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Update (January 14, 2013) On Status Of Vertex Pharmaceuticals’ Orphan Drug Kalydeco In The UK

January 15, 2013

The Scottish Medicines Consortium (SMC) made a decision on Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF), according to a January 14, 2013 CF Trust UK Press Release.  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.

About 11% of CF patients in Scotland have the G551D mutation that Kalydeco is approved for. Approximately 80 people in Scotland will be eligible for Kalydeco.

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. This Fund is to “ … cover the cost of medicines for patients with rare conditions (“orphan drugs”), which are not available for routine prescription …. Only those medicines not recommended for routine use by the Scottish Medicines Consortium will be covered by the fund ….. “.  In the Scottish Government’s Press Release, an orphan drug is defined for illnesses which “affect fewer than 1 in 2,000 people”.

Media Coverage On Kalydeco In Scotland

***  1)  Scottish TV, “Decision by SMC on Assessment of Kalydeco”

2) January 14, 2013 article titled, “Patients suffering from rare conditions to get vital treatment after wonder-drug fund approved”

3)  BBC News January 14, 2013 article titled, “Scottish orphan drugs fund launched”

4) January 15, 2013 article titled, “£21M Fund to Tackle drugs postcode lottery facing patients”

5) January 15, 2013 article titled, “£21M Heal Deal – New fund to fork out on pricey wonder drugs”.

Regulatory Status Of Kalydeco In UK

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

–   Mid-January 2013, National Centre for Pharmacoeconomics (NCPE) in Ireland decision on Kalydeco

–  The Health Technology Appraisal (HTA) is ongoing with the relevant authorities in Wales.


Scottish Government January 1, 2013, Announcement of Creation of the £21 Million Rare Conditions Medicines Fund

The Scottish Medicines Consortium (SMC) Decision on Kalydeco ­

Vertex Pharmaceuticals January 14, 2013 Statement on Scotland’s Appraisal of Kalydeco.

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

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