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7 Newest Publications about the topic orphan drug designationrss
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05-Aug-2017 | Joëlle Micallef, Pierre Boutouyrie, Olivier Blin, Fundamental & Clinical Pharmacology, 2017
Abstract Developing drugs for rare disease can be challenging due to specific rare disease characteristics. The French Medical Pharmacology is structured and positioned to play a major role in Orphan Drug Research and Development due to the required expertise concentrated into pharmacology ...
15-Jul-2016 | Segundo Mariz; James H. Reese; Kerstin Westermark; Lesley Greene; Takahiro Goto; Tatsuro Hoshino; Jordi Llinares-Gar ..., Nature Reviews Drug Discovery, 2016
Nature Reviews Drug Discovery 15, 588 (2016). doi:10.1038/nrd.2016.143 Author: Segundo Mariz, James H. Reese, Kerstin Westermark, Lesley Greene, Takahiro Goto, Tatsuro Hoshino, Jordi Llinares-Garcia & Bruno Sepodes
14-Oct-2014 | Michael Schlander; Charles Christian Adarkwah; Afschin Gandjour, Expert Review of Pharmacoeconomics & Outcomes Research, 2014
Background/aim: Ultra-orphan diseases (UODs) have been defined by a prevalence of less than 1 per 50,000 persons. However, little is known about budget impact of ultra-orphan drugs. Methods: For analysis, the budget impact analysis (BIA) had a time horizon of 10 years (2012–2021) and a ...
22-Oct-2012 | Roberta Joppi, Vittorio Bertele’, Silvio Garattini, European Journal of Clinical Pharmacology, 2012
Purpose To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number of products licensed. Methods Information about orphan drug designation, approval, refusal or withdrawal was obtained from the website of the European ...
29-Apr-2011 | The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat, Nature Reviews Drug Discovery, 2011
European regulation on orphan medicinal products: 10 years of experience and future perspectives Nature Reviews Drug Discovery 10, 341 (2011). doi:10.1038/nrd3445 Authors: The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat ...
01-Apr-2011 | Dupont, Alain G.; Van Wilder, Philippe B., British Journal of Clinical Pharmacology, 2011
WHAT IS ALREADY KNOWN ABOUT THIS SUBJECT • The clinical evidence level of orphan medicines in European market authorization submissions is low: few randomized controlled trials, short treatment follow‐up, rarely hard clinical endpoints. • The success rate at centralized market ...
07-Mar-2011 | Monica Heger, Nature Medicine, 2011
Despite surge in orphan drug designations, approvals still lag Nature Medicine 17, 236 (2011). doi:10.1038/nm0311-236b Author: Monica Heger