国内外孤儿药注册制度探究与启示

Exploration and inspiration of orphan drug registration systems at home and abroad

目前国外一些发达国家的儿童类孤儿药注册制度比较完善,如美国FDA建立了孤儿药的资格认证制度。欧美一些发达国家均建立了针对罕见病和孤儿药的专门监管机构和措施,我国与其不同,对罕见病的防治和孤儿药的注册还未给予足够关注,我国的孤儿药注册制度明显不如某些发达国家完善。本文通过文献综述对不同国家的孤儿药注册制度进行比较分析,并结合我国国情提出了促进我国孤儿药注册制度建立的方法。

At present,the registration systems of pediatric orphan drugs in some foreign developed countries are relatively perfect,for example,the US FDA has established the qualification system for orphan drugs.Unlike some developed countries in Europe and the US that have established special regulatory agencies and measures for rare diseases and orphan drugs.In China,the prevention and treatment of rare diseases and registration of orphan drugs have not received sufficient attention and are even marginalized.The orphan drug registration system in China is obviously less developed than those in some developed countries in Europe and the US.This paper presents a comparative analysis of orphan drug registration systems in different countries through a literature review and proposes ways to promote the establishment of an orphan drug registration system in China,taking into account the national conditions of China.