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A Decade Of Orphan Drug Legislation In The EU: Journal Article

November 2, 2012

       A recent journal article analyzes and reviews what the possible reasons are for the small number of drug designations, approvals, and refusals/withdrawals for orphan drugs in the EU, between 2000 – 2010. The authors used the European Medicines Agency (EMA) website and the European Public Assessment Reports as a source of their analysis. The authors are from the Laboratory of Drug Regulatory Policies in Milan, Italy. 

       Some statistics the authors found : 

–  From 2000 – 2010, 80.9% of 845 orphan drug candidate designations receive a positive opinion from EMA’s Committee on Orphan Medicinal Products (COMP) 

–  Of the 108 Orphan Medicinal Product (OMP) marketing authorizations applied for, 63 or approximately 58% are granted 

–  33% of the OMPs are tested in trials involving < 100 patients & > 50% in trials with only 100 – 200 cases 

–  42.9% of approved OMPs have clinical trials lasting < 1 year.

        The authors conclude that “ …. the # of patients studied, the use of placebo as control, the type of outcome measure and the follow-up have often been inadequate… to find better ways of fostering the development of effective & sustainable treatment for patients with orphan diseases. Public funds supporting independent clinical research on OMPs could bridge the gap between designation and approval  ……”. 

        The PubMed Abstract and citation :  

–      Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.  Joppi R, Bertele V, Garattini S.  Eur J Clin Pharmacol. 2012 Oct 23. [Epub ahead of print]. PMID: 23090701 [PubMed – as supplied by publisher].

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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