Insights into post-marketing clinical validation of companion diagnostics with reference to the FDA, EMA, PMDA, and MFDS

Abstract

Companion diagnostics are increasing clinical demand globally, regulatory frameworks for clinical validation are strengthening. Post-marketing verification is an important aspect of providing high-quality, personalized treatment to patients because it can ensure long-term safety and effectiveness, while also generating effective risk management and performance evidence. Certain compliance issues related to the requirements for post-marketing clinical trials can potentially impact manufacturers, so it is essential to have a clear understanding of the regulatory process. In this study, we perform an institutional comparison as well as a case analysis by country (U.S. Food & Drug Administration, European Medicines Agency, Pharmaceuticals and Medical Devices Agency, and Ministry of Food and Drug Safety) on the post-marketing safety and effectiveness of companion diagnostics. We collected guidelines and guidance documents published by each regulatory agency and Post-marketing research case analysis examined the data collection items as well as the materials or templates required to be submitted. The results indicate that there are institutional differences in the post-marketing surveillance activities carried out by different regulatory authorities, and the data required may vary accordingly. The findings of this study are expected to provide new insights that can support manufacturers and developers of companion diagnostics in securing evidence regarding post-marketing safety and effectiveness.

Keywords: clinical validation; companion diagnostics; effectiveness; post-marketing; safety.