英国罕见病用药纳入报销体系的主要途径及对我国的启示

为我国建立合理且多元化的罕见病用药纳入报销体系制度提供经验及启示，本研究通过查阅英国国家卫生与临床优化研究所(National Institute for Health and Care Excellence，NICE)与英国国民健康保障体系(National Health Service，NHS)官网，收集英国有关罕见病用药纳入报销体系的资料，归纳并分析相关文献，对罕见病患者药品的可及性进行理论研究。在NHS体系中，英国罕见病用药纳入报销体系主要可以通过7种途径:由NICE负责评估的3种途径(多技术评估MTA、单一技术评估STA、高度专业化技术评估HST)与NHS直接管理的4种途径(专项购买服务、肿瘤药物基金CDF、个人资金申请IFRs、对缺乏临床数据项目的评估购买计划CtE)。通过分析英国罕见病用药纳入报销体系的7种主要途径及英国在实施多种纳入报销途径中出现的问题，对我国罕见病用药的报销制度的建立有一定借鉴意义。

Major routes for orphan drugs to be incorporated into the reimbursement system of UK and the enlightenment to China

In order to provide enlightening experience for the establishment of reasonable and diversified orphan drugs into the reimbursement system in China, the official websites of the National Institute for Health and Care Excellence(NICE)and the National Health Service(NHS)in the UK were inspected, collecting and summarizing the relevant documents, on the inclusion of orphan drugs into the reimbursement system. Relevant literatures were analyzed with theoretical studies on the accessibility of medicines for patients with rare diseases. In the NHS system, the inclusion of orphan drugs into the reimbursement system in the UK can be achieved mainly through seven routes, with three routes that are evaluated by NICE(MTA, STA and HST)and four are directly managed by the NHS(specialized commissioning, CDF, IFRs, CtE). Through the analysis of the inclusion of orphan drugs into the reimbursement system and the various problems in the UK, we have found some enlightening experience for the establishment of the reimbursement system for orphan drugs in China.