Cystic fibrosis drug to change child's life

Matilda would live for longer and suffer from infections less, her mother said

A four-year-old girl from Devon who has cystic fibrosis will live a longer, healthier, happier life now that a drug is available to her on the NHS, her parents say.

Matilda Baltchford will soon be able to have Kaftrio prescribed as it has been licensed for children over two years old.

However, the National Institute for Clinical Excellence (NICE), which guides the NHS, is consulting on the use of Kaftrio for future patients due to its cost.

NICE said existing patients would still get the treatment regardless of its decision. 

Rose Blatchford said it was "disgusting" that future children may not get access to the drug on cost grounds

Matilda's mother Rose Blatchford said: "The drug she is taking at the moment or can be, generally, only treats the symptoms of the condition - it does an OK job.

"Kaftrio will totally purge out whatever's in her lungs; it will clear out any infection, help her to gain weight easier, just give her a longer, more free, happier, healthier life."

She went on: "Quality of life as well, it's less hospital admissions ... She'll be able to have children when she's older, she'll be able to retire.

"It's going to be amazing, it's going to be the best Christmas present ever."

However, she said she would continue to "keep fighting" for children who may not be able to access it in future, depending on NICE's decision.

"It's disgusting - we know there is a drug available that is going to prolong their lives and the thought that they're not going to get it is just wrong," she said.

Scott Blatchford said: "It's life-changing, it's a drug that works - it's proven, so we are hoping a deal can be agreed".

'Cost-effectiveness'

The drug costs £8,346 for a packet of 56 tablets, according to NICE.

It means NHS treatment costs could amount to between £100,000 and £200,000 per year for each patient.

Helen Knight, director of medicines evaluation at NICE said: "We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure that taxpayers continue to get value for money."

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".

David Ramsden, CEO of Cystic Fibrosis Trust, 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.”