2010年-2019年国外孤儿药研发概况简析

Analysis of Foreign orphan drug research and development in 2010-2019

目的:了解国外孤儿药研发现状及管理经验,为我国孤儿药研发提供参考。方法:分析近10年美国和欧盟国家孤儿药物的研发特点和管理体系建设。结果:美国是世界上最早颁布孤儿药法令的国家。2010年—2019年,美国食品及药品管理局(FDA)和欧盟药物委员会批准上市的孤儿药分别为163个和113个,欧美国家每年孤儿药物批准上市数量总体呈现上升趋势,药物主要分布在肿瘤、消化系统(或新陈代谢)、神经系统、血液及造血功能系统。结论:美国是世界上孤儿药物管理制度最为完善的国家,欧盟国家管理制度框架已基本完成,处于快速发展阶段;肿瘤药是孤儿药研究重点分布领域。

Objective:To understand the current status and management experience of foreign orphan drug research and development,and to provide reference for my country's orphan drug research and development.Methods:A comparative analysis of the development characteristics and management system construction of orphan drugs in the United States and EU countries in the past 10 years.Results:The United States was the first country in the world to promulgate an orphan drug law.From 2010 to 2019,the number of orphan drugs approved for marketing by the U.S.FDA and the european commission for medicines was 163 and 113,respectively.The number of orphan drugs approved for marketing in European and American countries shows an overall upward trend each year.The drugs are mainly distributed in tumors,digestive system and metabolism,nervous system,blood and hematopoietic system.Conclusion:The United States has the most complete orphan drug management system in the world.The European Union country management system framework has been basically completed.European and American countries are in a stage of rapid development.Tumor drugs are the key area of orphan drug research.