Rising EAP Rejection Rates and Their Impact on Drug Access in France
Author(s)
Fernando Rosas, MEng, MBA1, Maximilian Hunt, BSc2, Ismail Ismailoglu, PhD3, Andreia Ribeiro, PhD4.
1Trinity Life Sciences, Ciudad de Mexico, Mexico, 2Trinity Life Sciences, New York, NY, USA, 3Principal, Trinity Life Sciences, Minneapolis, MN, USA, 4Trinity Life Sciences, Matosinhos, Portugal.
1Trinity Life Sciences, Ciudad de Mexico, Mexico, 2Trinity Life Sciences, New York, NY, USA, 3Principal, Trinity Life Sciences, Minneapolis, MN, USA, 4Trinity Life Sciences, Matosinhos, Portugal.
OBJECTIVES: Since the implementation of France's Early Access Program (EAP) reform in July 2021, rejection rates have increased substantially, with significant implications for patient access to innovative therapies. This study aims to analyze the evolution of EAP rejection patterns from 2021-2024 and evaluate their subsequent impact on standard reimbursement assessment outcomes, particularly examining cases where SoC evolution during drug development contributed to EAP denial
METHODS: We have leveraged a proprietary large language model for a retrospective analysis of all HAS EAP decisions from July 2021 to December 2024. Each case is categorized by rejection rationale, therapeutic area, and subsequent reimbursement pathway. Special attention is given to cases where SoC changed during development, particularly in oncology, where rapid therapeutic advances often create comparative evidence gaps. For rejected EAPs, we tracked subsequent ASMR (clinical benefit) ratings and reimbursement decisions, analyzing whether initial EAP rejection predicts less favorable outcomes or if full assessments account for evidentiary limitations linked to SoC evolution
RESULTS: Our review suggests EAP rejections increasingly reflect stricter HAS interpretations of “appropriate treatment” and “presumption of innovation,” with many denials linked to lack of comparative data versus the latest SoC. However, some therapies rejected for EAP, especially where SoC evolved during development, may still achieve moderate to positive ASMR ratings at full assessment, indicating a more nuanced approach by HAS during standard review. Oncology applications appear especially impacted, highlighting a potential misalignment between early and standard access criteria
CONCLUSIONS: This research addresses the critical impact of France's evolving EAP criteria on patient access to innovative therapies and provides insights into HAS's evolving risk-benefit assessment standards. Understanding the relationship between increasing EAP rejection patterns and subsequent reimbursement outcomes will inform evidence generation strategies and highlight growing patient access challenges in France's evolving regulatory landscape
METHODS: We have leveraged a proprietary large language model for a retrospective analysis of all HAS EAP decisions from July 2021 to December 2024. Each case is categorized by rejection rationale, therapeutic area, and subsequent reimbursement pathway. Special attention is given to cases where SoC changed during development, particularly in oncology, where rapid therapeutic advances often create comparative evidence gaps. For rejected EAPs, we tracked subsequent ASMR (clinical benefit) ratings and reimbursement decisions, analyzing whether initial EAP rejection predicts less favorable outcomes or if full assessments account for evidentiary limitations linked to SoC evolution
RESULTS: Our review suggests EAP rejections increasingly reflect stricter HAS interpretations of “appropriate treatment” and “presumption of innovation,” with many denials linked to lack of comparative data versus the latest SoC. However, some therapies rejected for EAP, especially where SoC evolved during development, may still achieve moderate to positive ASMR ratings at full assessment, indicating a more nuanced approach by HAS during standard review. Oncology applications appear especially impacted, highlighting a potential misalignment between early and standard access criteria
CONCLUSIONS: This research addresses the critical impact of France's evolving EAP criteria on patient access to innovative therapies and provides insights into HAS's evolving risk-benefit assessment standards. Understanding the relationship between increasing EAP rejection patterns and subsequent reimbursement outcomes will inform evidence generation strategies and highlight growing patient access challenges in France's evolving regulatory landscape
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA291
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas