Evaluation of the Early Access Program Evolution: A Three-Year Retrospective Analysis

OBJECTIVES: Early access program (EAP) has been a success since its implementation. Three years have passed since the introduction of the new EAP. Since the reform, data for two complete years are available: 2022 and 2023, allowing for a time-based comparison. The aim of our study was to analyze the evolution of the EAP over the years and to focus on one drug of particular interest.

METHODS: We conducted a retrospective analysis of all HAS decisions published between July 1, 2021 and June 14, 2024, by years. Our analysis concerns only first decisions and not renewal.

RESULTS: During the period covered by our analysis, 164 initial EAP decisions were published. The average time between submission of the application and publication of the EAP decision remained relatively stable between 2022 and 2023 (88 days and 96 days, respectively) but increased significantly in the first half of 2024 (120 days).

The number of EAP decisions published by the HAS in 2023 was almost divided by two compared to 2022, 45 versus 82. This decrease could be explained by the smaller number of drugs in compassionate access (CA) or in nominative temporary utilization authorization (ATUn) that needed evaluation in 2023 compared to 2022, 27% versus 44%. However, the number of unfavorable decisions significantly increased from 18% in 2022 to 38% in 2023, a trend that continued in 2024 with 60% of unfavorable decisions.

The most evaluated product was KEYTRUDA with 9 EAP decisions published, of which 7 were positive.

CONCLUSIONS: After a 3-year follow-up of published EAP decisions, despite an increased number of refusals, obtaining an EAP remains a positive indicator of innovation for the Transparency Committee, with around 75% of products granting a clinical added value (ASMR) III or IV.