Hope For Harley: Families come forward in bid to stop 'life-saving' cystic fibrosis drugs from being scrapped

Harley is like any other toddler: he loves running and playing with his parents. But behind his infectious smile, the 20-month-old from Luton has cystic fibrosis - a genetic condition that affects his lungs and digestive system, one that could cut his life short.

When Harley was born, his mum, Bethany, was devastated to find out that her baby had cystic fibrosis. She said: "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."

In November, the National Institute for Health and Care Excellence (NICE) recommended that no new patients get the drugs, because of its cost to the NHS. But NICE says the recommendation is not yet final – and could change.

Bethany and her husband, Adam, had hoped Kaftrio would increase Harley’s life expectancy. The NHS says currently, only about half of people with cystic fibrosis will live past the age of 40. With Kaftrio, a person with the condition could live until around 80. But if the draft guidance is finalised, Harley may never get the chance to access these drugs.

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. If funding is kept the same, Harley could receive the drug as early as April, when he turns two.

NICE met last week to discuss the guidance after getting feedback from patients and other stakeholders. The final guidance from NICE is due to be published in March next year. Unfortunately, Harley Whiting’s story is all too familiar as families across the country have come forward to call for the funding to be kept as they fear for their children’s health.

In Chichester, Jess Meyrick is left worrying about her daughter, Ottilie. She said: “We have held onto hope and optimism because of a recent breakthrough – a promising drug that has the potential to significantly improve the quality of life for cystic fibrosis patients.

“Unfortunately, our hope is now under threat. This decision has left our family and countless others in our community devastated, as we have come so close to providing our children with a brighter future.”

Kirsty Ginnelly, from Altofts near Wakefield, had also been expecting her son, Oscar, to have Kaftrio from the age of two. She said: “When a miracle drug and a future for your child is dangled in front of you and then taken away it is unfathomable.”

A petition calling for continued drug funding already has over 65,000 signatures, and 35,000 more would mean the issue is debated in Parliament.

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

In response to the petition, the government said: “The government recognises that cystic fibrosis can have a huge impact on the physical and mental health of those affected. We recognise that cystic fibrosis presents significant challenges to the quality of life of patients and their families, and are committed to supporting timely access to effective new treatments in a way that is fair to all parties.”