摘要
目的:为我国孤儿药资格认定和管理、后续激励政策的制定提供相关参考。方法:结合文献研究、数据分析(2000—2020年欧盟孤儿药资格认定审批数据)等方法,对欧盟孤儿药资格认定程序及政策实施效果开展分析。结果:欧盟孤儿药资格认定程序大致由提交前会议阶段、申请提交阶段、验证阶段以及评估并形成最终建议阶段四个环节组成,2000—2020年,欧盟共受理了3678件孤儿药资格认定申请,其中2399件由孤儿药委员会(COMP)给予了积极意见,1047件已撤销,2379件由欧盟委员会(EC)认定,最终共有190件孤儿药上市申请获得授权批准。建议:建立孤儿药资格认定机制,对罕见病及孤儿药均实施目录管理,为罕见病患者用药提供法律依据;建立罕见病及孤儿药相关数据库,与国际罕见病数据库接轨,助力孤儿药研发,形成全球罕见病诊疗、药品保障共同体。
Objective:To provide references for the qualification and management of orphan drugs and the ensuing incentive policies in China.Methods:By combining literature research with data analysis(on EU orphan drug qualification review data in the period of 2000—2020),the EU orphan drug qualification process and effects of policy implementation were analyzed and studied.Results:The EU orphan drug qualification process is roughly composed of four stages:the pre-submission meeting stage,the application submission stage,the verification stage,and the evaluation and final suggestion stage.From 2000 to 2020,the EU has processed a total of 3678 cases of orphan drug qualification applications.Among them,2399 were provided with positive comments by the Committee for Orphan Medicinal Products(COMP),1047 were withdrawn,and 2379 were qualified by the European Commission(EC).Eventually,a total of 190 orphan drugs were approved for marketing.Suggestions:a qualification mechanism should be established for orphan drugs,and catalog management should be established for rare diseases and orphan drugs,to provide legal basis for the medication of patients with rare diseases;a database for rare diseases and orphan drugs should be established,which should be connected to the international rare disease database,so as to assist the research and development of orphan drugs,and form a global community for the diagnosis and treatment of rare diseases and security of orphan drug stock.
作者
王苑如
胡紫馨
谢金平
邵蓉
WANG Yuan-ru;HU Zi-xin;XIE Jin-ping;SHAO Rong(The Research Center of National Drug Policy & Ecosystem, China Pharmaceutical University, Nanjing Jiangsu 211198, China)
出处
《中国卫生政策研究》
CSCD
北大核心
2022年第6期24-29,共6页
Chinese Journal of Health Policy
基金
国家社会科学基金重大项目(15ZDB167)。
关键词
罕见病
孤儿药
资格认定
政策分析
Rare diseases
Orphan drug
Qualification
Policy analysis
作者简介
王苑如(1998年—),女,硕士研究生,主要研究方向为医药政策与法规。E-mail:17748854326@163.com;通讯作者:谢金平。E-mail:15151870834@163.com。
