中国孤儿药现状及与国外对比分析研究

Current Status of Orphan Drugs in China and Comparative Analysis with Foreign Countries

目的通过对比分析我国与国际发达国家或地区孤儿药现状,探索我国孤儿药发展的建议。方法基于国家卫生健康委员会等5部门联合制定的《第一批罕见病目录》,对比研究国内外罕见病药物的上市可及性。结果截至2017年12月31日,美国上市的480种孤儿药中国占比38. 33%;欧盟上市的115种孤儿药中中国占比29. 57%。《第一批罕见病目录》中的36种疾病有获美国FDA批准的孤儿药60种,中国上市药物占比43. 33%;18种疾病有获欧盟EMA批准的孤儿药27种,中国占比29. 63%。中国上市的针对《第一批罕见病目录》的27种孤儿药中51. 85%完全依赖进口。结论我国孤儿药上市情况与国际发达国家和地区相比仍有一定差距,应加快孤儿药的研发、仿制,提高我国孤儿药的保障水平。

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.