An Updated Analysis of the Early Access Reform and Trends Observed in the Evaluation by the HAS

OBJECTIVES: Early access in France has established an overhaul of the former system of temporary authorization for use (ATU). This study aims to provide an overview of this program, 23 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 April 30, 2023.

RESULTS: In total, 148 decisions were issued by the HAS College. Of these, 136 followed an opinion by the Transparency Committee (TC). Oncology was the main therapeutic area concerned by TC assessment (51%). Most of the requests concerned post-MA early access (AP2: 68%). Among these applications, 85% were accepted (81% among AP1; 87% among AP2). The presence of appropriate treatment remains the most represented parameter in the reasons for refusals by the HAS College, both directly (54%) and indirectly (75% and 62% of refusals relating to the criteria “impossibility to defer treatment initiation” and “presumptively innovative nature”, respectively). Among accepted APs, 81 evaluations for reimbursement were published by the HAS. Important clinical benefit (SMR) was most often granted (79%) and most of the clinical added value (ASMR) granted by the TC were level III-IV (73%). Among these positive opinions, 27 were added to the reimbursement lists (52% as first-time registration, 48% as extension of indication).

CONCLUSIONS: This updated retrospective study confirms that early access reform has allowed many promising medicinal products to be available before reimbursement within the common law framework. Direct access, a new derogation, was introduced in the LFSS 2022 (Social Security Financing Act) and should enable drugs with an important SMR and ASMR I-IV to be reimbursed as soon as the TC opinion is published. A more detailed analysis would be interesting to see how the two early access programs will work together.