Presentation (CTI)

OBJECTIVES: Unlike several European health technology assessment bodies with explicit cost-effectiveness thresholds, France’s Haute Autorité de Santé (HAS) does not define a formal willingness-to-pay (WTP) threshold. While the Economic and Public Health Evaluation Committee (CEESP) critiques health economic analyses, it remains unclear whether it applies a consistent implicit ICER ceiling, or whether that threshold varies between orphan and non-orphan treatments. We aimed to determine whether an implicit WTP threshold can be inferred for orphan and non-orphan treatments from CEESP opinions issued between 2024 and 2025.
METHODS: All single-technology CEESP opinions on the HAS website from 1 January 2024 to 30 April 2025 were screened. Records were eligible if they included at least one ICER expressed in euros per QALY or per life-year gained. Orphan status was assigned if explicitly stated in the economic analyses. For each dossier, the base-case ICER and Service Médical Rendu (SMR) ratings were collected. ICER distributions were summarised with medians and interquartile ranges (IQR) and stratified by orphan and non-orphan treatments.
RESULTS: Of the 23 CEESP assessments released between 2024 and 2025, 22 were eligible for analysis: 9 orphan treatments and 13 non-orphan treatments. The median ICER for orphan treatments was €495,000 per QALY (IQR: €162,000 - €2,174,000), compared to €104,000 per QALY for non-orphan treatments (IQR: €59,000 - €166,000). Notably, 60% of orphan ICERs exceeded €300,000 per QALY, with a maximum of €4.75 million. In contrast, two-thirds of non-orphan ICERs fell below €150,000, while 90% of orphan submissions exceeded this threshold. All treatments received an SMR rating of important, moderate, or minor, and were reimbursed accordingly.
CONCLUSIONS: The WTP threshold for orphan treatments are, on average, approximately five times higher per QALY than non-orphan treatments; with non-orphan treatment ICERs rarely exceeding €150,000 per QALY and orphan treatment ICERs often reaching greater than €300,000 per QALY.