| Case study: determinants for successful marketing authorisation of orphan medicinal products in the EU. | |
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MedLine Citation:
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PMID: 22094244 Owner: NLM Status: Publisher |
Abstract/OtherAbstract:
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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. |
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Authors:
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Michelle Putzeist; Harald E Heemstra; Jordi Llinares Garcia; Aukje K Mantel-Teeuwisse; Christine C Gispen-De Wied; Arno W Hoes; Hubert G M Leufkens |
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Publication Detail:
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Type: JOURNAL ARTICLE Date: 2011-11-7 |
Journal Detail:
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Title: Drug discovery today Volume: - ISSN: 1878-5832 ISO Abbreviation: - Publication Date: 2011 Nov |
Date Detail:
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Created Date: 2011-11-18 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 9604391 Medline TA: Drug Discov Today Country: - |
Other Details:
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Languages: ENG Pagination: - Citation Subset: - |
Copyright Information:
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Copyright © 2011. Published by Elsevier Ltd. |
Affiliation:
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Utrecht Institute for Pharmaceutical Sciences, Division of Pharmacoepidemiology & Clinical Pharmacology, Utrecht University, The Netherlands; Medicines Evaluation Board, The Hague, The Netherlands. |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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