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Kalydeco: Disappointment In Scotland

June 12, 2013

Access to Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for patients with Cystic Fibrosis (CF) in the UK – Republic of Ireland, England, Wales, and Scotland – is an ongoing, lengthy regulatory and assessment process.

In January 2013, the Scottish Medicines Consortium (SMC) doesn’t recommend Kalydeco for use within NHS Scotland because the cost outweighs the benefits. 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. Vertex Pharmaceuticals decides to resubmit to the SMC for the reassessment of Kalydeco.

Following Vertex Pharmaceuticals resubmission to the SMC for Kalydeco, the SMC publishes its opinion on June 10, 2013, after its second assessment. The SMC once again does not recommend the drug for use within NHS Scotland:

Ivacaftor has demonstrated superiority over placebo measured by absolute change in forced expiratory volume in one second (FEV1) % predicted in two Phase III, double-blind randomized studies. The submitting company’s justification of the treatment’s cost in relation it its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic assessment to gain acceptance by SMC.

In response to SMC’s second assessment, Vertex Pharmaceuticals releases a June 11, 2013 statement expressing their disappointment and stating that:

Today’s decision will not impact people with CF in Scotland who are eligible for Ivacaftor, as their current and future ability to get Ivacaftor is assured through the individual patient treatment request scheme, as was announced by the Scottish Government on 14 January 2013. These patients are currently funded by the Scottish Rare Conditions Medicines Fund, announced on the same day.” 

References

SMC’s Detailed Advice Document for 2nd Assessment

Vertex Pharmaceuticals’ June 11, 2013 Update On Second Assessment of Kalydeco in Scotland.

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

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

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