(B-077) Evolving Landscape of Post-Marketing Surveillance (PMS) Studies in Japan Utilising Secondary Real-World Data (RWD)

Background: PMS studies are conducted to monitor the safety and effectiveness of a drug in real-world settings and have traditionally relied on primary data collection methods in Japan. Recently, Japan has recognised the importance of RWD and followed EMA and FDA in promoting secondary data sources including for research for regulatory submission.

Objectives: We describe the evolving landscape related to RWD use for PMS studies in Japan and discuss the implications compared to global practices.

Methods: We reviewed the guideline from the Pharmaceuticals and Medical Devices Agency (PMDA); latest guidance from the Japan Pharmaceutical Manufacturer Association (JPMA) on incorporating secondary RWD in PMS; and completed/ongoing PMS studies using secondary RWD.

Results: PMDA issued a 2014 guideline on pharmacoepidemiology research for drug safety assessment using secondary data sources. Following the 2017 Ministerial Ordinance on Good Post-marketing Study Practice revised to include secondary RWD in PMS studies, a PMS study has been completed using MID-NET, a trusted database for conducting PMS studies which was developed by PMDA, and several ongoing PMS studies use secondary patient-level databases, including Medical Database Vision. In 2024, JPMA issued guidance on secondary RWD use for PMS studies, providing standardised methods for implementing and reporting PMS studies including instructions regarding feasibility assessment and tools for secondary data sources assessment, similar to NICE’s Data Suitability Assessment Tool (DataSAT). The JPMA guidance describes a practical approach to using RWD in PMS for regulatory decision-making, which differs from the more general PMDA guideline.

Conclusions: PMDA and JPMA endorse using secondary RWD for PMS studies and provide clear guidance. PMS studies using secondary RWD are expected to comply with these to ensure efficiency and scientific rigour. These developments align with global trends in utilising RWD for regulatory decisions, including those intended for submission to authorities such as for EMA and FDA.