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Update (January 22, 2013) On Status Of Vertex Pharmaceuticals’ Orphan Drug Kalydeco In The UK

January 22, 2013

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 did not 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) The Health Technology Appraisal (HTA) is ongoing with the relevant authorities in Wales. CF Trust UK response for approval of Kalydeco in Wales.

4) The National Centre for Pharmacoeconomics (NCPE) in Ireland did not recommend Kalydeco concluding that “ …. in view of the very high drug acquisition cost, the significant budget impact, the absence of long term clinical data and the fact that the company has failed to demonstrate the cost-effectiveness of Ivacaftor we cannot recommend reimbursement …. price of £234,804 (approximately US $372,000) per patient per annum ”. There are an estimated 113 – 120 Irish with the CF gene, G551D, that Kalydeco treats out of a total estimate of 1,200 CF Irish patients.

Media Coverage On Kalydeco In Ireland

1)  Irish Independent January 22, 2013 article titled, “I prepared for the worst but I still cried”

2)  Irish Independent January 22, 2013 article titled, “Drug maker urged to cut price of CF treatment”

3)  Irish Journal article titled, “Negotiations underway over new CF drug deemed too expensive”

4)  CF Association of Ireland response to Kalydeco not being recommended

5)  Dr. Michael Barry of National Centre for Pharmacoeconomics explaining the decision not to recommend Kalydeco on RTE Radio.

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

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