As the House of Representatives voted to approve legislation that helps move drugs through the pipeline this afternoon, Margaret Hamburg, the commissioner of the US Food and Drug Administration (FDA) praised the bill’s revised approach to risk management.
Hamburg sat on a panel with leaders in biotechnology and patient advocacy at the 2012 BIO International Convention this week in Boston, Massachusetts. Prompted by questions from the moderator, the five panelists discussed the revision of the Prescription Drug User Fee Act (PDUFA), which reauthorizes the FDA’s programme to grant companies faster drug and medical-device approval in exchange for money.
The bill, PDUFA V, has received widespread support in Congress thus far. As long as the Senate approves the version that the House voted on today, President Obama will sign it into law.
What follows is an edited version of the panel’s discussion, moderated by Steve Usdin, editor at BioCentury, at BIO today.
Q: How is PDUFA V remarkably different?
Marc Boutin, chief executive of the patient-advocacy group National Health Council: If you look forward five years, people will see PDUFA V as a turning point. At the heart of the issue is a respect for what the patient wants. We said we want treatments quickly and we want a clear framework for addressing risks and benefits.
Hamburg: With respect to the engagement of patients, we know how much that matters. I cut my teeth as a young doctor and researcher as the AIDS epidemic emerged, and I worked at the NIAID [US National Institute of Allergies and Infectious Diseases] on AIDS research and policy in those early days when patient advocates, health-care providers, researchers and industry all sat around a table and thought about solutions. The patient community not only conveyed a sense of urgency, but also deepened our understanding of what mattered in terms of risks and benefits.
Q: How is risk treated in PDUFA V?
Hamburg: We all know that no drug is absolutely safe or absolutely effective. It’s all relative in terms of how drugs compare with one another, and in the context of risks versus benefits. There has been an apparent evolution in the thinking on safety and efficacy. We have seen a shift in the expectation that an FDA approval means that something is absolutely safe. Instead, safety is relative to benefits.
Jonathan Leff, managing director, Healthcare, Warburg Pincus investment firm: It’s my belief and hope that when we ask about the most important thing in PDUFA V, it will be this risk–benefit framework. There’s just a paragraph in the legislation that tells the agency to develop a framework. That was something that the agency had already committed to do, but I urged them to put this in the bill itself. I said I want to see this codified in Congress’s own words in the legislation. Congress, as well as the media and the public, has this history of boiling down the issue to whether drugs are safe or not safe. But it’s benefit versus risk. I hope that this little paragraph leads to a more intelligent dialogue about what drugs are meant to do.
Boutin: I think risk–benefit analyses now happen in the minds of [FDA] reviewers but their thinking is not communicated, and we don’t know if their judgments are aligned with the patients who take that medicine. We need a framework to communicate how the decision was made in a uniform way. And I’m happy to say that’s already begun to happen. The FDA has already planned meetings with patient-advocacy societies. This truly is a watershed moment.