Orphan drug development is progressing too slowly
Orphan drug development is progressing too slowly
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DOI：
10.1111/j.1365-2125.2006.02579.x
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发表时间：
2006-03-01
影响因子：
3.4
通讯作者：
Garattini, S
中科院分区：
文献类型：
--
作者：
Joppi, R;Bertele, V;Garattini, S
AimsTo评估的方法质量的OMP档案，并讨论可能的原因，少量的产品licensed.MethodsInformation有关孤儿药的指定和批准，从欧盟委员会的网站，企业和工业DG和欧洲公共评估reports.ResultsOut的255 OMP指定，只有18个被批准（7.1%）。他们的档案往往表现出方法的局限性，如不适当的临床设计，缺乏有效的比较，如果可用和使用的替代endpoint.ConclusionsThe缺乏欧洲的激励措施的制造商和不良的文件支持的应用程序可能限制了新的OMP的数量。5000多种等待治疗的罕见病是一个重要的公共卫生问题。
AimsTo assess the methodological quality of OMP dossiers and to discuss possible reasons for the small number of products licensed.MethodsInformation about orphan drug designation and approval was obtained from the website of the European Commission-Enterprise and Industry DG and from the European Public Assessment Reports.ResultsOut of 255 OMP designations, only 18 were approved (7.1%). Their dossiers often showed methodological limitations such as inappropriate clinical design, lack of active comparator where available and use of surrogate end-points.ConclusionsThe paucity of European incentives for manufacturers and the poor documentation underpinning the applications may have limited the number of new OMP. The over 5000 rare diseases awaiting therapy are an important public health issue.