欧盟罕用药管理的成就与经验及其对中国的启示-中国现代应用药学.PDFVIP

·药事管理 · 欧盟罕用药管理的成就与经验及其对中国的启示 林禹鸿，吴晓明( 中国药科大学国际医药商学院，南京 210009) 摘要：目的 提出我国罕用药制度的建议。方法 制度比较分析。结果 欧盟通过颁布《罕用药管理规定》以促进罕用 药的研发、投产和上市，取得了较为显著的成绩。这得益于欧盟在管理罕用药领域内采取各项行之有效的政策措施，主 要体现在夯实主要立法基础、增强制度可操作性、完善特别审批程序以及大力资助研发投入等方面。这些举措给中国未 来罕用药管理政策的制定和完善提供了丰富而又宝贵的经验。结论 我国需要建立罕用药管理制度、完善特殊审批制度、 促进罕用药基础研究的成果向应用领域转化。 关键词：罕用药；管理；罕见病 中图分类号：R926 文献标志码：A 文章编号：1007-7693(2012)12-1154-05 Achievements and Experience of EU Orphan Drug Administration and Its Implications for China LIN Yuhong, WU Xiaoming(China Pharmaceutical University, School of International Pharmaceutical Business, Nanjing 210009, China) ABSTRACT: OBJECTIVE To put forward suggestions for orphan drug administration in China. METHODS System comparison was used. RESULTS According to issued Orphan Drug Act for promoting orphan drug RD, manufacturing and marketing, European Union achieved quite significant results. It was benefited from that EU taking various effective policy measures to manage orphan drug of this field, mainly in consolidating the key legislative basis and enhancing system operability, improving special approval procedures and financial support to RD investment, etc.. These measures would confidently benefit China of orphan drug development and improving policies management in the future by providing abundant and valuable experience. CONCLUSION We should better build the orphan drug management in China, complete the orphan drug approval system, promote transformation from the achievement of basic research for orphan drug to application area. KEY WORDS: orphan drug; administration; rare diseases 目前世界范围内已知的罕见病约有 5 000~ 盟罕用药委员会(Committee for Orphan Medicinal 6 000 种，罕见病患者数量少，制药企业因利润少 Products ，COMP)共完成了 848 项罕用药认定，见 不愿研发，即使研发也难以达到临床试验的最低人 图 1。在认定工作中常常会出现这样的情况：一种 数而难