Analysis and Impact of PDUFA V

As the House of Representatives voted unanimously to approve legislation to approve the Prescription Drug User Fee Act (PDUFA) V, industry leaders gathered at the 2012 BIO International Convention for a lively discussion on the changes the new legislation, focused on modernizing Food and Drug Administration regulations, will bring to the biotechnology industry. The super session panel discussion was moderated by Steve Usdin, Washington Editor of BioCentury and Co-Host of BioCentury This Week, and included Marc Boutin, Executive Director and Chief Operating Officer of the National Health Council; Peter S. Greenleaf, President of MedImmune; Margaret Hamburg, MD, Commissioner of the FDA, and Jonathan Leff, Managing Director, Healthcare at Warburg Pincus.

Usdin opened the discussion with a question about the House vote, asking the panelists what would be different two to four years from now. Greenleaf weighed in, stating that by year two, his hope is that we’ll have greater transparency and movement on patient initiatives. Leff, meanwhile, added that the good news is that we are in a better place, but the bad news is there’s a lot of work to do before the industry can translate science into cures. Boutin and Hamburg agreed that we were witnessing a “watershed” moment.

We are “positioned to do some terrific and urgently needed activities in a more effective, meaningful and responsive way,” Hamburg said.

Moving on to the accelerated approval provision in the legislation, all the panelists agreed the new pathway would lead to getting treatments into the hands of patients faster, in a responsible way. According to Leff, the accelerated approvals pathway will lead to a “tremendous amount of innovation.”

Each panelist also commented on the improved dialogue that took place leading up to the creation of PDUFA V, explaining that the conversations included all stakeholders — the industry, patient groups, consumer groups, investors and regulators. Adding patient and consumer groups to the mix ensured their voices were heard, Boutin noted. This was a new element that everyone agreed added immeasurable benefits to the process.

Key among those benefits: a frank discussion of benefits and risk around new medicines.  Boutin explained that that the previous PDUFA was focused on safety, but not necessarily on getting new medicines and treatments to patients. He added that the benefit-risk assessment is a personal judgment. PDUFA V is slated to include language stipulating that the benefit-risk judgment be clearly laid out for all new medicines

The panel closed with a look to the future, with a call to the industry to change the status quo. Leff pointed out that biopharma R&D production is in a steady decline, and urged the audience to come up with new ways to keep costs and development time down. Greanleaf added the industry needs to think about treatments versus prevention. He called on the industry to think more about vaccinations. Hamburg agreed, telling the audience that we need to advance science and approach problems with new thinking.

For more information on why PDUFA matters to patients, please see Marc Boutin’s post on BIOtechNOW.

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