我国进口孤儿药可及性的调查研究

The Accessibility of Imported Orphan Drugs in China:A Survey Study

目的检索国外上市孤儿药品目录,了解这些药物在我国的上市情况,为提高我国孤儿药可及性提供参考。方法计算机检索已发布孤儿药物管理政策的国家和地区(美国、新加坡、日本、澳大利亚、欧盟、中国台湾地区和韩国)的官方药品管理网站,按事先设计的数据提取表提取已上市孤儿药品信息。检索中国国家食品药品监督管理局网站,查询这些药品在我国的上市和报销现状。药品分类参照《中国药典》(2010年版),疾病分类参照《疾病和有关健康问题的国际统计分类》(ICD-10),医保信息参照《国家基本医疗保险、工伤保险和生育保险药品目录》(2009年版)。结果美国、日本、澳大利亚和欧盟出台孤儿药目录,上市药物共1 133种,覆盖疾病类别26个。抗肿瘤药物,内分泌、营养和代谢疾病用药,血液系统疾病用药位列前三,分别为329种、168种和122种,占所有药物的29.04%、14.83%和10.77%。中国近十年进口国外孤儿药数量持续增长,但数量不及国外上市三成,上市数量最多的为抗肿瘤药物、抗传染病和寄生虫病用药及内分泌、营养和代谢疾病用药。医保报销目录中覆盖孤儿药33种,含甲类5种,乙类28种,占西药报销品种数的2.9%。结论我国进口孤儿药数量虽不断增加,但与国外差距明显。为提高孤儿药物的可获得性和可负担性,保障罕见病患者权益,我们建议国家相关部门:(1)尽快出台孤儿药物管理政策;(2)循证遴选进口孤儿药物清单,建立适合本国国情的孤儿药目录;(3)建立孤儿药进口审批绿色通道,缩短进口时间;(4)激励本土企业的研发积极性,重视孤儿药的研发与仿制。

OBJECTIVE To search the orphan drug list from the countries which had initiated orphan drug policies and give ref- erence to improve the orphan drug accessibility in our country. METHODS We searched the official medicine management wehsites of the United States ,Singapore ,Japan, Australia ,European Union, Chinese Taiwan and South Korea and extracted information of orphan drugs which had been approved to the market. Then those orphan drugs were investigated whether had been imported and reimbursed in China, through the website of China Food and Drug Administration. Drug classification was according to the ＂pharmacopoeia of the People＇s Republic of China＂ （2010 version）. Disease classification was according to the ICD-10. Insurance information was according to the ＂ national basic medical insurance, employment injury insurance and maternity insurance drug catalogue＂ （2009 version ） . RESULTS The United States,Japan and Australia had launched orphan drug list. A total of 1 133 drugs were improved to the mar- ket, covering 26 diseases. 329 anticancer drugs,168 endocrine, nutritional and metabolic disease drugs,122 hematologic disease drug ranked in the top three, accounted for 29. 04% , 14. 83% and 10. 77% of all drugs respectively. China has imported no more than 30% , with the anticancer drugs,itffectious and parasitic diseases drugs ,endocrine, nutritional and metabolic disease drugs ranking in the top three. In the imported drugs, 33 were included in the insurance list. CONCLUSION Orphan drug accessibility is lack in our country. To improve this status, the related department were suggested to develop national drug policies for orphan drugs, evidence- based select the orphan drug list appling to burden of disease, establish priority approach for approval of imported orphan drug and mo- tivate the R＆D enthusiasm of local companies.