摘要
中国罕见病患者数量众多,大多数患者面临着无药可治,境外有药、境内无药等问题。梳理了美国与欧盟的孤儿药激励政策,包括孤儿药立法与设置专门的管理机构,孤儿药资格认定,研发过程中政府提供资助、税收减免以及协议援助等,审批过程中提供加速上市通道,流通阶段给予市场独占期、优先审评劵等激励措施;并且探究了非营利组织在孤儿药研发中的激励措施,包括提供资金支持、临床研究等。从保障罕见病患者生命权与健康权、用药公平性与可及性以及临床试验中受试者知情权与隐私权的视角出发,为健全与完善中国孤儿药研发激励机制提供参考。
There are a large number of patients with rare diseases in China,and most of them are faced with problems such as no medicine to cure,and have drugs outside of China but not inside.This paper combed the incentive policies of orphan drugs in the United States and the European Union,including orphan drugs legislation and setting up special management institutions,orphan drugs qualification certification,government funding,tax reduction and agreement assistance in the research and development process,providing accelerated listing channels in the examination and approval process,giving market monopoly period in the circulation stage,and giving priority to review.On this basis,it also explored the incentive measures of non-profit organizations in the research and development of orphan drugs,including providing financial support,clinical research and so on.From the perspective of guaranteeing the right to life and health of patients with rare diseases,the fairness and accessibility of medication,and the subjects’ right to know and privacy in clinical trials,this paper provides reference for perfecting the incentive mechanism of orphan drugs research and development in China.
作者
张金子
宋晓琳
王泽钊
王萍
尹梅
ZHANG Jinzi;SONG Xiaolin;WANG Zezhao;WANG Ping;YIN Mei(School of Humanities and Social Sciences,Harbin Medical University,Harbin 150081,China)
出处
《中国医学伦理学》
2022年第9期971-977,共7页
Chinese Medical Ethics
基金
中央支持地方高校改革发展资金(高水干人才项目)“药物临床试验伦理审查规范化问题研究”。
关键词
孤儿药
罕见病
激励机制
孤儿药研发
医学伦理
Orphan Drugs
Rare Diseases
Incentive Mechanism
Orphan Drugs Research and Development
Medical Ethics
