Kalydeco set to be made available for Rutherglen girl Maisie
Jan 30 2013
by Will Henshaw, Rutherglen Reformer
Rutherglen girl Maisie Black has been assured she WILL receive a drug that could transform her life.
The Burnside seven-year-old, from Glenlui Avenue, suffers from a rare form of cystic fibrosis.
Kalydeco, which is also known as ivacaftor, helps sufferers who have the G551D gene mutation. Around four per cent of CF patients worldwide have the gene but in Scotland that figure rockets to a 11 per cent, hence it is also known as the “Celtic mutation”.
However, as previously reported in the Reformer, the Scottish Medicine Consortium, who advise NHS boards on the effectiveness of newly-licensed drugs, took the decision not to recommend Kalydeco for the NHS in Scotland.
But the drug will be funded through the new £21million fund to help improve access to drugs for people with rare medical conditions, health secretary Alex Neil MSP said.
Maisie’s mum Tilda said: “We are very grateful to Alex Neil for meeting with the Ivacaftor Patient Interest Group (IPIG), CF Trust and myself and my MSP, James Kelly. We are also hugely grateful to him for recognising and eliminating the application problems of the Individual Patient Treatment Request (IPTR) route for Kalydeco. He has assured us that the funding will be available for all G551D CF patients across Scotland from March 1. He struck me as a very genuine and caring man and we can’t thank him enough for his help with this matter. “
Alex Neil and the Ivacaftor Patient Interest Group issued the following joint statement: “The Scottish Government has recently announced the introduction of a £21m fund to cover the cost of orphan medicines for individual patients with rare conditions, which are not available for routine prescription.
“Access to these rare medicines is currently available through the Individual Patient Treatment Request (IPTR) process.
“However, we recognise the fact that as Ivacaftor benefits 100 per cent of the patients with the ‘Celtic gene’ or G551D genetic mutation, it presents a challenge to the IPTR process as it stands.
“That is why the Scottish Government will take swift action to address how best to ensure that where specialist clinicians are supportive of prescribing Ivacaftor, the IPTR process does not present a barrier to accessing it. Guidance on the prescribing of Ivacaftor will be issued to all NHS Boards. The Cabinet Secretary was clear that patients with the G551D gene mutation should not end up competing against each other for access to medicines.”
MSP James Kelly added: “I’m delighted that Maisie will now receive Kalydeco. I know that this will come as a great relief to her family.”