Presentation (CTI)

OBJECTIVES: This study retrospectively analysed health technology assessments (HTAs) submitted in 2024, to evaluate differences in the approval of orphan versus non-orphan drugs across selected European countries. Specifically, examining the extent to which submissions for orphan drugs incorporated real-world evidence (RWE) and whether its inclusion was associated with a higher likelihood of receiving a positive reimbursement recommendation.
METHODS: HTAs published in 2024 in 7 European countries were screened and categorised as orphan or non-orphan, with outcomes analysed to evaluate the likelihood of HTA success according to orphan-designation and use of orphan-specific reimbursement processes. The HTA documents for all orphan products were subsequently screened for the inclusion of RWE, determined by targeted searches for RWE-related terminology and common real-world data sources. HTA outcomes were then comparatively analysed to examine if the inclusion of RWE was associated with a higher likelihood of successful reimbursement.
RESULTS: In total, 1040 reimbursement decisions were included in the analysis: orphan (n=285) and non-orphan (n=755). For countries with orphan-specific routes, likelihood of a positive outcome was 87% for orphan drugs versus 66% for non-orphan drugs. Whereas for countries without separate orphan processes, this was 76% for orphan drugs versus 78% for non-orphan drugs. After de-duplication, 195 HTA submissions for orphan drugs were screened for the inclusion of RWE, with some excluded due to insufficient information available (n=43). Of the remaining 152 orphan submissions, 61% (n=93) referenced the use of at least one type of RWE. Furthermore, analysis found that the rate of positive reimbursement with RWE was 84% versus 81% without RWE.
CONCLUSIONS: In 2024, orphan drug submissions referencing RWE had higher reimbursement success, particularly in countries without orphan-specific HTA processes. These findings underscore RWE’s growing influence in HTA outcomes and highlight the need for orphan-specific considerations in Europe ahead of implementing Joint Clinical Assessments for orphan drugs.