ABSTRACT
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. As of this date, the eligibility criteria, the timelines and the process have evolved. Now, EAP is assessed by the Haute Autorite de Sante (HAS) including the Transparency Committee (TC), which is responsible of reimbursement decision. Our study consisted of a descriptive analysis of EAP first decisions. Method(s): We conducted a retrospective analysis of all HAS decisions published between July 1, 2021 and Avril 21, 2022. Result(s): Among 46 EAP decisions published, 37% concerned medicines without marketing authorization (MA) and 63% concerned medicines with a MA. The most represented therapeutic area was oncology (46%) and 17% decisions were related to COVID-19 therapies. The average time from dossier submission to EAP decision publication was 69 days, with an increasing trend in 2022 compared to 2021 (85 vs 43 days). Most evaluated medicines (65%) had comparative phase III results at the time of their evaluation. The main reasons for denying an EAP were the lack of a presumption of innovation (18%), the identification of appropriate comparators (16%), and the lack of a presumption of efficacy and safety (15%). EAP was granted for 37/46 (79%) of treatments. Of these, 25/37 medicines were evaluated for reimbursement in France. The TC granted ASMR III (12/37), ASMR IV (3/37) and AMSR V (6/37). Conclusion(s): 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. Copyright © 2022