罕见病药品资格认定的国际实践与启示

Practice of the Qualification and Recognition for Orphan Drugs in the World and its Inspiration

摘要: 分析目前国内外罕见病药品资格认定现状，包括其负责机构、认定流程、认定及上市数量等。通过横向对比，分析典型发达国家和部分发展中国家在罕见病药品资格认定方面的差异，探究中国在罕见病药品监管方面的不足。借鉴典型发达国家和部分发展中国家的先进经验，以期为中国罕见病药品认定管理提供建议，为加快中国罕见病药品研发上市，为最大程度提高患者治疗可及性提供参考。

Abstract: We have analyzed the current status of recognization and qualification of orphan drugs in China and abroad, looking at the aspects of the authority institutions, identification and qualification process, and the number of orphan drugs identified and available in the market. By comparing and analyzing horizontally the differences in orphan drugs identification between representative developed countries vs. some developing countries, we discuss the inadequacy of orphan drugs supervision in China. We introduce the advanced experience from the developed countries and some developing countries to provide suggestions for the identification and management of orphan drugs, hoping to speed up the process of development and market availability of orphan drugs and to maximize patient's accessibility to treatment in China.