Presentation (CTI)

OBJECTIVES: In France, the Commission for Economic and Public Health Evaluation (CEESP) assesses the methodological robustness of cost-effectiveness analysis, based on guidelines issued by the French National Authority for Health (HAS). It identifies methodological reservations and, since 2021, also characterizes the level of uncertainty.Orphan drugs, often developed in contexts of high unmet need and limited evidence, present specific challenges for economic evaluation. This study focuses on CEESP evaluations of orphan drugs since 2021, to explore how these challenges are reflected in recent appraisals.The objective is to analyze CEESP evaluations of orphan drugs issued between 2021 and 2024, with a focus on the reported ICERs, their methodological validity, and the nature of major methodological reservations.
METHODS: All CEESP health economic opinions on orphan drugs published between January 2021 and December 2024 were reviewed using the Vyoo Agency database.
RESULTS: Between 2021 and 2024, 31 CEESP health economic evaluations on orphan drugs have been published. Among them, 9 were validated, 16 were invalidated due to major methodological reservations and 6 cases of high overall uncertainty. The average published incremental cost-effectiveness ratio (ICER) was €1,041,448 per QALY, with values ranging from €7,392 to €4,753,788 per QALY. Three dominant cases and one dominated were identified, but all were invalidated by CEESP. The average validated ICER was €954,174 per QALY.The 22 major reservations leading to invalidation were mainly related to utility estimation (n=9), lack of data or robustness (n=8), sensitivity analyses (n=2), model structure (n=1), economic analysis (n=1) and objective (n=1).
CONCLUSIONS: The average ICER of the economic evaluations of orphan drugs between 2021 and 2024 is greater than €1 million per QALY. Most economic evaluations for orphan drugs were invalidated. Fulfilling the requirements of CEESP seems difficult for rare diseases.