OBJECTIVES: Early access program (EAP) was reformed on July 1, 2021. The Transparency Committee (TC), which is responsible of reimbursement decision, issue an opinion whereas the final decision is take by the College of the HAS. Our study consisted of a descriptive analysis of EAP decisions from the reform.

METHODS: We conducted a retrospective analysis of all HAS decisions published between July 1, 2021 and May 31, 2023.

RESULTS: Among 119 EAP decisions published, 41% concerned medicines without marketing authorization (MA) and 59% concerned medicines with a MA. The most represented therapeutic area was oncology (52%). The average time from dossier submission to EAP decision publication was 81 days and remained stable between 2022 and 2023 (88 days versus 89 days) in contrast to 2021 (42 days) which was the first year of the reform. The average time of evaluation is longer for products without MA (93 days) compared to products with MA (73 days). Most evaluated medicines (76%) had comparative phase III results at the time of their evaluation ; this number slightly increased compared to 2022 (65%). EAP was granted for 94/119 (79%) of treatments and 21% of the decision was a refusal and the main reasons for denying an EAP were the lack of a presumption of innovation (16%), the treatment that can be deferred (15%) and the identification of appropriate comparators (15%). Of these, 79 (95%) are reimbursed on the 83 evaluated for reimbursement in France. The TC granted ASMR III (30/79), ASMR IV (27/79) and AMSR V (22/79).

CONCLUSIONS: Since the reform, evaluation for EAP is conducted according to TC requirements and became a very important preparatory step for reimbursement. Pharmaceutical companies must ensure consistency between EAP and reimbursement dossiers and think about their market access strategy earlier.