Comparative Analysis of Medicines Reimbursement in France: Rare Diseases Versus Other Conditions

OBJECTIVES: In Europe, a rare disease is defined as one that affects fewer than one person per 2,000 individuals. The objective was to assess the impact of the disease rarity on the Transparency Committee (TC) evaluation for reimbursement eligibility.

METHODS: We conducted a comparative analysis based on the opinions issued by the TC in 2021 and 2022 for which a clinical added value (ASMR) has been recognized (I to IV). Two groups were considered for the comparison: drugs indicated for a rare disease and other drugs.

RESULTS: A total of 134 TC assessments were included in the analysis. Among them, nearly half (47%) involved a rare disease of which the majority (62%) of drugs were granted orphan designation. The average time (SD) between marketing authorization and publication of the TC opinion was similar for both groups (Rare diseases: 226 (242) days vs. Others: 228 (297) days; p = 0.9736). Overall, there was no statistically significant difference in clinical benefit (SMR) and ASMR levels between the two groups, with most cases receiving a high SMR (95% vs. 97%; p = 0.7994) and ASMR IV (60% vs. 52%; p = 0.321). Results of an additional analysis within the rare diseases group indicated a significant positive impact of the orphan status on the ASMR rating (p = 0.0111). Specifically, there were no ASMR II for drugs without orphan status, compared to 8% for those with orphan status, and nearly three times more ASMR III for drugs with orphan status (46% vs. 17%).

CONCLUSIONS: The main challenge of rare diseases lies in designing clinical studies that provide sufficiently robust evidence of the benefit/risk ratio. Despite this difficulty, rare diseases appear to be assessed by the TC in the same way as more widespread diseases, with similar SMR and ASMR ratings.