Little Maisie Black could be set to receive the wonder drug that will transform her life.
The Burnside seven-year-old suffers from a rare form of cystic fibrosis.
Her parents Tilda and John of Glenlui Avenue, want her to have a drug, kalydeco, which they hope will improve her life significantly.
On Monday 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.
It costs £180,000 per patient each year but is already available through the NHS in England.
The decision was a setback for Maisie and her parents John and Tilda of Glenlui Avenue.
However, on same day the couple were contacted by Health Secretary Alex Neill and later he said they could make an “individual patient request” for the drug.
Funding for the medicine could come from a special fund set up this week to help patients with rare diseases.
“If clinicians and the health board think she would benefit from the drug then she should get full access to it and money is not going to be a problem,” Mr Neill added.
Mrs Black said they were now hopeful that those wanting the drug would get it but she said they would only be happy when they had the drugs in their hand.
Maisie was three months ago diagnosed with a rare form of cystic fibrosis known as the G551D gene mutation.
CF is one of the UK’s most common life-threatening inherited diseases. It damages the organs, especially the lungs and digestive system, by clogging them with sticky mucus.
As a result of the condition, Maisie, who attends Burnside Primary, has to undergo physiotherapy every day and as take 40 tablets to manage the symptoms of the disease.
Although not a cure, it is hoped kalydeco could improve Maisie’s life significantly.
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”.
The Scottish Government have set up a £21m fund to help individual patients with rare conditions who require drugs not available for routine prescription.
Speaking to the Refomrer Maisie’s mum Tilda said: “We’re devastated about the result but it wasn’t unexpected, we knew what the decision was going to be.
“Alex Neil phoned us yesterday afternoon to ask after Maisie and explain about the drug fund that had been launched parallel to their refusal. I think (by announcing the drug fund) he went some way to ensuring patients get the drug.
“However I don’t think it’s far enough for what’s needed in Scotland.”
James Kelly, who raised the issue with Mr Neil in the Scottish Parliament yesterday (Tuesday), said: “The handling of this entire situation is hugely concerning.
“That the family had their hopes raised over the festive period and then dashed in the New Year just strikes me as utterly senseless.
“This decision is a blow to cystic fibrosis sufferers who need Kalydeco to provide much needed relief from their condition. It is unacceptable that the treatment is available in England but not in Scotland.
“It is time for Alex Neil to stand up be counted and intervene.”
A Scottish Government spokesman said: “The SMC operate independently of Ministers. There is a review underway to consider how new medicines are appraised and accesses in Scotland. “Medicines such as this will be made more accessible through the new £21 million Rare Conditions Medicines fund launched.
“The new fund will cover the cost of successful individual treatment requests for medicines for patients with rare conditions, which are not otherwise available for routine prescription.”
A spokesperson for the Scottish Medicines Consortium said: “Ivacaftor is a promising new medicine designed to address a gene mutation in some CF patients.
“The company’s own evidence on the medicine’s benefits is based on a well designed short-term study of less than one year but the company used this study to propose an increase in life expectancy of 18 years. SMC considered this estimation to be highly uncertain. The cost of ivacaftor in NHS Scotland would be around £180,000 per year for each patient for life.”
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