A Case Study Assessment on the Rationale for, and Relevance of, Non-Core Protocol Data

Abstract

To better understand the nature of Non-Core procedures and derive new insight into protocol simplification and optimization, Tufts CSDD collaborated with the FDA and sponsor companies to assess alignment on the rationale for collecting, and relevance of, Non-Core protocol data. Twelve sponsor companies classified and rated 700 distinct procedures from 19 pivotal trials supporting new drug and biologics approvals. FDA reviewers classified and rated 80 distinct procedures for three of the 19 pivotal trials. The results of this assessment indicate areas of alignment and misalignment. Sponsors and FDA reviewers agreed on the classification for more than half of endpoints. However, FDA reviewers classified a much higher percentage of procedures as Non-Core (26% vs. 18%) with the largest proportion (50%) of these procedures perceived as Core by sponsor companies. Sponsors indicated that one-out-of-six Non-Core procedures were administered due to perceived regulatory requirement and expectation. The results of this study characterize the challenge in aligning the different-and potentially conflicting-imperatives of sponsors and regulators and speak to the importance of more effective FDA-sponsor communication to help simplify protocol designs.

Keywords: Non-Core procedures; Protocol complexity; Protocol optimization.