Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases.

Thérapie
Joëlle Micallef, Olivier Blin

Abstract

The research and drug development process in rare diseases is challenging in addition to those for common diseases. To stimulate its development, the orphan drug designation (ODD) was introduced in in European Union in 2000. In the present paper, we describe the main characteristics of ODD in European Union in particular the requested criteria for ODD, the overview of the general procedure and the main incentives for Sponsors and finally the predicted factors related to successful development and marketing approval of orphan drugs after designation. In accordance with regulation, an application for ODD must be submitted to European Agency including a scientific part based on relevant scientific literature related to the condition and results on experimental studies with the specific product (and clinical studies if available). Three following criteria are a central position in this application: medical plausibility, rarity and medical significant benefit. The Committee for Orphan Medicinal Products (COMP) is the European Medicines Agency's (EMA) committee responsible for recommending orphan designation of medicines for rare diseases. Even if pre-submission meetings are not mandatory, EMA strongly encourages sponsors to request ...Continue Reading

References

Oct 3, 2002·Nature Reviews. Drug Discovery·Marlene E HaffnerMarie Moses
Aug 3, 2004·Nature Reviews. Drug Discovery·Ted T Ashburn, Karl B Thor
Feb 3, 2006·The New England Journal of Medicine·Marlene E Haffner
Jun 23, 2006·Journal of Internal Medicine·M WästfeltJ-I Henter
Jan 23, 2008·European Journal of Clinical Pharmacology·Harald E HeemstraHubert G M Leufkens
Jun 28, 2008·Drug Discovery Today·Harald E HeemstraHubert G M Leufkens
Oct 13, 2009·Drug Discovery Today·Harald E HeemstraRemco L A de Vrueh
May 3, 2011·Nature Reviews. Drug Discovery·UNKNOWN Committee for Orphan Medicinal Products and the European MedicinesChantal Belorgey
Sep 7, 2011·Orphanet Journal of Rare Diseases·Anne E M BrabersRemco L A de Vrueh
Sep 18, 2012·Drug Discovery Today·Pedro Franco
Feb 1, 2014·Nature Reviews. Drug Discovery·Helene LinckerHans-Georg Eichler
Dec 6, 2014·Orphanet Journal of Rare Diseases·Stelios TsigkosBruno Sepodes
Jun 2, 2016·Nature Reviews. Drug Discovery·Segundo MarizBruno Sepodes
Jun 26, 2017·Drug Discovery Today·Stelios TsigkosBruno Sepodes
Oct 13, 2018·Nature Reviews. Drug Discovery·Sudeep PushpakomMunir Pirmohamed

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Citations

Apr 6, 2020·Thérapie·Olivier BlinJoëlle Micallef

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