We have to think about positioning and building for the next five to ten years – not just for the next fiscal year,” Hamburg said.
Asked about the specter of sequestration, Hamburg said she hopes for a better solution, but this is exactly why it is important to identify priorities. “We may have to step away from certain activities that we value in favor of programs that may have more impact,” she said, while noting that there is a need to protect non-user fee programs (for example, the “food side,” which does not collect user fees). Hamburg left the group with a final thought: “It is important to remember that in terms of return on investment, FDA is extraordinary.”