OBJECTIVES: Access to orphan drugs (OD) varies considerably across EU countries and is generally slower than for non-orphan. In France, the early access (EA) scheme has been simplified in June 2021. The aim of our analysis was to assess the impact of this change on ODs access in France.

METHODS: New EA regulation and completed EA assessments from July 1^st, 2021 until June 3^rd, 2022 in France were reviewed. The following parameters were of interest: time to access, key criteria and evidence considered in EA assessments and subsequent transparency committee (TC) recommendation.

RESULTS: To be granted EA in France before or after marketing authorization (MA), the following criteria are considered as of July 2021: 1) serious or disabling disease, 2) lack of appropriate treatment, 3) initiation of the treatment cannot be postponed, and 4) innovative treatment. In May 2022, definitions of innovativeness and lack of appropriate treatment were refined. As of June 3^rd, 2022, 54 requests for EA were submitted, of which 47 were granted. Sixteen submissions related to OD of which one was refused and 15 granted (11 after MA and 5 before). Therapeutic indications of approved OD EA included cancer (7), genetic disease (5) and others (3). Average time to obtain EA for OD was 97 and 70 days before and after MA, respectively. Key considerations in therapeutic innovativeness assessment were appropriate development plan and efficacy. Of the 6 OD with EA subsequently submitted to TC, 4 received significant improvement in added benefit (ASMR III) and 2 no improvement (ASMR V).

CONCLUSIONS: Changes in French EA allows for pharmaceutical companies to enter through simplified EA process with clearly specified criteria, particularly for the OD putting them in better position for TC formal evaluation providing they meet the 4 criteria and have a plan in place to collect RWE.