Keeping the End in Mind: Reviewing U.S. FDA Inspections of Submissions including Real-World Data.

Abstract

The increasing use of real-world data (RWD) to generate real-world evidence (RWE) presents unique opportunities and challenges for drug development and regulatory decision-making, particularly in the area of good clinical practice inspections. FDA typically focuses their application review-based inspections on pivotal studies that generate evidence submitted to support new drug and biological product applications. This focus applies regardless of the data sources used in those studies. In this article, we discuss the fundamental role of good clinical practice inspections in verifying the quality, integrity, and reliability of RWD used in regulatory submissions. Through case examples, we highlight specific challenges related to accessing RWD source records, assessing data quality, and evaluating processes for data curation, transformation, and analysis. Our experience underscores the importance of early engagement with regulatory agencies as well as the implementation of robust quality management practices throughout the study lifecycle. As RWD continues to shape the regulatory landscape, these case examples provide insights in navigating the complexities associated with submissions utilizing RWE for drug approval.