摘要
目的 近年来，罕见病及其治疗药品（孤儿药）的监管已引起世界各国政府的广泛重视。除美国食品药品管理局外，欧盟药品管理局也已在罕见病及孤儿药管理领域建立了较完善的管理体系，如在法律法规、孤儿药认定、国际合作等方面的措施日趋成熟。我国的罕见病及孤儿药管理尚处在起步阶段，本研究为我国在罕见病及孤儿药管理上提供参考。方法 笔者主要就欧盟采取的罕见病及孤儿药管理思路进行研究和总结。 结果 欧盟的管理措施对罕见病的治疗和孤儿药的获取具有积极的推动作用，推进了罕见病患者获得平等治疗权利的进程，并将提高公众对罕见病的认识和改善罕见病患者的生命质量纳入了药品监管机构的监管内容。结论 我国相关管理机构应积极的借鉴国外经验，尽早完善我国罕见病的管理体制和制度。
关键词
欧盟 /
罕见病 /
孤儿药 /
管理
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刘玉聪 董江萍 .
欧盟罕见病及孤儿药管理现状的研究[J]. 中国药学杂志, 2012, 47(5): 395-398
Studies on Management Status of Rare Diseases and Orphan Drug in European Union[J]. Chinese Pharmaceutical Journal, 2012, 47(5): 395-398
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