国外罕见病法律和保障体系与我国现状的对比分析 - 中国药事.pdfVIP

182 中国药事 2017 年 2 月 第 31 卷 第 2 期 ·国外药事· 国外罕见病法律和保障体系与我国现状的对比分析 * 莫昕，程峰 （清华大学公共健康研究中心， 北京 100084） 摘要 目的：为建立我国罕见病患者医药保障体系提供建议。方法：通过研究国外罕见病药物（孤儿 药）研发政策和罕见病患者医药保障法律，找到我国与其他国家罕见病诊治和保障体系之间的差距和不 足，并依照我国国情和医疗现状，提出建立我国罕见病患者保障体系的建议。结果：经过系统数据收集 和分析后发现，发达国家和地区都有特定的部门负责制定和执行罕见病法律，罕见病的定义明确，制定 了孤儿药研发的鼓励性政策并提供经费支持，罕见病的药物研发取得了极大进展，研发的积极性和研发 的速度均得到了很大提高。同时，这些国家和地区的孤儿药支付体系比较健全，建立了国家基金、医疗、 社会、商业保险等多重渠道不同比例的报销机制。相对来说，我国罕见病患者生存现状不佳，诊治状况 不容乐观，用药不规范、依从性低。造成这些现象的原因包括治疗费用高而报销比例低，未引进相关治 疗药物，供药不及时或停止供应，误诊率高等等。结论：建立我国罕见病患者保障体系是一项艰巨的工 作，应从 3 个方面展开：一是明确我国罕见病和罕见病用药的定义、完善罕见病立法；二是将孤儿药， 特别是通过药品治疗可以治愈的，以及针对儿童罕见病的药品纳入医疗保险，从而提高孤儿药的可及性； 三是建立罕见病诊疗流程，提高罕见病的诊疗水平。 关键词： 罕见病；罕见病保障；孤儿药；药物可及性；诊疗疗程 中图分类号：R97; R19-0 文献标识码：A 文章编号： 1002-7777(2017)02-0182-07 doi:10.16153/j.1002-7777.2017.02.013 Comparative Analysis of Legal and Social Security System of the Rare Diseases between Foreign Countries and China * Mo Xin, Cheng Feng (Tsinghua University Research Center for Public Health, Beijing 100084, China) Abstract Objective: To provide references for the establishment of social security system for rare diseases in China. Methods: Based on the study of drug research, development policy and social security system of rare diseases in foreign countries, the gap of the rare disease diagnosis and treatment as well as social security system was identified. It was suggested that Chinas social security for rare diseases be established in accordance with national and healthcare conditions. Results: After the systematic data collection and analysis, it was found that developed countries and regions have a clear definition for rare diseases. Besides, they have set up specific departments responsible for making and executing the laws of rare diseases, and they have issued policies to encourage and develop the orphan drug research and provide financial support. The research and developmen