Presentation (CTI)

OBJECTIVES: The French Early Access (EA) scheme aims to provide rapid access to innovative medicines before permanent reimbursement assessment. This study investigated whether increasing EA refusal rates over time were associated with greater alignment between EA decisions and subsequent assessments of clinical value (ASMR) by the Transparency Committee (TC).
METHODS: Using publicly available HAS data, we analyzed all EA decisions issued between 2021 and June 2026.Temporal trends in EA approvals and refusals were first described. Among medicines subsequently assessed by the TC, the proportion receiving ASMR V rating (absence of clinical added value) was compared according to EA outcome and year of decision.
RESULTS: EA refusals increased substantially following implementation of the process, from 22% of decisions in 2021 to 48% in 2024, before stabilizing thereafter. Over the same period, the proportion of ASMR V ratings among medicines granted EA declined markedly, from 21-25 % between 2021 and 2023, to 9% in 2024, 8% in 2025 and 0% in 2026 (until June 2026). In contrast, ASMR V ratings remained common among products denied EA, representing 36-67% of subsequent TC assessments depending on the year considered. These findings suggest that, in the early years of the scheme, a non-negligible proportion of products receiving EA were ultimately considered to provide no added clinical value. More recent decisions appear to better discriminate between products likely and unlikely to demonstrate innovation at reimbursement assessment.
CONCLUSIONS: The increasing selectivity of the French EA appears to be associated with improved consistency between EA decisions and subsequent HTA outcomes. While EA refusals have become more frequent, products granted EA are increasingly less likely to receive an ASMR V rating, suggesting closer alignment between early access eligibility and later recognition of therapeutic innovation.