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Kalydeco Triumphs In Ireland

February 1, 2013

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. The decision follows an assessment by the National Centre for Pharmacoeconomics (NCPE). NCPE acknowledges the benefits of Kalydeco. This decision follows an onoing lengthy regulatory and assessment process for making Kalydeco available to patients in the UK – Republic of Ireland, England, Wales, and Scotland.

Kalydeco funding will help approximately 120 people suffering from CF in Ireland who have at least 1 copy of the G551D mutation. Minister for Health James Reilly says that a provision has been made for “EUR20 million plus funding a year” to provide for the supply of Kalydeco, which will become available starting March 1, 2013. Per the Independent, Kalydeco is to cost cost €234,000/patient/year.

Regulatory Status Of Kalydeco In UK

1) NHS in England decides to provide funding starting January 1, 2013; reference CF Trust UK statement on receiving Kalydeco in England

2) The SMC doesn’t recommend Kalydeco for use within NHS Scotland because the cost outweighs the benefits. Vertex Pharmaceuticals will resubmit to SMC for reassessment. Kalydeco costs £182,000/patient/year in Scotland. In response to the SMC’s negative recommendation, the Scottish Government announces the creation of the £21 Million Rare Conditions Medicines Fund which will be available from March 1, 2013 – April 2014 and which will cover the cost of Kalydeco for qualified CF patients

3)  Kalydeco now funded in the Republic of Ireland

4) The Health Technology Appraisal (HTA) is ongoing with the relevant authorities in Wales. CF Trust UK response for approval of Kalydeco in Wales.

Media Coverage On Kalydeco In Ireland

1) February 1, 2013 article titled, “Fundng Approval for Life-Changing CF Drug”

2) February 1, 2013 article titled, “CF wonder drug Kalydeco sanctioned after deal on price”

3)    RTE News Ireland 1,February  2013 article + 2 Videos titled, “Kalydeco drug cleared for cystic fibrosis patients”

4) February 1, 2013 article titled, “Kalydeco to be made available to Cystic Fibrosis patients in Ireland”.


Ireland’s Department of Health February 1, 2013 Announcement for Kalydeco availability

Vertex Pharmaceuticals February 1, 2013 Press Release titled, “Republic of Ireland Approves Funding for Kalydeco (Ivacaftor) ….”.

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

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