“Through its Advancing Medical Innovation effort—led by former Senate Majority Leader Bill Frist, MD and former Congressman Bart Gordon—the Bipartisan Policy Center (BPC) is advancing policies that promote medical innovation and reduce the time and cost associated with the discovery, development, and delivery of safe and effective medical products for patients in the United States.
“BPC commends the FDA for recognizing—within the PDUFA VI commitment letter—the need for reforms in the following areas, which were included in BPC’s July 2015 report, Advancing Medical Innovation for a Healthier America.
- Incorporating the Patient’s Voice in Drug Development and Decision-making. The FDA will strengthen staff capacity and develop a series of guidance documents on approaches and methods for collecting meaningful patient and caregiver input to inform drug development and regulatory decisions.
- Enhancing Use of Real-World Evidence for Use in Regulatory Decision-making. The FDA will conduct public workshops to gather input on real-world evidence, including benefits; challenges associated with availability, quality, and access, as well as ways to address them; and methodological approaches. It will also publish guidance on how real-world evidence can contribute to the assessment of safety and effectiveness in regulatory submissions.
- Improving the Review of Combination Products. The FDA will enhance staff capacity and capabilities across centers to more efficiently, effectively, and consistently review drug and device-led combination products. It will also streamline processes for combination product review and establish new performance goals.
- Enhancing Benefit-Risk Assessment in Regulatory Decision-making. The FDA will further its implementation of structured benefit-risk assessment, including incorporation of the patient voice in regulatory decision-making, by updating its implementation plan, conducting an evaluation of the implementation of the benefit-risk framework, developing guidance, and revising policies and procedures to include new approaches.
- Enhancing Drug Development Tools Qualification Pathway for Biomarkers. The FDA will convene a public meeting on biomarkers used in drug development; develop guidance on biomarker taxonomy, contexts of uses, and general evidentiary standards; and improve transparency of biomarker qualification submissions and decisions.
- Improving FDA Hiring and Retention of Review Staff. The FDA will implement corporate recruiting practices, augment hiring capacity with expert contractor support, complete establishment of a dedicated function to ensure needed scientific staffing for drug review, and conduct a comprehensive and continuous assessment of hiring and retention performance.”