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Kalydeco Triumphs In Wales

May 11, 2013

On May 10, 2013, it is announced that 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, will be made available in Wales. Health Minister Mark Drakeford made the decision after going against advisors. According to a BBC article, approximately 4% of UK patients have the G551D mutation, with about 13 patients (19 patients per CF Trust UK) in Wales who could benefit.

On Wednesday, May 8, 2013, the All Wales Medicines Strategy Group (AWMSG), which advises Mr. Drakeford, announces that it did not recommend approval of Kalydeco for use on the Welsh NHS. AWMSG says the drug is not cost-effective at an estimated cost of up to £180,000 (approximately US $276,000)/patient/year. Mr. Drakeford made the final decision about Kalydeco on Friday, May 10, 2013, making it available on the Welsh NHS. Mr. Drakeford also said according to BBC news, that he is to looking into how such new, innovative medicines for rare diseases can be effectively evaluated in Wales.

Wales is the final UK decision to be made, as Kalydeco funding decisions have already been made in :

•   England
•   Scotland
•   Northern Ireland
•   Republic of Ireland.


CF Unite June 29, 2013, Live Forum, “CF Genes & Gene Therapy”

Cystic Fibrosis Trust UK May 10, 2013, News Release

Daily Post UK article titled, “Celebrations for Cystic Fibrosis Sufferer”

Vertex Pharmaceuticals’ May 10, 2013, Update on the appraisal of Kalydeco in Wales

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

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

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