کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
2081490 | 1545138 | 2012 | 7 صفحه PDF | دانلود رایگان |
عنوان انگلیسی مقاله ISI
Determinants for successful marketing authorisation of orphan medicinal products in the EU
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موضوعات مرتبط
علوم زیستی و بیوفناوری
بیوشیمی، ژنتیک و زیست شناسی مولکولی
بیوتکنولوژی یا زیستفناوری
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چکیده انگلیسی
In 2010, the European Regulation for Orphan Medicinal Products (OMPs) was in force for ten years. In this study we assessed possible determinants of applications for OMPs in the EU since 2000 that are associated with a successful marketing authorisation. Our analysis shows that clinical trial characteristics such as demonstrating convincing evidence of a beneficial effect on the primary endpoint, the selection of a clinically relevant endpoint, providing RCT data as pivotal study evidence and the submission of sound dose finding data are critical success factors. In addition, high medical need seems to counterweigh uncertainties about the scientific evidence in the benefit–risk assessment of OMPs.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Drug Discovery Today - Volume 17, Issues 7–8, April 2012, Pages 352–358
Journal: Drug Discovery Today - Volume 17, Issues 7–8, April 2012, Pages 352–358
نویسندگان
Michelle Putzeist, Harald E. Heemstra, Jordi Llinares Garcia, Aukje K. Mantel-Teeuwisse, Christine C. Gispen-De Wied, Arno W. Hoes, Hubert G.M. Leufkens,