A SYSTEMATIC REVIEW OF REAL-WORLD EVIDENCE IN HEALTH TECHNOLOGY ASSESSMENTS FOR MULTIPLE MYELOMA IN EUROPE
Author(s)
BLANCA GROS OTERO, MSc1, Giselle Lostaunau Costa, M.D.2, Christina Wennerström, MSc, PhD3, Khavya Ramachandran, M.Sc.4, Francesca Galea Hales, BSc, MSc, PhD5.
1Johnson & Johnson, MADRID, Spain, 2Johnson & Johnson, Madrid, Spain, 3Johnson & Johnson, Solna, Sweden, 4IQVIA, London, United Kingdom, 5Johnson & Johnson, Colchester, United Kingdom.
1Johnson & Johnson, MADRID, Spain, 2Johnson & Johnson, Madrid, Spain, 3Johnson & Johnson, Solna, Sweden, 4IQVIA, London, United Kingdom, 5Johnson & Johnson, Colchester, United Kingdom.
OBJECTIVES: Real-world evidence (RWE) is increasingly referenced in European health technology assessment (HTA) submissions for multiple myeloma (MM). This study assessed the acceptability of MM-specific RWE in European HTAs and identified the principal methodological and contextual drivers and barriers.
METHODS: A systematic review of publicly available MM HTA reports was conducted across 11 European HTA bodies between 2021-2025: AEMPS-Spain, CRM/CTG-Belgium, Medicinradet-Denmark, FIMEA-Finland, HAS-France, IQWiG/G-BA-Germany, NICE-England, NoMA-Norway, SMC-Scotland, TLV-Sweden, and ZIN-Netherlands. Frequencies reflect each appearance of an RWE study in HTA reports; identical sources were counted again if submitted/resubmitted to different HTAs. Acceptability was categorized as: accepted, accepted with adjustments, accepted despite uncertainties, or not accepted. Descriptive analyses were performed overall and by HTA body, by evidence gap covered, and RWD source type.
RESULTS: 324 RWE studies were identified across HTA reports. The most common use cases were target-population sizing (N=114), control arms (N=64), contextualizing effectiveness/safety (N=56), and burden-of-illness (N=31); England accepted the widest range of RWE use-cases (12) versus Germany, Belgium and Spain (3, 3 and 2 respectively). RWD sources were mainly disease registries (N=101) and electronic health records (N=100). Across all submissions 66% were accepted, 1% accepted with adjustments, 15% accepted despite uncertainty, and 18% not accepted, varying from 100% in Belgium/Denmark to 63%/67% in the Netherlands/Sweden. Acceptance depended primarily on RWE use case and data source and independent of IPD access: noncomparative objectives were more often accepted (93%) than comparative effectiveness (34%); prospective observational studies had the lowest acceptance (56%), while early access datasets reached 100%.
CONCLUSIONS: RWE was frequently submitted but acceptability varied by HTA body and depended on by evidence gap covered and data source type.
METHODS: A systematic review of publicly available MM HTA reports was conducted across 11 European HTA bodies between 2021-2025: AEMPS-Spain, CRM/CTG-Belgium, Medicinradet-Denmark, FIMEA-Finland, HAS-France, IQWiG/G-BA-Germany, NICE-England, NoMA-Norway, SMC-Scotland, TLV-Sweden, and ZIN-Netherlands. Frequencies reflect each appearance of an RWE study in HTA reports; identical sources were counted again if submitted/resubmitted to different HTAs. Acceptability was categorized as: accepted, accepted with adjustments, accepted despite uncertainties, or not accepted. Descriptive analyses were performed overall and by HTA body, by evidence gap covered, and RWD source type.
RESULTS: 324 RWE studies were identified across HTA reports. The most common use cases were target-population sizing (N=114), control arms (N=64), contextualizing effectiveness/safety (N=56), and burden-of-illness (N=31); England accepted the widest range of RWE use-cases (12) versus Germany, Belgium and Spain (3, 3 and 2 respectively). RWD sources were mainly disease registries (N=101) and electronic health records (N=100). Across all submissions 66% were accepted, 1% accepted with adjustments, 15% accepted despite uncertainty, and 18% not accepted, varying from 100% in Belgium/Denmark to 63%/67% in the Netherlands/Sweden. Acceptance depended primarily on RWE use case and data source and independent of IPD access: noncomparative objectives were more often accepted (93%) than comparative effectiveness (34%); prospective observational studies had the lowest acceptance (56%), while early access datasets reached 100%.
CONCLUSIONS: RWE was frequently submitted but acceptability varied by HTA body and depended on by evidence gap covered and data source type.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PT30
Topic
Health Technology Assessment, Organizational Practices, Real World Data & Information Systems
Disease
Oncology