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
Hauptverfasser: 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
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container_end_page 480
container_issue 7
container_start_page 479
container_title Nature reviews. Drug discovery
container_volume 18
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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