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Rare Diseases And Orphan Drugs: Recent Developments In Ireland

March 25, 2013

The Orphan Druganaut Blog continues its series on international rare diseases and orphan drugs with a look at recent developments in Ireland. Countries that have been presented by the Blog are Australia, Japan, Mexico, UK, and the Middle East.  In Ireland, it is estimated that between 6 – 8% of the population (280,000 – 370,000 people) has a rare disease.

The most recent orphan drug development in Ireland is in relation to the regulatory status of Vertex Pharmaceuticals’ Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) :

1) The Health Service Executive (HSE) in the Republic of Ireland, announces on February 1, 2013, that it will fund Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) patients, ages 6 and older who have at least 1 copy of the G551D mutation

2)  On March 12, 2013, it is announced that the National Health Service (NHS) in Northern Ireland is to provide Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for patients with Cystic Fibrosis (CF), ages 6+ and who have at least one copy of the G551D mutation.

Just this past week in the Irish news, there is a call for “ … builders, trades people, and manufacturers to lend a hand with the construction of Ireland’s first ever National Respite Center for children with rare diseases and their families.” It is being led by Tony and Mary Heffernan, who lost a daughter to Battens Disease and has a son diagnosed with the rare disease. Check out the LiamsLodge website for more information.

Next month in Ireland, April 16 – 17, the International Rare Diseases Research Consortium (IRDiRC) will meet in Dublin. IRDiRC teams up organizations and researchers for the purpose of achieving 2 objectives by 2020 :

1)       To deliver 200 new therapies for rare diseases

2)       Means to diagnose most rare diseases.

A very active organization in rare diseases is the Irish Platform for Patients’ Organizations, Science and Industry (IPPOSI), established in 2001, to “… provide a structured way of facilitating interaction between these three key membership groups and where possible with State Agencies  on policy, legislation, and regulation around the development of new medicines, products, devices and diagnostics for unmet medical needs in Ireland.” IPPOSI publishes, “An Easy Guide to Rare Diseases in Ireland ”. The purpose of the report is to :

1)       Increase awareness of rare diseases

2)       Highlight the priorities that need to be included in the upcoming National Plan for Rare Diseases (NPRD) – to be completed by the Irish Government by 2013

3)       Contribute to development of a National Clinical Program on rare diseases.

The following are several patient advocacy organizations concerned with rare diseases in Ireland :

Ataxia Ireland

Bee for Battens

Cystic Fibrosis Ireland

Cystinosis Foundation Ireland.


Genetic and Rare Disorders Organization (GRDO)

Orphanet Ireland National website

Please Note : “Ireland Map” [Public domain because from US CIA’s World Factbook] | Wikimedia Commons.

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

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