Presentation (CTI)

OBJECTIVES: Early access (EA) pathways have become a major mechanism to accelerate access to innovative anticancer therapies in France. Since its reform in 2021, little is known about the evolution of the number of patients treated through these derogatory access mechanisms at the national level. We described national trends in patient exposure to oncology drugs delivered through EA pathways between 2019 and 2024.
METHODS: A nationwide retrospective population-based study using the French National Health Data System (SNDS) was conducted. All oncology indications benefiting from EA authorization between 2019 and 2024 were identified. Monthly patient counts were aggregated at the national level overall and by therapeutic class. Temporal trends in patient exposure were analyzed descriptively over the period.
RESULTS: A total of 165 drug-indication pairs corresponding to 75 anticancer drugs were identified. The monthly number of patients treated through EA pathways increased from 330 patients in January 2019 to 5,523 patients in December 2024. Distinct temporal patterns were observed: while the number of patients EA increased markedly between 2019 and 2023, reaching a peak of approximately 8,200 patients/month, a decline was observed in 2024. This decrease coincided with a reduction in the number of EA authorizations and the transition of several therapies toward routine reimbursement.
CONCLUSIONS: This nationwide analysis provides the first comprehensive overview of patient exposure to oncology EA pathways in France over a six-year period. After a marked increase between 2019 and 2023, the number of patients may now be declining in France. Additional analyses, which will be presented at the congress, will help determine whether this trend is confirmed in 2025 and quantify the respective contributions of the reduction in new EA authorizations and the transition toward routine reimbursement.