Acceptance of Real-World Evidence in HTA in Japan: Is the Potential Being Realized?
Author(s)
Yan Ran Wee, BSocSci, MSc1, Natsumi Fujita, BSc1, Katherine Massey, PhD1, Jennifer Sara Evans, BSc, PhD2.
1Costello Medical, Singapore, Singapore, 2
1Costello Medical, Singapore, Singapore, 2
OBJECTIVES: To understand the use and acceptance of real-world evidence (RWE) within the health technology assessment (HTA) process in Japan.
METHODS: We reviewed cost-effectiveness evaluation reports (in English or Japanese) for pharmaceutical products published on the Center for Outcomes Research and Economic Evaluation for Health (C2H) website (https://c2h.niph.go.jp/results/item.html) between 2019-25th March 2025. Details of RWE and its use in the evaluation were extracted from the reports, along with any discussion/critique by the Academic Technology Assessment Group (ATAG).
RESULTS: Out of 37 drug evaluation reports, the majority (n=32, 86.5%) included some discussion of RWE. RWE was presented as evidence for additional (efficacy) benefits in six evaluations, but was critiqued by the ATAG due to issues with selection bias or study design. In one evaluation, where data were not yet available from an interventional trial, the claim of additional benefit was accepted based on observational studies. RWE was most often utilised within the economic analysis (n=19), to inform Japan-specific population inputs like patient characteristics (n=17) and healthcare resource use (n=17). This use case was generally accepted; in many evaluations, database studies were also used by the ATAG to validate manufacturer estimates. However, ATAG acceptance of RWE to inform OS extrapolations or mortality estimates (n=6) or utility values (n=8) was mixed, with concerns around local generalisability and high uncertainty of estimates.
CONCLUSIONS: While the majority of HTAs in Japan report RWE use in the decision-making process, acceptability by the ATAG varied based on the use case. Use of database analyses to inform patient characteristics and economic analysis inputs was common and accepted, likely reflecting Japan’s mature health data landscape. However, concerns with methodological rigour limited the acceptability of RWE in supporting clinical effectiveness. Ensuring rigour and transparency in RWE generation is paramount to increase its value and acceptance for demonstrating comparative effectiveness in HTA.
METHODS: We reviewed cost-effectiveness evaluation reports (in English or Japanese) for pharmaceutical products published on the Center for Outcomes Research and Economic Evaluation for Health (C2H) website (https://c2h.niph.go.jp/results/item.html) between 2019-25th March 2025. Details of RWE and its use in the evaluation were extracted from the reports, along with any discussion/critique by the Academic Technology Assessment Group (ATAG).
RESULTS: Out of 37 drug evaluation reports, the majority (n=32, 86.5%) included some discussion of RWE. RWE was presented as evidence for additional (efficacy) benefits in six evaluations, but was critiqued by the ATAG due to issues with selection bias or study design. In one evaluation, where data were not yet available from an interventional trial, the claim of additional benefit was accepted based on observational studies. RWE was most often utilised within the economic analysis (n=19), to inform Japan-specific population inputs like patient characteristics (n=17) and healthcare resource use (n=17). This use case was generally accepted; in many evaluations, database studies were also used by the ATAG to validate manufacturer estimates. However, ATAG acceptance of RWE to inform OS extrapolations or mortality estimates (n=6) or utility values (n=8) was mixed, with concerns around local generalisability and high uncertainty of estimates.
CONCLUSIONS: While the majority of HTAs in Japan report RWE use in the decision-making process, acceptability by the ATAG varied based on the use case. Use of database analyses to inform patient characteristics and economic analysis inputs was common and accepted, likely reflecting Japan’s mature health data landscape. However, concerns with methodological rigour limited the acceptability of RWE in supporting clinical effectiveness. Ensuring rigour and transparency in RWE generation is paramount to increase its value and acceptance for demonstrating comparative effectiveness in HTA.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD24
Topic Subcategory
Data Protection, Integrity, & Quality Assurance
Disease
No Additional Disease & Conditions/Specialized Treatment Areas