我国罕见病用药医疗保障政策及医保目录收载情况分析

Analysis on the Medical Insurance Policy and Medical Insurance Catalog for Rare Diseases in China

目的:通过分析国际、我国国家及地方罕用药保障制度、政策以及我国医保目录罕用药收录品种,探索我国罕用药医疗保障可及性现状及发展建议。方法:计算机检索国家及地方官方政府网站和中文数据库等,获取罕见病及罕用药相关政策法规;系统提取《国家基本医疗保险、工伤保险和生育保险药品目录》中有关罕见病的药品信息。结果:我国尚无罕见病的官方定义和官方名录。近年来关于罕见病、罕用药研发审批政策措施正日渐丰满。在医疗保障方面,很多省、市地区已经针对地方罕见病的流行性特征出台并落实了罕见病的保障政策,但国家层面的整体政策方面仍是空白。2017年版国家基本医疗保险、工伤保险和生育保险药品目录其中罕用药共53种,与2009年版目录相比,新增7个品种,增幅为15.22%;共覆盖17种罕见病,涉及脑部或神经系统病变、先天性代谢异常、血液疾病、骨及软骨病变多个疾病领域。与往年相比,新版医保药品目录中罕用药的剂型也有所增多,其中甲类相对乙类的剂型种类更多。结论:我国罕见病保障政策集中于研发审批领域,医疗保障以地方政策为主,新版国家医保药品目录罕用药范围扩大,但配套政策有待完善。因此,我国罕用药保障问题需要进一步引起重视。

Objective： To explore the status quo and development suggestions of the availability of medical insurance for orphan drugs in China through the analysis of international, national and local rare disease drug use system and policy, and the species of or- phan drugs in China＇s health insurance directory. Methods： The website of local government and the Chinese database were retrieved by computer, and the relevant policies and regulations of rare. diseases and orphan drugs were obtained. The information of rare diseas- es and orphan drugs in National Basic Medical Insurance, Employment Injury Insurance and Maternity Insurance Drug Catalog was systematically extracted. Results： There was no official definition of rare diseases and the official list of rare diseases in China. In re- cent years, with regard to the approval policy and measures of rare diseases and the R＆D（research and development） of orphan drugs fully developed. In the medical security, many provinces and municipalities introduced the popular characteristics of local rare diseas- es and the implementation of the rare diseases protection policy, however, the whole policy of nation level was still blank. The edition of 2017 of the basic medical insurance, industrial injury insurance and maternity insurance drugs directory contained 53 kinds of or- phan drugs, added 7 kinds compared to the version of 2009 version, an increase of 15.22%. 17 kinds of rare diseases were covered, in- volving blood diseases, bone or cartilage diseases, congenital metabolic disorders, brain or nervous system diseases, immune system diseases, skin diseases, digestive system diseases, endocrine system diseases and other treatment fields. Compared with previous years, the new version of the medicare drug list of orphan drugs also increased the dosage form. of which class A had more types of formulations rather than class B. Conclusion： China＇s rare disease social security system policy focused on the field of R＆D approv- al, medical security was dominated by local policy-based. The scope of orphan drugs in the new edition of national health insurance drug list was expanded, but the supporting policies needed to be improved Therefore. the problem of rare disease social security system policy in China needed to be paid more attention.