Heemstra Harald E, de Vrueh Remco L A, van Weely Sonja, Büller Hans A, Leufkens Hubert G M
Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, PO Box 80.082, 3508 TB Utrecht, The Netherlands.
Drug Discov Today. 2008 Aug;13(15-16):670-6. doi: 10.1016/j.drudis.2008.05.001. Epub 2008 Jun 24.
With the assignment of the 500th European Union orphan drug designation in 2007, the Regulation on Orphan Medicinal Products truly begins to show its potential for delivering new medicines to patients with rare diseases. Here, we analysed European orphan drug development at a national level and unveil a strong relationship between orphan drug development and pharmaceutical innovation performance in Europe. Moreover, we identify gaps in transition from science into orphan drug development as important bottlenecks that exist in several European countries. Our findings underline the importance of innovation-based policies to enhance the development of orphan drugs in Europe.
随着2007年第500个欧盟孤儿药认定的授予，《孤儿药产品条例》真正开始展现出其为罕见病患者提供新药的潜力。在此，我们在国家层面分析了欧洲孤儿药的研发情况，并揭示了孤儿药研发与欧洲制药创新表现之间的紧密关系。此外，我们发现从科学到孤儿药研发的转化过程中存在的差距是几个欧洲国家存在的重要瓶颈。我们的研究结果强调了基于创新的政策对于促进欧洲孤儿药研发的重要性。
