Real-World Evidence Use in Japanese HTA: Insights From Manufacturer Submissions and Public Analyses (2019-2025)
Author(s)
Grace Kiyabu, PhD, MPH1, Radoslaw Skowron, M.Sc.2, Mariko Nomoto, MBA3, Yoshie Onishi, DrPH, RPh3, Renata Majewska, MSc2, Clement Francois, MSc, PhD4, Shunya Ikeda, MSc, PhD, MD5.
1Putnam, San Francisco, CA, USA, 2Putnam, Krakow, Poland, 3Putnam, Tokyo, Japan, 4Putnam, Paris, France, 5International University of Health and Welfare, Ōtawara, Japan.
1Putnam, San Francisco, CA, USA, 2Putnam, Krakow, Poland, 3Putnam, Tokyo, Japan, 4Putnam, Paris, France, 5International University of Health and Welfare, Ōtawara, Japan.
OBJECTIVES: This study evaluates the use of real-world evidence (RWE) in Health Technology Assessments (HTAs) conducted by Japan’s Center for Outcomes Research and Economic Evaluation for Health (C2H), comparing utilization between manufacturer submissions and public analyses.
METHODS: All HTAs published on the C2H website between April 2019 and March 2025 with a “completed” status were reviewed. HTAs that were suspended, categorized as H5, or in progress were excluded. Publicly available documents were analysed to assess the presence and type of RWE used by manufacturers and/or public analysis groups. The types of data sourced (e.g., healthcare resource utilization [HCRU], costs, epidemiology), databases used, and the degree of RWE adoption in public reviews were documented.
RESULTS: A total of 31 completed HTAs were identified. Of these, 16 used RWE in company submissions, 14 in public analyses, and 8 in both. For 10 assessments, company submission details were not publicly available. Infectious diseases (n=3) and endocrinology (n=3) were the most common therapeutic areas using RWE. Manufacturers primarily used RWE for cost and HCRU inputs (n=12), followed by epidemiology plus cost/HCRU (n=3). Public analyses similarly relied on RWE for cost and HCRU (n=10), with limited use of epidemiological data alone (n=2). Manufacturers mostly used commercial claims databases, while public analyses consistently relied on the National Database (NDB). Nearly half of RWE from manufacturers was reanalysed by public reviewers.
CONCLUSIONS: Approximately half of completed HTAs in Japan incorporated RWE, with comparable frequency across manufacturer and public analyses. However, divergent data source preferences and frequent public reanalysis suggest the importance of aligning RWE strategies with local expectations. Early integration of NDB-compatible data may strengthen submission credibility in Japan’s HTA process.
METHODS: All HTAs published on the C2H website between April 2019 and March 2025 with a “completed” status were reviewed. HTAs that were suspended, categorized as H5, or in progress were excluded. Publicly available documents were analysed to assess the presence and type of RWE used by manufacturers and/or public analysis groups. The types of data sourced (e.g., healthcare resource utilization [HCRU], costs, epidemiology), databases used, and the degree of RWE adoption in public reviews were documented.
RESULTS: A total of 31 completed HTAs were identified. Of these, 16 used RWE in company submissions, 14 in public analyses, and 8 in both. For 10 assessments, company submission details were not publicly available. Infectious diseases (n=3) and endocrinology (n=3) were the most common therapeutic areas using RWE. Manufacturers primarily used RWE for cost and HCRU inputs (n=12), followed by epidemiology plus cost/HCRU (n=3). Public analyses similarly relied on RWE for cost and HCRU (n=10), with limited use of epidemiological data alone (n=2). Manufacturers mostly used commercial claims databases, while public analyses consistently relied on the National Database (NDB). Nearly half of RWE from manufacturers was reanalysed by public reviewers.
CONCLUSIONS: Approximately half of completed HTAs in Japan incorporated RWE, with comparable frequency across manufacturer and public analyses. However, divergent data source preferences and frequent public reanalysis suggest the importance of aligning RWE strategies with local expectations. Early integration of NDB-compatible data may strengthen submission credibility in Japan’s HTA process.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD297
Topic Subcategory
Health & Insurance Records Systems
Disease
No Additional Disease & Conditions/Specialized Treatment Areas