SW-2A - Real-World Evidence in Regulatory Decision-Making for Medical Products

Background: FDA centers that regulate medical products have robust programs that evaluate the use of real-world data (RWD) to generate real-world evidence (RWE) for regulatory decision-making. Cross-cutting Agency initiatives seek to enhance opportunities for generating evidence on medical product safety and effectiveness, evaluating innovative study designs for patient-centric evidence generation, and advancing regulatory science through RWE demonstration projects.

Objectives: 1. Review ongoing FDA RWE-related activities.
2. Describe how FDA evaluates whether RWD are fit-for-use, study designs provide adequate scientific evidence, and study conduct meets regulatory requirements.
3. Summarize key messages from FDA RWE guidance documents.
4. Explain how FDA-supported RWE demonstration projects have addressed gaps in knowledge across product centers to support medical product development.

This session represents a forum wherein activities and accomplishments of FDA’s RWE programs for medical products can be shared with ISPE attendees. The session plans to discuss topics of broad interest to ISPE membership, including RWE-related regulatory decisions, demonstration projects, new initiatives, and activities to advance RWE policy to support regulatory decisions.

Description: Marie Bradley will provide an overview of RWE-related activities conducted by CDER, CBER, OCE, and CDRH. Notable accomplishments since the passage of the 21st Century Cures Act will be highlighted. For example, the CDER-CBER-OCE RWE Program for drugs and biological products has engaged external interested parties, published a “first generation” of guidance documents to advance policy on how real-world data (RWD) can be used to generate RWE in support of the Agency’s regulatory decisions, supported RWE-related demonstration projects, and developed internal agency processes. In addition, a new Advancing RWE initiative was launched in 2023. CDER serves in a coordinating role for these efforts.
Brief presentations from cross center representatives: CDER (Gerald Dal Pan), CBER (Richard Forshee), OCE (Donna Rivera), and CDRH (Daniel Caños) will follow. Each speaker will provide additional information on current center-based RWE programs and initiatives, notable RWE-based product approvals, draft and final RWE guidance documents, and selected RWE-related demonstration projects. The symposium will conclude with a 30-minute panel discussion with all speakers. Marie Bradley (CDER) will moderate.