Mother of cystic fibrosis sufferer demands access to new drug

Reporter

Tuesday 9 October 2012

THE mother of a young cystic fibrosis sufferer is calling for a life-saving new drug to be introduced in Scotland.

CAMPAIGN: Marion Ferguson believes the new drug Kalydeco could transform the life of her son, Thomas, 15. Picture: James Galloway SUPPORT: Michael McMahon.

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VICTORIA WELDON

Marion Ferguson, whose 15-year-old son Thomas suffers from the disease, wants Kalydeco, also known as ivacaftor, to be approved by the Scottish Medicines Consortium.

The drug is the first of its kind to treat the cause of a certain strain of cystic fibrosis called G551D – known as the "Celtic gene" because of its prevalence in people of Celtic origin – rather than just the symptoms.

The disease is caused by a gene mutation and the new treatment – already used in the US and some European countries – targets this.Patients who have used the drug have reported remarkable results. Some whose only hope of recovery was a lung transplant are reported to be leading active lives just weeks after starting treatment.Mrs Ferguson, 44, of Hamilton in South Lanarkshire, who runs the Ivacaftor Patient Interest Group, said: "The horrible thing about this disease is it affects young people and it's just soul- destroying to think there's something out there that could help them and we can't get at it."We're watching other people benefit from a drug we know our children desperately need."Thomas was diagnosed with cystic fibrosis at 14 months and has been receiving regular intravenous antibiotics ever since.Mrs Ferguson, whose campaign has received the backing of her MSP, Michael McMahon, claims the drug would transform her son's life.She said: "Thomas is in and out of hospital. He has to take pills everyday. He gets intravenous antibiotics. He uses a nebuliser. All while trying to study for his standard grades."Cystic fibrosis causes damage to the internal organs. The longer Thomas doesn't get the drug, the more damage that's going to be done, and it's irreversible."Kalydeco was licensed by the European Medicines Agency in July this year and has been approved by the US Food and Drug Administration. It is being considered by the Scottish Medicines Consortium and a decision is expected in January.However, the cost of the drug is high, with manufacturer Vertex pricing it at £182,635 a year, or £15,218.75 per person per month.Mr McMahon, MSP for Uddingston and Bellshill, said: "When you're a parent of a young person who could be given hope when at the moment the prognosis and prospect of living a long life is not great, of course you would fight to get this drug."We need to get it into the system so it's available."A total of 888 people in Scotland have cystic fibrosis. Of these, 638 have been genotyped and 72 have the G551D gene mutation. This means that in Scotland a higher percentage of cystic fibrosis sufferers (11.3%) have this strain compared to the rest of the UK (5.7%).A Scottish Government spokeswoman said: "Scotland has robust and transparent arrangements to appraise newly licensed medicines for clinical and cost-effectiveness through the Scottish Medicines Consortium (SMC) and Healthcare Improvement Scotland. "An application for approval of this drug is with the SMC and it will respond in due course."

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Mother of cystic fibrosis sufferer demands access to new drug

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