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Update (January 6, 2013) On The Status Of Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) In The UK

January 6, 2013

       The Orphan Druganaut Blog reports on December 30, 2012, that Vertex Pharmaceuticals announces on December 19, 2012, that a decision is made by the National Health Service (NHS) in England to fund starting January 1, 2013, Kalydeco (Ivacaftor). This decision is only for Cystic Fibrosis (CF) patients in England. There is a separate process for the NHS to decide if CF patients will have access to Kalydeco in Northern Ireland, Scotland, and Wales. These 3 NHS decisions are in the process of being made.

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

The Cystic Fibrosis Association of Ireland (CFAI) announces that the National Centre for Pharmacoeconomics (NCPE) in Ireland, is in the process of evaluating Kalydeco – “Health Technology Assessment (HTA)”.  A decision on Kalydeco is to be made in mid-January 2013.  The HTA is to determine :

1) If Kalydeco is effective

2) Payment for Kalydeco by Health Service Executive (HSE) in Ireland based on an “assessment of the cost effectiveness of the drug”.

The Scottish Medicines (SMC) is due to make a decision on January 14, 2013, on Kalydeco according to a December 31, 2012 CF Trust UK Press Release. 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.

Here is recent media coverage in the UK on the topic of getting approval of Kalydeco in Scotland :

1) Daily Record UK December 31, 2012 article titled, “Family’s frustration as cystic fibrosis wonder drug becomes available for patients in England … but not Scotland”

2) Herald Scotland December 31, 2012 article titled, “Medics back campaign for access to life-saving drug”

3) Daily Mail UK December 31, 2012 article titled, “Woman with cystic fibrosis is denied vital new drug because she live in Scotland …. While her brother gets it as he’s based in England”.


1) January 7, 2013 Vertex Pharmaceuticals presentation, at the 31st Annual J.P. Morgan Healthcare Conference

2) January 12, 2013, CFUnite virtual conference, where Dr. David Sheppard, from The University of Bristol, will discuss Kalydeco. Also, presentation on clinical trial results of Kalydeco for 2 different types of CF mutations. One can access the website, can follow on social media via Twitter at @CFUnite and Facebook/CFUnite

3) January 14, 2013, Scottish Medicines Consortium (SMC) decision on Kalydeco

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

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