Hope For Harley: Luton mum shares worries about son’s future as ‘miracle’ cystic fibrosis drug funding could be scrapped

It was once hailed as a ‘miracle’ drug by the NHS, but now funding for life-changing Kaftrio medication could be snatched away from little Harley Whiting.

The Luton toddler is only 19 months old and has cystic fibrosis. His family had hoped the drugs would prevent the boy from constantly “battling infections”, but draft guidance from the National Institute for Health and Care Excellence (NICE) could mean he never has access to this medicine.

Harley’s mum, Bethany, fears the country will be “going back 50 years” if vital funding for modulator drugs for cystic fibrosis patients is withdrawn by NICE. The body, which looks at the cost-effectiveness of medicines provided by the NHS, closed its consultation at the end of November as it evaluates the drugs that have been a lifeline for so many already.

She said: “I just don't understand how they can have this drug that is saving lives, and then they're going to stop the funding. Yes, so many people have accessed it already. But there's going to be so many babies born who won't have access to this treatment, and it's not right to deny parents and families of these children access to these life-saving drugs.”

NICE are set to meet on December 14 for a committee meeting, when a decision is expected to be made. Under the draft guidance, people who are taking the drug will be allowed to carry on taking it, but no new patients will be able to get it through the NHS.

Last month, the Medicines and Healthcare Products Regulatory Agency extended the licence of the cystic fibrosis medicines Kaftrio and Kalydeco for children aged two to five years old. This means that if funding is kept the same, Harley could receive the life-enhancing and prolonging drug as early as April next year, when he turns two.

This would mean the world for Harley’s family who monitor him closely every day. His parents do physiotherapy with the tot, weigh out his food, and give him his nebuliser and preventative antibiotics. Mum said: “Cystic fibrosis is a hidden disability. The only difference [between him and other children] is his routine and how we protect him.”

She added: "When he was born, we just assumed that he's going to have a really short life. Then one of the nurses said that there was a drug called Kaftrio. We thought that Harley would have an almost normal life. Then the news came from NICE and our world turned around again."

Currently, about half of people with cystic fibrosis will live past the age of 40 and children born with the condition nowadays are likely to live longer than this. However, a person with cystic fibrosis taking Kaftrio could live until around 80 years old.

The condition means that the toddler lives with sticky mucus that builds up in his lungs and digestive system, making him more susceptible to infections. Despite this, he is a happy chap, Bethany explains: “He's a really, really active toddler who loves to run, but that actually helps him with his physio, because any sort of exercise is really good for their lungs.”

Without funding, the chance of a longer and brighter future is cruelly being taken away. Kaftrio, which is manufactured by American pharmaceutical company Vertex, costs £8,346 for a packet of 56 tablets. Harley would need to take two of these a day - meaning NHS treatment could tally up to more than £180,000 per year.

Bethany said: “How can anyone afford that every month unless you're extremely rich? But it is not a rich person's disease. It's a potluck, you don't know if you're a carrier.”

A petition calling for continued funding for the drugs has already got over 60,000 signatures, and 40,000 more would mean the issue is debated in Parliament.

The petition can be found here and takes less than a minute to sign.

NICE did not respond to the Luton News’ request for comment following the end of the consultation period. But in a statement last month 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 after interim access where further data was collected.”

She added: “We are continuing to work collaboratively 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. Existing patients and new patients who are started on treatment while the NICE evaluation is ongoing will continue to have access to the treatments after NICE has issued its final recommendations irrespective of the outcome."

Neither Bethany nor her husband knew they were carriers of the faulty CFTR gene, which causes the genetic condition. She explained: “If me and my husband knew we were carriers, then we would have looked at other options having children rather than going naturally.”

Cystic Fibrosis Trust responded to the consultation and highlighted the experiences of people within the community. The charity said: “We made sure we told NICE how important it is that everyone who could benefit is able to access these drugs in the long term, and we were really clear that these drugs are changing the lives of people with CF. Many people within the CF community also shared their views on the impact of the modulator drugs.

"In our submission, we gave the committee further experiences of people with CF to demonstrate the importance of these benefits, which include reduced treatment burden, improved gastrointestinal symptoms, reduction in antibiotic use, increased health stability and improved parent and carer mental wellbeing.”