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Orphan Drugs And Rare Diseases: Global News (04.22.13)

April 22, 2013

Recent orphan drug and rare disease global developments are in the news. Here is a list and description of recent developments :

I – Europe

The European Medicines Agency (EMA) publishes on April 16, 2012, its 2012 Annual Report that highlights the main trends recently observed in the Agency’s activities. Some of the numbers for orphan drugs are as follows :

•    36% increase in # of Marketing Authorization Applications (MAAs) for medicines with orphan designation in 2012 compared with 2011
•    148 orphan designations granted in 2012 compared to 107 in 2011; 38% increase
•    > 150 orphan designations are expected in 2013
•    Most-represented therapeutic areas for orphan designation were oncol­ogy (35%) & metabolic diseases (17%)
•    72% of medicines receiving orphan designation concern conditions affecting children.


The National Institute for Health and Clinical Excellence (NICE), the health technology appraisal body in England and Wales, issues a recommendation in March 2013 for InterMune’s orphan drug Esbriet (Pirfenidone) for the treatment of a rare lung disease, Idiopathic Pulmonary Fibrosis (IPF). According to InterMune, there are approximately 6,100 – 9,500 IPF patients living in England and Wales.

The National Centre for Pharmacoeconomics’s (NCPE) in Ireland decides not to make orphan drug Esbriet available to sufferers of IPF because of its high cost, €32,700 (approximately US $43,000) per patient per year. The Irish Lung Fibrosis Association (ILFA) issues a statement in response to the NCPE decision. The number of patients anticipated to receive the drug is 97 in year one, rising to 207 after five years. The Health Service Executive (HSE) is in negotiations with the manufacturers of Esbriet.

The International Rare Diseases Research Consortium Conference (IRDiRC) is held in Dublin, Ireland, April 16 – 17, 2013 :

•    PatientCrossroads announces the partners that are selected for the CONNECT open-access patient registry
•    The EspeRare Foundation is presented to the public.

III – Japan

According to Elsevier Business Intelligence (may need subscription), Shire is opening an office in Japan with the aim of selling two drugs, including Gaucher’s Disease drug within 2 years.

IV – Nepal

Efforts are under way in Nepal to build a rehabilitation center for Muscular Dystrophy patients. The center will be established by a charity organization, Muscular Dystrophy Foundation (MDF). It is estimated that there are 5,000 – 7,000 suffering from Muscular Dystrophy in Nepal, with Duchenne Muscular Dystrophy (DMD) being the most common among Nepali children.

Please Note: “Globe” by Augiasstallputzer at Wikimedia Commons [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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