OBJECTIVES: Advanced Therapy Medicinal Products (ATMP) are costly breakthrough innovations generally responsible for a paradigm shift in the way targeted diseases are treated. Most ATMP granted marketing authorization (MA) at an early stage of development based on single arm trial.

METHODS: We conducted a retrospective analysis of ATMP market access in France by collecting information related to EAP, reimbursement and pricing of ATMP published between May 29, 2013 and June 21, 2023.

RESULTS: A total of 18 ATMP were assessed, including 5 (28%) in oncology which is the most represented therapeutic area. Since the introduction of the new EAP process (July 2021), 12 EAP application were submitted to the HAS of which 10 (83%) granted an EAP authorization (only 2 before MA). Two applications were rejected because of insufficient clinical data. Average delay between application and EAP decision publication was 80 days . In terms of reimbursement, 26 appraisals were published of which 20 were accepted for reimbursement (sufficient actual benefit) and 6 were not accepted (insufficient actual benefit). The Transparency Committee (TC) granted 1 (5%) ASMR II (important), 10 (50%) ASMR III (moderate), 4 (20%) ASMR IV (low), and 5 (25%) ASMR V (no clinical added value). Of the 16 ATMP evaluated by the HAS, only 5 are already commercialized (costly drug list). Regarding pricing, the mean delay between TC appraisal and price publication was around 15 months.

CONCLUSIONS: EAP represent an opportunity for ATMP in France. The lack of robust comparative clinical data does not appear to be a barrier to launch an EAP. On the other hand, the lack of comparative data is always criticized by the HAS when assessing reimbursement and make it challenging to obtain high levels of ASMR (I-III), which is essential to obtain a level of price in line with the cost of these therapies.