Six‐Year Trends in Real‐World Data Use for Post‐Marketing Surveillance of New Medical Products in Japan

The Ministerial Ordinance on Good Post-Marketing Study Practice for Drugs was amended by the Ministry of Health, Labour and Welfare (MHLW) in 2018 to clearly define post-marketing database studies (DBS) as a measure of pharmacovigilance activities for approved medical products in Japan. This review describes and characterizes DBS in the landscape of post-marketing safety/effectiveness investigations of approved medical products in Japan for the past 6 years. New drugs and regenerative medical products approved by MHLW between January 1, 2019, and December 31, 2024, were included (N = 697). Of the total 572 planned post-marketing surveillances, 91 (15.9%) DBS were identified. The percentage of DBS initially decreased from 2019 to 2021 and then increased to 18.9% by 2024. Compared with other drugs, DBS were less likely planned for orphan (10.5%) and pediatric (7.4%) drugs. Control arms were used in 71.4% of DBS. The safety specifications evaluated in DBS using administrative data tended to focus on specific types of conditions, such as those related to the circulatory system. The findings suggest that DBS has begun to increase in recent years while highlighting challenges in accessing fit-for-purpose data and the need for appropriate pharmacoepidemiologic methods to support comparative evaluations. Access to large sample sizes provides DBS several advantages, including utility for evaluation of rare adverse events and the ability to establish a control group. Multi-sector and multi-stakeholder efforts may have the potential to further advance the utilization of real-world data in post-marketing benefit/risk investigations of medical products.