A FROME mum whose two-year-old daughter has cystic fibrosis is urging residents to take part in a petition to save a ‘life saving’ medication from being scrapped for new patients.
The National Institute for Health Care Excellence (NICE) recently said it was ‘evaluating the cost-effectiveness of medicines’ as it says modulator medications for cystic fibrosis (CF) like Kaftrio, have become expensive to deliver on the NHS.
The medication is currently prescribed for children over the age of two, but a consultation by health bosses is considering whether the drug is too expensive for new patients. Kaftrio can cost £100,000 a year to treat patients with CF, which causes a sticky mucous to build up in the lungs and digestive system, significantly shortening life expectancy for many.
Uncertain
“Everyone already on the drug can stay on it for life, but anyone under the age of two that is not already on it will not be able to have it,” says Jade Wilson, whose daughter Callie has the genetic condition.
“My daughter, two years old in January, is supposed to be starting the Kaftrio drug then, but it is uncertain at this current time whether she will be able to start it and to be honest, it is unbearable to even think about.
“She is such a delightful, beautiful, clever girl, she means so much to us, she deserves to have a life that is not cut short because of money.
Miracle drug
“I understand it is a lot of money but with poorly managed CF it’s going to cost the NHS a lot of money with all the unwell children and hospital stays and other medications, so they may as well keep the miracle drug and keep everybody healthy.”
“Kaftrio, is a miracle drug, the closest thing to a cure,” explains Jade. “If this drug is taken away then toddlers and babies with this condition face a poor quality of life, shorter life expectancy, lung infections, digestive problems, breathing problems, long hospital stays and the list goes on. My whole world will fall apart, my whole family’s world will be ripped apart.”
Two other drugs to treat CF, Symkevi and Orkambi, are also at risk of no longer being delivered by the NHS. NICE is currently holding a consultation before deciding on whether the drugs can continue to be delivered on the NHS.
Chief executive officer for the Cystic Fibrosis Trust, David Ramsden said, “NICE’s initial recommendation today that the modulator drugs Kaftrio, Orkambi and Symkevi are highly effective for people with cystic fibrosis, but just too expensive to be available on the NHS, is disappointing news.
“It is important to emphasise that those already taking any of the modulator drugs are not affected by the NICE process because of the agreements already in place, but this update creates uncertainty for those not yet on treatment. Vertex [pharmaceuticals], NICE, and the NHS must now urgently work together to find a solution to make these treatments available for all those who could potentially benefit.
“We must never return to a situation where people with CF die far too young, knowing there’s a treatment that could change that.”
Petition
To take part in the petition, that aims to gain 100,000 signatures to be taken to parliament visit: https://petition. parliament.uk/petitions/650674
