OBJECTIVES: To review and analyze the new early access authorization program in France launched in July 2021.
METHODS: Treatments that were processed under the early access authorization program between July 2021 and June 2022 were analyzed. The assessments were examined for therapy area, processing timeline, and data used in the evaluation.
RESULTS: As of 30 June 2022, 55 of the 66 decisions (83%) that have been published by HAS were granted early access, and 11 (17%) applications were refused. Among those granted early access, 37 (67%) already had a marketing authorization (MA), of which 25 (68%) had conditional MA and 11 (30%) were granted orphan status; 18 (33%) applied to the program before obtaining regulatory approval (pre-MA). Among the treatments granted early access after MA, one received ASMR II, 15 received ASMR III, 2 Received ASMR IV, 5 received ASMR V rating, and 13 had not been evaluated for the indication. The average time taken for the processing of all applications (receipt of complete file to date of adoption) was 65 days (range, 1-121); mean time for ANSM opinion on the product’s presumed efficacy and safety in the absence of MA was 55 days (range, 4-112). Oncology drugs accounted for 21 (39%) of applications. Most decisions were based on phase 2-3 clinical data for technologies with or without MA. Only two positive, pre-MA early access authorization requests used phase 1 clinical data for treatments indicated in pre-exposure prophylaxis of coronavirus 2019 and unresectable or metastatic triple-negative breast cancer. Contributions from stakeholders, including patient and user groups, were involved in 39 (59%) applications (both pre-MA and post-MA).
CONCLUSIONS: Overall, the time taken to evaluate the early access applications is significantly lower compared with the 3-month examination period stipulated by the program, facilitating a quick access to patients with high unmet need.
