ABSTRACT
Objectives: The publication of the French Social Security Financing Act for 2021 enacted the reform of the authorizing procedure for early and derogatory access to medicinal products by regrouping former derogatory access processes into 2 distinct pathways: pre/post-marketing authorization early access (EA) and compassionate access. The reform is effective since July 2021 and the HAS (Haute Autorite de Sante) is now responsible for evaluating EA requests based on a set of pre-defined criteria. We aimed at analyzing all EA opinions issued by the HAS since July 2021. Method(s): A review of HAS early access opinions was conducted. All published opinions since the reform was implemented and up to March 2022 were included. Outcomes of interest included: number of positive opinions, therapeutic areas, percentage of eligibility criteria met, market authorization (MA) status, indication, and mean/median review time. Result(s): A total of 34 opinions was extracted with the main therapeutic areas being oncology (n=12;35%), infectiology (n=11;32%, of which 10 were related to COVID-19) and rare diseases (n=4;12%). Additionally, 13 requests (38%) were submitted pre-MA and 21 (62%) post-MA. Out of the 34 opinions, 26 were approved (76%), with a mean/median time of 53/51 days (56/55 days when excluding infectiology-related opinions) and 8 were rejected (24%), with a mean/median time 56/51 days (62/54 days when excluding infectiology-related opinions). All approved opinions matched the 'severe disease' and 'invalidating' criteria, and only 65%, 88% and 96% matched the 'rare disease', 'no existing treatment' and 'innovation' criteria respectively. Conclusion(s): The reform allows for a faster access to treatment for patients. The reform will also improve HAS review time for reimbursement dossiers if an early access request is submitted beforehand. An update of the reform further describing HAS criteria for EA has recently been published in April 2022. Copyright © 2022