Cystic fibrosis drug Kaftrio approved for Wiltshire toddler

Alice says it's "incredible news" her daughter will have access to Kaftrio next year

A family say they are "over the moon" that their three-year-old daughter will be given a new "life-prolonging" cystic fibrosis medication.

Olive, from Wiltshire, is only expected to reach her mid-30s, but it is hoped the drug, Kaftrio, will change that.

Kaftrio costs the NHS up to £200,000 per year for each patient and may not be available on the NHS for much longer due to the cost.

Olive's mum, Alice, said: "What is the value of life?"

She told BBC Radio Wiltshire that their "world was crumbling" when they were first told Olive's life expectancy, with no hope of treatment.

'Life-changing drug'

"It was just devastating to learn that there was this life-prolonging drug out there that we could not have," the mum from Ashton Keynes said.

"It is a lot of money and it is a struggle with the NHS, with resources and funding, but when it is your daughter and you are in this position you would do anything to fight to get what they need."

Olive, who received approval for the drug on Monday, is expected to begin treatment early next year, funded by the NHS.

Alice is hopeful that a normal life is in store for her daughter, but said it was "really upsetting" and unfair for those people and children who could face not having access to the "life-changing drug".

Kaftrio was recently licensed for children two to five-years-old

Kaftrio has previously been available for some adults and the Medicines and Healthcare Products Regulatory Agency recently extended the license for children aged two to five-years-old.

But its use is being reviewed by the National Institute for Health and Care Excellence (NICE), which is consulting on whether it is too expensive to prescribe to new patients, at a cost of £8,346 for a packet of 56 tablets.

Helen Knight, director of medicines evaluation at NICE, said it was about ensuring taxpayers "continue to get value for money".

"We continue to work with the company, NHS England and other stakeholders including the Cystic Fibrosis Trust to deliver the best outcome both for people with cystic fibrosis and for the wider NHS," she said.

What is cystic fibrosis?

• Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system

• It can cause lung infections and problems with digesting food

• Symptoms usually start in early childhood and vary for each individual, but the condition gradually gets worse over time

• There is no cure for the condition, but a range of treatments can help control symptoms and reduce complications

• Currently, about half of people with cystic fibrosis will live past the age of 40. Children now born with the condition are likely to live longer than this

Source: NHS

The Cystic Fibrosis Trust has been urging people to have their say before the NICE consultation ends at 17:00 GMT.

Chief executive, David Ramsden, said: "We must never return to a situation where people with cystic fibrosis die far too young, knowing there's a treatment that could change that."

NICE said existing patients on Kaftrio would still get the treatment regardless of its decision next year.

A Department of Health and Social Care spokesperson said: "Cystic fibrosis can be a devastating illness, which is why we have invested over £1.1bn of funding into research of rare diseases.

"It is vital that patients have access to new and innovative medicines like these, but the NHS must use its budget fairly and for the good of all patients".

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