Presentation (CTI)

OBJECTIVES: In France, the Economic and Public Health Assessment Committee (CEESP) of the French National Authority for Health (HAS) assesses the methodology of health economic evaluations for innovative treatments. Rare diseases present significant challenges in this context. Their low prevalence and the frequent scarcity of clinical, quality-of-life, and cost data increase the uncertainty surrounding these assessments. This study aimed to review economic opinions pertaining to rare diseases, identify their distinct methodological features compared to non-rare diseases, and assess the impact of these differences on cost-effectiveness results.
METHODS: We developed a database of all economic opinions published by the CEESP between 2020 (the release year of the latest HAS methodological guidelines) and the beginning of 2025. Data were extracted on the methods and outcomes used to determine the Incremental Cost-Effectiveness Ratio (ICER).
RESULTS: From 106 opinions identified, 57 (54%) were deemed methodologically sound by the CEESP, with a lower proportion for rare diseases than for non-rare diseases (45% vs. 59%). Evaluations for rare diseases were more likely to rely on external sources for utility data (19% vs. 7%). Regarding cost-effectiveness, many ICERs for both groups were concentrated in the €100,000-€200,000 per QALY range. However, the median ICER for rare diseases was substantially greater than for non-rare diseases (€227,075 vs. €154,000 per QALY). Consequently, 53% of interventions for rare diseases had ICERs exceeding €200,000 per QALY, compared to just 24% for non-rare diseases. Median Quality-Adjusted Life Year (QALY) gains were also higher for rare diseases (0.82 vs. 0.64).
CONCLUSIONS: These findings reveal significant differences in the economic evaluations for rare and non-rare diseases in France. It is particularly important to assess efficiency in the context of rare diseases, given that treatments tend to be expensive and involve small target populations.