Page 25 - 《中国药房》2023年13期

·药事管理·

英国罕见病用药医保准入政策分析及对我国的启示
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邓韬毅 ，刘德阳，周 静，武志昂，胡 明（四川大学华西药学院，成都 610041）
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中图分类号 R951 文献标志码 A 文章编号 1001-0408（2023）13-1555-07
DOI 10.6039/j.issn.1001-0408.2023.13.03

摘 要 目的 通过分析英国的罕见病用药医保准入政策，基于已有的准入途径及框架为我国罕见病用药医保准入提供参考依
据。方法 收集英国的罕见病用药相关指南、政策文件等，使用内容分析法对罕见病用药评估机制、报销决策标准、利益相关者参
与程度、处理不确定性及风险的应对策略、政策实施效果等进行分析，提炼英国罕见病用药医保准入的关键点，为我国建立完善罕
见病用药医保准入制度提供可参考建议。结果与结论 从准入途径来看，英国采取了单独的路径和明确的考量标准对罕见病用药
进行评估和报销。从评估机制来看，英国有医生、患者、申请人等多方利益相关者参与决策流程。英国通过收集更充分的临床证
据，利用患者可及计划来处理不确定性和风险。英国在实施罕见病用药相关政策之后，在罕见病用药资金投入、药品申请报销通
过率、受资助患者人数等方面卓有成效。建议我国在建立和完善罕见病用药评估报销制度过程中，通过建立单独的罕见病用药准
入评估路径，再加上更多利益相关的参与，实现我国罕见病用药可及性的提高。
关键词 罕见病用药；英国；医保体系；医保准入政策

Analysis of medical insurance access policy for rare disease drugs in the United Kingdom and enlightenment
to China
DENG Taoyi，LIU Deyang，ZHOU Jing，WU Zhiang，HU Ming（West China College of Pharmacy， Sichuan
University， Chengdu 610041， China）

ABSTRACT OBJECTIVE To provide reference for the access to medical insurance for rare diseases in China based on the
existing access pathway and framework by analyzing the access policy of medical insurance for rare diseases in the United Kingdom
（UK）. METHODS After collecting relevant guidelines and policy documents related to drug use for rare diseases in the UK，
content analysis method was used to analyze the evaluation mechanism of drug use for rare diseases， reimbursement decision-
making standards， stakeholder participation， coping strategies for dealing with uncertainties and risks， and policy implementation
effects， and extract the key points of medical insurance access for drug use for rare diseases in the UK， to provide some
suggestions for the establishment of medical insurance access system for rare diseases in China. RESULTS & CONCLUSIONS
From the perspective of access， the UK had adopted a separate approach and clear criteria to assess and reimburse drugs for rare
diseases. From the perspective of evaluation mechanism， multi-stakeholders such as doctors， patients and applicants participated in
the decision-making process in the UK. The UK addressed uncertainty and risk by gathering better clinical evidence and using the
patient access programme. After the implementation of the policy related to drug use for rare diseases， the UK had achieved
remarkable results in terms of funding for drug use for rare diseases， the reimbursement rate of drug application， and the number of
funded patients. It is suggested that in the process of establishing and improving the evaluation and reimbursement system for rare
diseases drugs in China， the availability of rare diseases drugs should be improved by establishing a separate access assessment path
for rare diseases drugs and involving more stakeholders.
KEYWORDS rare disease drugs； United Kingdom； medical insurance system； medical insurance access policy

罕见病（rare disease）又称“孤儿病”，各个国家或地 人数、发病率或者危害程度进行界定。罕见病用药
区对罕见病有不同的定义，但是一般都从罕见病的患病 （orphan medicinal product）是指用于预防、诊断、治疗罕

Δ 基金项目 国家自然科学基金资助项目（No.71473170） 见病的药品。因罕见病患病率低、特定罕见病的患者人
＊第一作者 硕士研究生。研究方向：药物政策与药物经济学。 数较少、药品生产企业的研发难度大且积极性不高、罕
E-mail：taoyia1109@163.com
见病用药价格昂贵，使得罕见病用药具有医保准入难、
# 通信作者 教授，博士生导师，博士。研究方向：药物政策与药物
[1]
经济学。E-mail：huming@scu.edu.cn 可负担性差的属性 。因此，如何合理制定罕见病用药

中国药房 2023年第34卷第13期 China Pharmacy 2023 Vol. 34 No. 13 · 1555 ·