Presentation (CTI)

OBJECTIVES: To systematically compare national requirements for the pharmacoeconomic evaluation and HTA analyses in a reimbursement process for orphan drugs across the European Economic Area (EEA) countries, United Kingdom (UK) countries and Switzerland based on direct institutional evidence.
METHODS: This study adopted a sequential mixed-methods design over a two-year timeline from 2022 to 2024 across EUnetHTA and INAHTA affiliated agencies. This design combines qualitative and quantitative methods to map orphan drug HTA requirements across the EEA, UK nations, and Switzerland. Phase I (2022-2023) gathered primary qualitative data via email inquiries, while Phase II (2024) verified document currency and addressed non-response via website audits. Qualitative content analysis and quantitative mapping integrated findings into absolute and relative specific requirements frequencies.
RESULTS: Marked methodological differences exist across jurisdictions. HTA is a statutory obligation for orphan drugs in 27 countries, though the majority apply modified criteria like elevated cost-effectiveness thresholds to accommodate rare disease data gaps. A separate specific HTA guidance for the assessment of orphan drugs exist only in 6 countries. There is no any HTA guidance in 6 countries. Full economic evaluation is mandatory in 12 jurisdictions in which only five countries mandate a full societal perspective, while Denmark requires a limited societal perspective excluding productivity losses. Most countries rigidly prioritize public payer or health system frameworks, treating wider societal impacts as secondary, while heterogeneity persists regarding indirect cost estimations.
CONCLUSIONS: European orphan drug HTA remains highly fragmented despite widespread statutory mandates. With only twelve jurisdictions requiring full economic evaluations and analytical perspectives diverging significantly, market access remains geography-dependent. This profound baseline heterogeneity poses a major challenge for future European policy harmonization.