国内外孤儿药注册制度比较研究

Analysis and Comparison of Foreign and Domestic Orphan Drug Registration Policies

目的借鉴部分发达国家与地区针对孤儿药注册管理的先进经验,探求适用于我国的孤儿药注册制度。方法对美国、日本、欧盟、澳大利亚、中国台湾等地所采取的孤儿药注册制度进行研究和对比,并且对我国孤儿药注册管理的历史和现状进行总结。结果国际上针对孤儿药注册普遍采用"身份认定+上市审批"的方式,这种方式切实有效地推动了各国及地区孤儿药产业的发展,提高了孤儿药的可及性。结论我国有关部门应借鉴先进经验,尽快建立起孤儿药制度体系并不断完善其注册制度。

OBJECTIVE To learn from several developed countries and regions about their experience of orphan drug registration management and to explore appropriate orphan drug registration policy for China. METHODS The orphan drug registration policies of America, Japan, EU, Australia and Taiwan were analyze and compared, and the history and current situation of the orphan drug registration in China were summarized. RESULTS It has been a common trend in the world to use ＂designation of drugs ＋ market approval＂ pattern for orphan drug registration, which has promoted the development of orphan drugs and increased the accessibility of orphan drugs in the above-mentioned countries and regions. CONCLUSION Related government departments in China should learn the ad- vanced experience and establish our orphan drug system as soon as possible, at the same time, we should keep improving the orphan drug registration policy in China.