Leeds mum devastated that daughter's cystic fibrosis 'miracle drug' Kaftrio could be denied to other families

Nellie Watson, six, was born with cystic fibrosis, a rare condition that causes sticky mucus to build up in the lungs and digestive system.

The condition causes lung infections and problems with digesting food. And while there are treatments for its symptoms, it has ultimately meant a significantly shorter life expectancy for many of its sufferers.

However, a drug that was made available to children over the age of six that has been proven to boost lung function and overall quality of life.

That drug is Kaftrio, which Nellie has just been given access to for the first time in the last few weeks. The treatment has been a breakthrough for the youngster and her family.

Mum Kirsty, 34, explained: "I gave birth to this beautiful baby girl and at four weeks old we were told that she'd only live to 40. That's no life at all.

"So, to find out that there was this miracle drug that could massively improve her life expectancy changed everything."

But despite its significant benefits, it emerged this week that the drug might not freely available for much longer.

The National Institute for Health and Care Excellence (NICE) has said the drug is too expensive to be recommended for use on the NHS and that there would be a month-long consultation to decide whether or not it should still be prescribed.

That uncertainty has caused great anxiety for many parents – particularly for those with children younger than six-years-old who were expecting to have access to Kaftrio in the near future.

Children like Nellie will continue to be prescribed the drug, as they are already in receipt of the medication. But for those who did not get to experience its effects, there is a chance they may never be given the treatment on the NHS.

Kirsty, from Roundhay, explained how it felt to learn that hundreds of families could be deprived of the treatment that she said has worked wonders for her daughter.

"It breaks my heart," she said. "I can see what other parents could have had, because I have it with Nellie. I see her improving every day."

Nellie has been using Kaftrio for a matter of weeks and already her family have noticed improvements.

Recently, she developed a viral infection that kept her off school. A doctor told her mum that while other children who suffer from cystic fibrosis have been hospitalised with the same infection, Nellie has been able to recover at home.

Kirsty, who is a full-time carer for her daughter, is among many parents calling for the price of Kaftrio to be brought down. A petition against NICE’s recommendation has accumulated hundreds of thousands of signatures.

The last dates for comments on NICE’s consultation is Friday (November 24) and a final decision on the funding of the drug is expected to be made soon after.