Nat Rev Drug Discov. 2011 May;10(5):341-9. doi: 10.1038/nrd3445.
In 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments.
2000 年，欧盟通过了关于罕见药品的法规，旨在使目前尚无满意治疗方法的患有严重、罕见疾病的患者受益。此后，根据孤儿药委员会（COMP）的积极意见，欧盟委员会已批准了 850 多项孤儿药认定，并且已有 60 多种孤儿药在欧洲获得上市许可。值此 COMP 成立十周年之际，我们在此对过去十年的成果和经验进行反思，并探讨未来的问题，例如为大量仍缺乏有效治疗方法的罕见疾病推动药物开发。
