OBJECTIVES: The reform of early access in France, which came into effect on July 1, 2021, has established a complete overhaul of the former system of temporary authorization for use (ATU). This study aims to provide an overview of this new system, 11 months after its implementation.

METHODS: A retrospective analysis of early access assessments from the National Authority for Health (HAS) was conducted. The HAS website was searched between July 1, 2021 and May 31, 2022.

RESULTS: In total, 64 decisions were issued by the HAS College. Of these, 59 followed an opinion by the Transparency Committee (TC) and 5 were minor adjustments. Oncology was the main therapeutic area concerned by TC assessment (n = 27), followed by infectious diseases (n = 10). Twenty-two requests concerned pre-marketing authorization (MA) early access (AP1), and 37 requests concerned post-MA early access (AP2). Among these applications, 81% were accepted (n = 17/22 AP1 ; n = 31/37 AP2). The most frequently refusal criteria by the TC were related to the presumptively innovative nature of the drug (n = 4) for AP1 and the absence of appropriate treatment (n = 6) for AP2. Of the accepted APs, 20 evaluations for reimbursement were published by the HAS. Important clinical benefit (SMR) was most often granted (n = 17). The clinical added value (ASMR) attributed by the TC was most often ASMR-III (n = 11), followed by ASMR-V (n = 7) and ASMR-IV (n = 2).

CONCLUSIONS: This retrospective study shows that early access reform has allowed a large number of promising medicinal products to be made available before reimbursement within the common law framework. A significant number of these drugs were considered innovative (ASMR I-IV) by the CT during their evaluation for reimbursement.