OBJECTIVES: In France, patients can benefit an early access to innovation, ahead of the marketing authorization or final reimbursement, since 1992. This early access program (EAP) was reformed on July 1, 2021. The new process involves a joint assessment by ANSM and HAS. Thus, HAS is now involved in granting EAP (HAS decision) while continuing to assess drugs for reimbursement (Transparency Committee (TC) opinions). According to HAS, this ensures consistency between derogation-based schemes (EAP) and the common law funding scheme (based on TC opinions). This study aimed at analyzing TC opinions for reimbursement of drugs which have also been assessed for EAP with new process.

METHODS: A retrospective analysis was conducted for all drugs with, between July 1, 2021 and April 21, 2022: 1) an HAS decision for EAP 2) a TC opinion for reimbursement.

RESULTS: 24 drugs were identified. During this period, most decisions regarding EAP (18/24) occurred shortly before TC opinions (7 to 79 days) and few of them (6/24) after (96 to 280 days). 20 drugs were granted EAP and 4 drugs were not granted EAP. Among drugs granted EAP, all had, according to TC opinions, an important SMR and majority had a clinical added value either moderate (ASMR III ; 12/20) or minor (ASMR IV ; 3/20). The remaining drugs had an ASMR V (no clinical added value) which could be explained by specific reasons (e.g. potential improvement of patient’s pathway even if not proven, more restrictive indications for EA or need for additional data).

CONCLUSIONS: With the new process consistency between EAP decisions and TC opinion for reimbursement is ensured. Nevertheless, in specific situations, the new process gives the opportunity to some drugs to be granted EAP whereas the available data is not sufficient to demonstrate a clinical added value as shown by the published TC opinions.