国内外罕见病用药保障体系对比研究及其启示  

The Enlightenment of Foreign Drug Security System for Rare Diseases to China

作　　者：唐彦[1] 张学杰 刘鑫[1] 梅丹[1] 付强[1] 李建涛[1] 张谦[1] 张鹏霄[1] 左玮[1] 张波[1] TANG Yan;ZHANG Xue-jie;LIU Xin;MEI Dan;FU Qiang;LI Jian-tao;ZHANG Qian;ZHANG Peng-xiao;ZUO Wei;ZHANG Bo(Department of Pharmacy,Chinese Academy of Medical Sciences,Peking Union Medical College Hospital,Beijing 100730,China;Department of Pharmacy,Qingdao Huangdao District People's Hospital,Qingdao 266400,China)

机构地区：[1]中国医学科学院北京协和医院药剂科,北京100730 [2]青岛市黄岛区人民医院药剂科,山东青岛266400

出　　处：《中国药学杂志》2020年第11期955-960,共6页Chinese Pharmaceutical Journal

摘　　要：目的通过分析我国与国外发达国家罕见病用药保障体系的进展,探索我国罕见病用药医疗保障体系发展的建议。方法通过文献检索,汇总分析国内外罕见病用药相关政策法规、研发政策和罕见病患者医疗保障政策。结果经过分析发现,发达国家在罕见病的定义、法规、药物研发激励方面均起步较早,并取得了较好发展,同时具有比较健全的罕见病医疗保障体系。目前我国尚未建立完善的罕见病政策法规和医疗保障体系,也没有罕见病的官方定义。但是,近年来我国关于罕见病用药相关研发审批以及医疗保障等政策正日臻完善。结论我国虽然尚未建立完备的罕见病政策体系,但也在根据我国具体国情制定一系列的相应政策,缩小了与发达国家之间的差距。我国应在借鉴国外经验的同时,考虑到我国的实际情况,进一步完善罕见病医疗保障相关政策,保障罕见病患者的诊疗。OBJECTIVE To explore the suggestions for the development of the medical security system and the medication for rare diseases(orphan drugs)in China and analyze the current progress of orphan drugs in China as well as other developed countries.METHODS Based on literature search,the policies related with orphan drugs well as the medical security policies for patients with rare diseases in China and abroad were analyzed and summarized.RESULTS Through analysis,it was found that the definition,regulation,and the research and development for orphan drugs started early in the developed countries and had made good progress.At the same time,they have relatively sound medical security system for rare diseases.At present,China has not established a complete policy,regulation and medical security system for rare disease,nor has an official definition of rare diseases.However,in recent years,China's policies on the research and approval of rare diseases and as well as medical serurity are improving day by day.CONCLUSION Although China has not yet established a complete and perfect policy system for rare diseases,it has already made a series of policies according to our specific national conditions,which narrowed the gap between China and other developed countries.Our country should learn from foreign experience and consider the actual situation of our country,further complete the policies for rare diseases,and improve the diagnosis and treatment of patients with rare diseases.

关 键 词：罕见病 孤儿药 罕见病政策 医疗保障 

分 类 号：R95[医药卫生—药学]