ABSTRACT
OBJECTIVE: To explore the suggestions for the development of orphan drugs in China through comparative analysis of the status of orphan drugs in China and international developed countries or regions. METHODS: Based on “the first list of rare diseases” developed by the National Health Commission of the People′s Republic of China and other four departments,the accessibility of orphan drugs in China and abroad was compared. RESULTS: Until the end of 2017,the orphan drugs in China accounted for 38.33% of the 480 orphan drugs approved in the US, and 29.57% of the 115 orphan drugs approved in the EU. From the “the first list of rare diseases”, there are 60 orphan drugs for 36 diseases in the US and 27 orphan drugs for 18 diseases in the EU, and the orphan drugs approved in China accounted for 43.33% and 29.63, respectively. The 51.85% of the orphan drugs approved in China for the rare diseases of “the first list of rare diseases” are completely dependent on imports. CONCLUSION: There are still gaps of orphan drugs for “the first list of rare diseases”between China and the international developed countries or regions. Thus, it is urgent to promote legislation to speed up the research and limitation of orphan drugs in order to improve the development of orphan drugs in China.