OBJECTIVES: Gene therapy, including chimeric antigen receptor T-cells (CAR-T cells), has led to remarkable clinical responses, providing new treatment opportunities for several conditions with poor prognosis. Balancing innovation with available resources for these innovative treatments poses challenges for budget-constrained European countries. This research aims to compare reimbursement decisions and timelines for the EU5 markets for all CAR-Ts currently available and to assess how this impacts patient access.

METHODS: A targeted literature review (01/2018-06/2022) was undertaken and fourteen EMA marketing authorisations for CAR-Ts were identified to June 2022. For each EU country, the CAR-T HTA process, reimbursement decision, and time taken for HTA decision publication were retrospectively reviewed. Tables and figures have been generated to facilitate a comprehensive analysis.

RESULTS: This study included 42 HTA and reimbursement decisions for 14 indications authorised by EMA across England, France, Germany, Italy, and Spain. Time from EMA approval to effective reimbursement varied dramatically across geographies (from 32 days in Germany to 615 in Spain, with an average of 236 days). The number of reimbursed treatments also varied significantly by country, (from 4 in Spain to 11 in Germany, with an average of 6 indications across EU5). Barriers limiting patient access to these treatments were adopted by all payers, with countries implementing some sort of outcomes-based payment model. However, the conditions governing these agreements, e.g., limitations, mandatory payment schedules and discounts varied greatly.

CONCLUSIONS: Gene therapy is providing novel clinical approaches that can result in improved and sustained long-term health outcomes. With a growing number of patients requiring these therapies, several obstacles remain and access is not uniform across EU5 with payers in some countries like Spain severely limiting options for patients. The delays and barriers identified in this analysis highlight potential shortcomings in the current reimbursement processes which can result in inequity for patients.