EVOLVING USE AND ACCEPTANCE OF REAL-WORLD EVIDENCE WITHIN HTA IN JAPAN BY MANUFACTURERS AND THE ATAG: INSIGHTS FROM C2H REPORTS
Author(s)
Yan Ran Wee, BSocSci, MSc, Natsumi Fujita, BSc, Naho Kaburaki, MSc, Jennifer Sara Evans, BSc, PhD.
Costello Medical Singapore Pte Ltd, Singapore, Singapore.
Costello Medical Singapore Pte Ltd, Singapore, Singapore.
OBJECTIVES: To investigate trends in the use and acceptance of real-world evidence (RWE) within the health technology assessment (HTA) process of Japan’s Center for Outcomes Research and Economic Evaluation for Health (C2H), based on appraisals from 2019-2026.
METHODS: HTA reports for pharmaceutical products published on the C2H website up to 31st March 2026 were reviewed. Details of RWE use by manufacturers and the Academic Technology Assessment Group (ATAG), including evidence type (effectiveness, healthcare resource use [HCRU], costs, quality of life [QoL]) were extracted, along with discussion/critique by the ATAG.
RESULTS: Of 46 HTA reports, the majority (n=41, 89.1%) included RWE, most commonly in economic modelling (n=28/46) to inform model structure or Japan-specific epidemiological inputs. RWE use in modelling was generally accepted, with >60% reports describing RWE use by the ATAG themselves (e.g. National Database [NDB] analyses to validate manufacturer estimates). RWE (e.g. JMDC database analyses) was also a common source for costs and HCRU estimates (manufacturers n=18; ATAG n=13). Citing RWE for effectiveness was less common (n=9, of which n=6 were inclusions of RWE in indirect treatment comparisons by manufacturers). The ATAG rarely accepted effectiveness claims based on RWE, highlighting methodological limitations (n=3 acceptances due to limited alternative data). In n=13 reports describing real-world QoL surveys, ATAG critique commonly focused on Japanese population applicability.
CONCLUSIONS: RWE use in economic modelling is becoming standard in Japanese HTA, with manufacturers frequently leveraging RWE to inform model structure and inputs, but less commonly for effectiveness and QoL. The ATAG perspective appeared more critical, with manufacturer RWE critiqued for methodological and local applicability concerns, especially for effectiveness/QoL, though NDB was frequently used by the ATAG to re-analyse manufacturer estimates. This inconsistency suggests a need for guidance regarding standards/processes for RWE use in Japan HTA submissions, to improve alignment and maximise the potential of RWE for reimbursement decision-making.
METHODS: HTA reports for pharmaceutical products published on the C2H website up to 31st March 2026 were reviewed. Details of RWE use by manufacturers and the Academic Technology Assessment Group (ATAG), including evidence type (effectiveness, healthcare resource use [HCRU], costs, quality of life [QoL]) were extracted, along with discussion/critique by the ATAG.
RESULTS: Of 46 HTA reports, the majority (n=41, 89.1%) included RWE, most commonly in economic modelling (n=28/46) to inform model structure or Japan-specific epidemiological inputs. RWE use in modelling was generally accepted, with >60% reports describing RWE use by the ATAG themselves (e.g. National Database [NDB] analyses to validate manufacturer estimates). RWE (e.g. JMDC database analyses) was also a common source for costs and HCRU estimates (manufacturers n=18; ATAG n=13). Citing RWE for effectiveness was less common (n=9, of which n=6 were inclusions of RWE in indirect treatment comparisons by manufacturers). The ATAG rarely accepted effectiveness claims based on RWE, highlighting methodological limitations (n=3 acceptances due to limited alternative data). In n=13 reports describing real-world QoL surveys, ATAG critique commonly focused on Japanese population applicability.
CONCLUSIONS: RWE use in economic modelling is becoming standard in Japanese HTA, with manufacturers frequently leveraging RWE to inform model structure and inputs, but less commonly for effectiveness and QoL. The ATAG perspective appeared more critical, with manufacturer RWE critiqued for methodological and local applicability concerns, especially for effectiveness/QoL, though NDB was frequently used by the ATAG to re-analyse manufacturer estimates. This inconsistency suggests a need for guidance regarding standards/processes for RWE use in Japan HTA submissions, to improve alignment and maximise the potential of RWE for reimbursement decision-making.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
HTA43
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas