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Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases

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Item Type:Review
Title:Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases
Creators Name:Sheean, M.E. and Malikova, E. and Duarte, D. and Capovilla, G. and Fregonese, L. and Hofer, M. and Magrelli, A. and Mariz, S. and Mendez-Hermida, F. and Nistico, R. and Leest, T. and Sipsas, N.V. and Tsigkos, S. and Vitezic, D. and Larsson, K. and Sepodes, B. and Stoyanova-Beninska, V.
Abstract:The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) is responsible for evaluating applications for orphan designation (OMPDs) and for deciding the orphan status at the time of marketing authorization of medicines in the European Union (EU). In this review, we provide transparency regarding assessment criteria and an in-depth review of nonclinical models and data that have been used to support OMPDs. Additionally, we present a literature-based analysis of existing nonclinical models and discuss key features of nonclinical studies, which are considered crucial for the support of future OMPDs. This could not only inform future drug development in rare infectious conditions, but also indicate areas where nonclinical models are indispensable or can be made more efficient.
Keywords:Communicable Diseases, Neglected Diseases, Orphan Drug Production, Rare Diseases, Animals
Source:Drug Discovery Today
Page Range:274-291
Date:February 2020
Official Publication:https://doi.org/10.1016/j.drudis.2019.10.015
PubMed:View item in PubMed

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