What We Learned at the FDA Public Meeting On PDUFA V Reauthorization

Yesterday, we attended the FDA's public meeting on the reauthorizaton of PDUFA V. Patients, consumer advocates, healthcare professionals, and industry groups all had a chance to give their perspectives on the proposed changes to PDUFA, while audience members contributed to the conversation during an open comment period.

At its heart, PDUFA V is about ensuring that patients have access to new medicines as quickly and as safely as possible. Marc Boutin, of the National Health Council, gave us a patient perspective on what the three major priorities of PDUFA V should be. First, there should be a structured, transparent framework to objectively assess risk. Patients need to be able to understand how the risks and benefits of a treatment are being evaluated. Secondly, the time frame of patient access to a new treatment needs to be accelerated. It currently takes nearly 15 years and approximately a billion dollars to get a new drug from discovery, through clinical trials and FDA review, and finally to market. Finally, the FDA ought to encourage the development of treatments for rare diseases by making the drug review process more flexible and increasing collaboration between industry, patients, and the FDA.

Patients rely on the FDA's decisions about the safety and efficacy of drugs, and PDUFA is a sound investment in making that decision process transparent, objective, and effective. Both consumer advocates and healthcare professionals emphasized that because the FDA has been underfunded for years, prescription drug user fees are essential to helping the FDA do its job. They also recommended that the FDA make an investment in new technologies that would modernize and streamline the review process, helping to ensure safety and monitor effectiveness.

One sign that the FDA drug review process has room to improve is its approval rate. Although 80% of applications submitted are ultimately approved, about half of those are not approved on their first submission. Industry contributors stressed that promoting strong scientific dialogue between the FDA and drugmakers throughout the drug development process would simplify the approval process and encourage innovation. By increasing timely, substantive communication between the FDA and biopharmaceutical companies, PDUFA V would promote wiser, more efficient utilization of the FDA's limited resources.

Throughout the open comment period, audience members stressed again and again that PDUFA is a critical tool in bringing innovative medications to patients as quickly and efficiently as possible. Several advocates from HIV advocacy groups noted that PDUFA has been especially helpful to patients with HIV; access to treatment is not only critical for the health of the patient, but is an important preventive public health measure as well. PDUFA has helped usher in over 100 innovative treatments for HIV, and as a result, has changed an HIV diagnosis from a death sentence into a survivable condition.

The meeting ended on the same point with which it began: ultimately, PDUFA is about patients. Because PDUFA V promotes an FDA process that is transparent, scientifically-based, and timely, it helps turn basic discoveries into medical breakthroughs. That is why we continue to urge Congress to reauthorize a clean PDUFA, one that gives patients access to innovative treatments and cures as safely and efficiently as possible.