Rare disease PMA trends in France: Price achieved vs. disease prevalence

CRA Life Sciences continues its series on leveraging RADAR, our orphan drug data repository. In this installment, we examine the relationship between launch prices in France and disease prevalence as reported in the Haute Autorité de Santé assessments. The analysis includes 67 rare disease products that were evaluated by the Transparency Commission between 2013 and 2023.

Key findings:

– Our analysis shows a correlation between reimbursed annual treatment costs achieved at launch (based on public list prices) and eligible patient numbers (used as an indication of disease prevalence), where costs decrease as patient numbers increase. This aligns with previous CRA findings across EU4 (France, Germany, Italy, Spain), the UK, and Japan (Matthews, C. et al., 2022).

– More than half of the orphan drugs captured are indicated for rare diseases with prevalence lower than 0.1 per 10,000 patients; approximately 20% are targeted to diseases with prevalence between 0.1 and 0.3 per 10,000 patients. While prevalence may be an important factor impacting negotiated price, other factors play a role.

– Waylivra is a notable outlier. This RNA therapy is indicated for the treatment of familial chylomicronemia syndrome, a severe disease due to its lifelong metabolic impact and associated risk of life-threatening complications (e.g., acute pancreatitis). The drug was granted an ASMR 4 and achieved a relatively high list price in a market with low-cost SoC alternatives, but price negotiations lasted 20 months.

– Note that this analysis does not account for incidence rates, which likely influence negotiated prices, and does not give an indication of the impact of prevalence on confidential net prices.

Key takeaways:

– There is a negative correlation between reimbursed annual treatment costs achieved at launch and eligible patient numbers.

– Prevalence alone does not explain price achieved at launch. Additional factors may come into play during negotiations, such as ASMR rating achieved, unmet need, availability of a clinical or pricing comparator, and robustness of clinical data.