Navigating Non-interventional Post-authorization Studies in East Asia: Regulatory Challenges, Opportunities, and Future Directions.

Abstract

Post-authorization studies (PAS) are often mandated by regulatory authorities as a condition of marketing authorization of pharmaceutical products. This article explores specific regulations and trends in China, Japan, and South Korea, highlighting the scientific and operational limitations that such PAS pose to the stakeholders in these regions including significant variations in regulatory requirements. Pharmacovigilance guidelines and publications on regional regulatory trends were reviewed. Active surveillance studies are widely adopted to fulfill post-authorization requirements in East Asia countries. These are primary data collection studies, i.e., traditional site-based studies that monitor the frequency of all adverse events (and clinical outcomes when requested) of the newly approved pharmaceutical product during a predefined treatment period. Such studies generally present limitations regarding the product's safety profile characterization, including the absence of a comparator group, selection bias, limited sample size, and considerable resources needed to conduct the studies. These limitations explain the trend toward hypothesis testing studies, conducted with secondary data (e.g., large electronic database studies) as preferred over traditional active surveillance studies. Harmonizing regulatory approaches and enhancing access to comprehensive data sources are critical for generating fit-for-purpose evidence to support regulatory decision making in these regions. Therefore, we propose a decision tool to assist with the planning of PAS in China, Japan, and South Korea. This article is endorsed by the International Society for Pharmacoepidemiology (ISPE).