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

Maria E Sheean; Eva Malikova; Dinah Duarte; Giuseppe Capovilla; Laura Fregonese; Matthias P Hofer; Armando Magrelli; Segundo Mariz; Fernando Mendez-Hermida; Robert Nistico; Tim Leest; Nikolaos V Sipsas; Stelios Tsigkos; Dinko Vitezic; Kristina Larsson; Bruno Sepodes; Violeta Stoyanova-Beninska

doi:10.1016/j.drudis.2019.10.015

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

Drug Discov Today. 2020 Feb;25(2):274-291. doi: 10.1016/j.drudis.2019.10.015. Epub 2019 Nov 5.

Authors

Maria E Sheean¹, Eva Malikova², Dinah Duarte³, Giuseppe Capovilla⁴, Laura Fregonese⁵, Matthias P Hofer⁵, Armando Magrelli⁶, Segundo Mariz⁵, Fernando Mendez-Hermida⁷, Robert Nistico⁸, Tim Leest⁹, Nikolaos V Sipsas¹⁰, Stelios Tsigkos⁵, Dinko Vitezic¹¹, Kristina Larsson⁵, Bruno Sepodes¹², Violeta Stoyanova-Beninska¹³

Affiliations

¹ Orphan Medicines Office, European Medicines Agency, Amsterdam, The Netherlands; Max-Delbrück Center for Molecular Medicine in Helmholz Association, Berlin, Germany. Electronic address: maria.sheean@ema.europa.eu.
² Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; State Institute for Drug Control, Bratislava, Slovak Republic; Comenius University, Department of Pharmacology and Toxicology, Bratislava, Slovak Republic.
³ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; INFARMED - Autoridade Nacional do Medicamento, Lisbon, Portugal.
⁴ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; C. Poma Hospital, Mantova, Italy; Fondazione Poliambulanza, Brescia, Italy.
⁵ Orphan Medicines Office, European Medicines Agency, Amsterdam, The Netherlands.
⁶ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; National Center for Drug Research and Evaluation, Istituto Superiore di Sanità, Rome, Italy.
⁷ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; Agencia Española de Medicamentos y Productos Sanitarios, Madrid, Spain.
⁸ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; Malta Medicines Authority, San Ġwann, Malta.
⁹ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; The Federal Agency for Medicines and Health Products, Brussels, Belgium.
¹⁰ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; Medical School, National and Kapodistrian University of Athens, Athens, Greece.
¹¹ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; University of Rijeka Medical School and University Hospital Centre Rijeka, Rijeka, Croatia.
¹² Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; INFARMED - Autoridade Nacional do Medicamento, Lisbon, Portugal; Universidade de Lisboa - Faculdade de Farmácia, Lisbon, Portugal.
¹³ Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, The Netherlands; Medicines Evaluation Board, Utrecht, The Netherlands.

PMID: 31704277
DOI: 10.1016/j.drudis.2019.10.015

Abstract

This review provides an overview of nonclinical in vivo models that can be used to support orphan designation in selected rare infectious diseases in Europe, with the aim to inform and stimulate the planning of nonclinical development in this area of often neglected diseases.

Publication types

Review

MeSH terms

Animals
Communicable Diseases / drug therapy*
Humans
Neglected Diseases / drug therapy*
Orphan Drug Production*
Rare Diseases / drug therapy*