摘要
Abstract
Objective The study aims to explore the differences in health technology assessment(HTA)and reimbursement policies for chimeric antigen receptor T-cell(CAR-T)therapy products across the EU5 countries(UK,France,Germany,Italy,and Spain),and to assess their implications for China.Methods Official documents from HTA agencies,reimbursement decision-making bodies,and payers in the EU5 countries were retrieved.Combined with data from the European Medicines Agency(EMA)and the UK Medicines and Healthcare Products Regulatory Agency(MHRA),HTA reports,reimbursement decisions,and pricing mechanisms for CAR-T therapy products approved in major European markets as of April 2025 were analyzed.Results As of April 2025,six CAR-T therapy products had been launched in these five countries.Differences existed in HTA timelines,conclusions,and reimbursement decisions.The UK had the shortest initial HTA cycle(average 9.2 months).Through the coverage with evidence development(CED)mechanism,the Cancer Drugs Fund(CDF)provided temporary reimbursement for products not meeting routine access criteria;however,the secondary assessment process,based on real-world evidence,had a longer cycle.France adopted an early patient access policy,resulting in a relatively short HTA cycle(average 10.7 months).Its HTA results directly determined the reimbursement rate and pricing strategy,also utilizing the CED mechanism.Secondary evaluations were more frequent in France,with shorter intervals between initial and follow-up evaluations.Italy,Spain,and Germany featured fragmented payment systems and discontinuous HTA and reimbursement decision processes.All three adopted the pay-for-performance(P4P)model but differed in reimbursement trigger points,outcome indicators,and payment mechanisms.Germany implemented a survival-based"refund-for-failure"model,while Italy and Spain adopted a staged payment model based on clinical results.Conclusions Both CED and P4P can effectively address the high value and high uncertainty of CAR-T cell therapy products.CED enables provisional reimbursement while conducting concurrent post-marketing studies,whereas P4P links routine access to efficacy milestones for risk-sharing;both rely heavily on real-world evidence.It is recommended to integrate CED and P4P into the medical insurance access framework to reduce decision-making uncertainty through dynamic evidence updates.关键词
嵌合抗原受体T细胞治疗产品/卫生技术评估/医保报销/按疗效付费Key words
CAR-T cell therapy products/health technology assessment/medical insurance reimbursement/pay-for-performance分类
医药卫生
