The Potential of Real-World Data (RWD) for Informing FDA Biosimliar and Interchangeable Biosimlar Regulatory Decisions: Recommendations From a Multi-Stakeholder Expert Panel

OBJECTIVES: U.S. Biosimilars are approved based on the totality of evidence, including molecular analysis and clinical studies. Our

METHODS: We engaged nine individuals with expertise in biosimilar development, regulatory science, policy, and RWD. The panel completed pre-meeting questionnaires providing views and strategies for RWD, and then met for two, two-hour, moderated virtual meetings to uncover where and how to integrate RWD into the U.S. biosimlar regulatory process. Discussion transcripts were summarized into recommendations for adopting RWD in the regulatory process.

RESULTS: Questionnaire responses demonstrated general optimism for using RWD in regulatory approvals citing past successes and continually growing pools of rich data, but also concern for the quality and relevance for meeting FDA needs. These views were echoed in the panel discussion where they coalesced around a set of recommendations to advance RWD by building on successes and overcoming uncertainty in operationalizing regulatory-grade RWD: 1) advocate for continued education for manufacturers and regulators on appropriate uses of RWD to facilitate approvals without compromising evidentiary standards for assessing safety and effectiveness;

CONCLUSIONS: While challenges remain in using RWD in the U.S. regulatory context, the panel provided actionable recommendations in advancing RWD that could meet evidentiary needs, emphasizing operational improvements, methods development, and data enrichment to build on past success.