Changing Evidence Requirements For France's Early Access Program (EAP): What Could We See In The Coming Year?

By Henri Leleu, MD, PhD, Martin Blachier, MD, MPH

France's Early Access Program (EAP) is a critical pathway for patients with severe or rare conditions to swiftly access innovative therapies, having granted over 120,000 patients early access since 2021. However, the program is at a pivotal turning point driven by mounting financial strains and concerns over whether preliminary clinical data is being sufficiently confirmed in longer-term trials or real-world use.

The Haute Autorité de Santé (HAS) has initiated a review of EAP's methodological doctrine in response to these challenges. Alarmingly, only 39% of evaluated early-access drugs ultimately demonstrated sufficient efficacy to secure a favorable long-term reimbursement rating, indicating gaps in predicting the true value of innovations. This, combined with projected EAP expenditure soaring to €700 million by 2025, necessitates change. The new framework will likely mandate stricter evidence requirements, including more robust clinical development plans and greater scrutiny of surrogate endpoints. HAS will also seek to integrate methodologically sound Real-World Evidence (RWE) and indirect comparisons, recognizing the limitations of Randomized Controlled Trials (RCTs) in certain contexts. Understand the drivers behind these impending changes and how the revised doctrine will reshape the landscape for securing early access.