THE mum of a 9-month-old baby with Cystic Fibrosis has urged the Scottish Government to act after a drug that could help her son was rejected for use on the NHS.
Louise Whytock said she felt “disheartened” after Ivacaftor-lumacaftor (Orkambi) which treats the underlying causes of the life limiting condition, and would be suitable for her son Ellis when he is older, was rejected by the Scottish Medicines Consortium (SMC) for use on the NHS.
It is the first drug of its kind which has been shown to improve lung function, reduce outbreaks of infection and cut hospitalisations by over 60%.
The drug is suitable for children, aged over 12, with the most common mutation, F508del - around 70% of cases and costs around £104,000 per year.
The treatment, manufactured by Vertex, was turned down by the SMC due to cost and uncertainty around long-term impact. It will not be available through the New Medicines Fund either, which offers support to health boards to fund medicines for rare diseases.
Louise Whytock’s nine-month-old son Ellis with diagnosed with Cystic Fibrosis when he was three weeks old.
Louise, 36, from Bearsden, said her and husband Craig, 43, are still struggling to come to terms with the diagnosis.
Louise, who is also mum to Keir, 4, said: “Our son Ellis was diagnosed with CF at three weeks old and it has been a terrible time for us.
“We have huge feelings of guilt, anger, worry and fear for his future.
“Ellis has the mutation which would make him suitable for Orkambi when he is older.
“Although he is too young for it yet, having that arsenal of drugs available is quite comforting to know.
“For us not to have that as an option is disheartening.
“I understand the cost is huge but when it’s your son, you can’t think about money. When they are doing all this research if the drugs aren’t going to be available, it’s like holding a carrot up and then taking it away.
“We are adjusting to it but we will do anything to help improve the quality of his life.
“I would like the SMC to know how much this condition impacts our whole family, not just Ellis.
“Ellis currently has a demanding and exhausting routine just to stay well - with five medications, physio sessions, clinic appointments.
“Sometimes it feels like our lives revolve around these essential treatments.
“Orkambi could change our lives and I urge all parties to reconsider this decision so that our son can live a life without the limits.”
In Scotland, one child in every 2,345 people is born with cystic fibrosis.
The condition mainly causes difficulty breathing and lung infections but also affects the liver, pancreas and intestines.
People diagnosed with the condition have a drastically reduced life expectancy, although it is improving thanks to medical advances.
Another drug, Ivacaftor (Kalydeco) which has been shown to slow the progression of the progressive lung condition, was turned down for patients under six.
Kalydeco has been shown to improve lung function and studies showed it led to a “dramatic” improvement in pancreatic function.
It is currently available to children over 6 years in Scotland with the G551D mutation via the new medicines fund, which offers support to health boards to fund drugs for rare conditions. It costs around £182,000 per year of treatment.
The Cystic Fibrosis Trust has written to the Secretary for Health, Wellbeing & Sport, Shona Robison, urging the NHS, the drug company and the New Medicine Fund to come together to find a way forward.
Ed Owen, chief executive of the Cystic Fibrosis Trust said: “People with Cystic Fibrosis in Scotland must not be denied Orkambi because of a battle between the NHS Scotland and Vertex over price and longer term impact.’
“Vertex, the NHS in Scotland and the New Medicines Fund must come together and make this happen.”
“Kalydeco is already being provided by the New Medicines Fund for those eligible for it and over six years old.”
A Scottish Government spokesperson said: “The £90 million New Medicines Fund was put in place to fund treatments for rare or end-of-life conditions. “Pharmaceutical companies also need to price their drugs fairly – we would encourage SMC and the pharmaceutical company to work together to find a way forward.
“There is also currently an independent process underway to review the system of access to new medicines in Scotland, which will look at what more can be done to build on the improvements already achieved and ensure all patients get timely access to newly licensed medicines, at a fair price for the NHS.”
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