PDUFA V: Enhancing Benefit/Risk Analysis at the FDA

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With the fifth reauthorization of the Prescription Drug User Fee Agreement (PDUFA V) one and half years underway, we welcome Dr. Rob Metcalf back to the CMM platform for a second blog in our series exploring how PDUFA V accelerates patient access to medicines. Dr. Metcalf is Vice President of Global Regulatory Affairs at Eli Lilly and Company and a member of the FasterCures Benefit Risk Advisory Council, which provides recommendations to expand patient input in shaping new medicine development and influencing regulatory decisions.

As I've written previously for the Campaign for Modern Medicines, PDUFA has helped the FDA streamline the new medicine approval process -- bringing new treatments to patients faster. The fifth reauthorization of PDUFA helped stimulate widespread patient advocacy engagement in FDA's regulatory processes in ways that haven't been seen over the past two decades. This is best demonstrated through two related key initiatives included in PDUFA V: the Patient-Focused Drug Development Initiative and Benefit-Risk Assessment. Both these efforts have emerged as promising and important initiatives for patients and developers of new medicines.

Incorporating the patient voice in benefit/risk assessment. Patient-focused drug development (PFDD) gives patients the opportunity to share their experiences directly with the FDA, helping the agency gain a more holistic and patient-centered view of the pros and cons of a potential medicine and the realities of living with a particular disease. Its goal is to provide the agency with a systematic method for obtaining patients' perspectives on the severity of a condition and available treatment options.

The FDA has committed to convening twenty public meetings each year throughout the course of PDUFA V. Each meeting will focus on a different disease area. So far, the FDA has held eight PFDD meetings and plans to hold twelve more meetings over the next three years. The product of each meeting is a "Voice of the Patient" report that summarizes what the FDA heard in the meeting.

Broad implementation of a benefit/risk framework in new drug reviews. PDUFA V also prompted the agency to advance wider implementation of a structured approach to benefit/risk assessment that could both serve as a template for product reviews as well as a vehicle for further explaining the basis for FDA's regulatory decisions. The assessment contains five decision factors, including an analysis of the condition and current treatment options. The levels of consideration outline what is known and unknown - as well as the conclusions that can be drawn. This all feeds into the bottom-line summary assessment - which succinctly sums up the decision and the factors which led to it.

The FDA continues to gather more information from patients and is phasing in its use of the new framework- with full implementation of all new drug applications by 2017. As mentioned last month, measuring benefit and risk is at the heart of every single regulatory decision made by the agency. The implementation of these two important programs, resulting in a well-informed structured benefit/risk assessment, gives the agency another tool as they review new drug applications.

Next month we'll take a look at how this important program is being implemented and what opportunities there might be for ongoing improvement to further benefit and engage patients in the benefit-risk assessment process for new medicines.