Defining orphan conditions in the context of the European orphan regulation: challenges and evolution
The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to defin...
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Veröffentlicht in: | Nature reviews. Drug discovery 2019-07, Vol.18 (7), p.479-480 |
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creator | O’Connor, Daniel J. Sheean, Maria E. Hofer, Matthias P. Tsigkos, Stelios Mariz, Segundo Fregonese, Laura Larsson, Kristina Hivert, Virginie Westermark, Kerstin Naumann-Winter, Frauke Stoyanova-Beninska, Violeta Barišić, Ingeborg Capovilla, Giuseppe Magrelli, Armando Sepodes, Bruno |
description | The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to define orphan conditions in the context of the European regulatory framework. |
doi_str_mv | 10.1038/nrd.2018.128 |
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subjects | 631/154/109 631/154/155 692/308/153 Biomarkers Biomedical and Life Sciences Biomedicine Biotechnology Cancer Research Comment Corporate sponsorship Disease Disease prevention Histopathology Laws, regulations and rules Medicinal Chemistry Molecular Medicine Orphan drugs Pharmaceutical industry Pharmacology/Toxicology Population Regulation |
title | Defining orphan conditions in the context of the European orphan regulation: challenges and evolution |
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