罕见病药物研发进展过于缓慢。
Orphan drug development is progressing too slowly.
作者信息
Joppi Roberta, Bertele Vittorio, Garattini Silvio
机构信息
Mario Negri Institute for Pharmacological Research, Milan, Italy.
出版信息
Br J Clin Pharmacol. 2006 Mar;61(3):355-60. doi: 10.1111/j.1365-2125.2006.02579.x.
AIMS
To assess the methodological quality of OMP dossiers and to discuss possible reasons for the small number of products licensed.
METHODS
Information 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.
RESULTS
Out 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.
CONCLUSIONS
The 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.
目的
评估孤儿药档案的方法学质量，并探讨获批产品数量少的可能原因。
方法
从欧盟委员会企业与工业总司网站及欧洲公共评估报告中获取有关孤儿药认定和批准的信息。
结果
在255项孤儿药认定中，仅有18项获批（7.1%）。其档案常常显示出方法学上的局限性，如临床设计不当、在有可用活性对照的情况下缺乏活性对照以及使用替代终点。
结论
欧洲对制造商的激励措施匮乏以及申请文件的质量欠佳可能限制了新型孤儿药的数量。超过5000种等待治疗的罕见病是一个重要的公共卫生问题。
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