PARENTS of children suffering from a distinct “Celtic” form of cystic fibrosis have made a last-minute plea for a drug that treats the condition to be made available in Scotland.
The Scottish Medicines Consortium (SMC) which rules on the availability of drugs in Scotland, is to reveal tomorrow whether they have decided to issue the life-transforming medicine – Kalydeco – on the NHS.
Approved for use in England three weeks ago, it has been hailed as a “game-changing” treatment for one specific strain of the disease, with high incidence in Scotland and Ireland.
But Scots families fear the drug will be barred north of the border because of the huge £182,000 a year cost per patient.
If so, families say they will take their fight to the Scottish Parliament this week. They are already calling on Health Secretary Alex Neil to abandon protocol by committing to over-rule the SMC decision if required.
The G551D form of the disease affects 12 per cent of CF sufferers in Scotland – around 80 people and double the proportion of those affected in England. Incidence is even higher in Ireland, the conclusion being it predominantly affects Celtic people.
Three of the five children in the West of Scotland who are known to be affected by the G551D strain were admitted to Yorkhill hospital in Glasgow at the end of last week having all suffered complications.
CF is one of the UK’s most common life-threatening genetic diseases which damages the lungs and digestive system. Symptoms include difficulty breathing and lung infections and sufferers can need lung transplants to survive.
Last night, one parent said First Minister Alex Salmond should intervene tomorrow if necessary, and that ministers should not sit back and deny Scottish children being treated for a problem which impacts them more than those elsewhere in the UK.
Dr Jonny Gordon, whose daughter Kate, aged 7, had to be admitted to hospital on Friday, said: “He can’t have the English treating the Celtic problem, but not us.”
Ed Owen, chief executive of the Cystic Fibrosis Trust, added: “This drug is transformational and offers real hope of a longer and better life and it will be appalling in the extreme if people in Scotland were denied a drug on the NHS which is available elsewhere in the UK.”
Kalydeco is available in England where the cost of supplying it to 270 eligible patients will account for about half the NHS budget for cystic fibrosis.
The concern follows several high-profile cases in which the SMC has been involved in evaluating drugs already approved in England. Two cancer drugs, Iressa and Zelboraf, have both been rejected for NHS funding.
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