| 引用本文：王苑如, 胡紫馨, 谢金平, 邵蓉.欧盟孤儿药资格认定管理政策分析及启示[J].中国卫生政策研究,2022,15(6):24-29 |
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| 欧盟孤儿药资格认定管理政策分析及启示 |
| 投稿时间：2022-03-29 修订日期：2022-06-09 PDF全文浏览 HTML全文浏览 |
| 王苑如, 胡紫馨, 谢金平, 邵蓉 |
| 中国药科大学国家药物政策与医药产业经济研究中心 江苏南京 211198 |
| 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3 678件孤儿药资格认定申请，其中2 399件由孤儿药委员会(COMP)给予了积极意见，1 047件已撤销，2 379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。 |
| 关键词:罕见病 孤儿药 资格认定 政策分析 |
| 基金项目:国家社会科学基金重大项目(15ZDB167) |
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| Analysis of the European Union's orphan drug qualification management policy and its enlightenment to China |
| 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 |
| Abstract: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Among them, 2399 were provided with positive comments by the Committee for Orphan Medicinal Products (COMP), 1047 were withdrawn, and 2379 were qualified by the European Commission (EC).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. |
| Key words:Rare diseases Orphan drug Qualification Policy analysis |
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