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7 Newest Publications about the topic orphan drug designation

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Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology

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 ...

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Worldwide collaboration for orphan drug designation

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

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Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe

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 ...

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Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

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 ...

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European regulation on orphan medicinal products: 10 years of experience and future perspectives

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 ...

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Access to orphan drugs despite poor quality of clinical evidence

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 ...

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Despite surge in orphan drug designations, approvals still lag

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

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