Hope For Harley: Luton mum shares relief as NHS signs deal to continue cystic fibrosis drug funding

Bethany Whiting, her husband and two-year-old son, Harley, had their worlds turned upside down in November when the National Institute for Health and Care Excellence (NICE) recommended that no new patients get the drugs, on the grounds of cost.

At the time, Harley had been constantly “battling infections” and his family had hoped for him to start taking the medication.

In March, ahead of NICE’s final consultation, the Whitings were told that their son could begin taking the modulators. And three months on, he is responding well to the treatment.

Mum Bethany said: “He seems to be doing really well, and he hasn't had a cough in a while.”

Last week, after months of uncertainty, Vertex Pharmaceuticals Incorporated, which makes Orkambi, Kaftrio and Symkevi revealed that it had come to an agreement with the NHS to give all existing and future eligible cystic fibrosis patients in England access to the drugs.

After hearing the news, Bethany said: “It was one of the best days ever because we have seen how it has benefitted Harley. To find out that it is going to be continued so other children will be able to access the drugs, me and my husband were over the moon. We can’t put into words how excited and happy we are.”

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. But thanks to Orkambi, little Harley is doing better.

The Cystic Fibrosis Trust’s chief executive David Ramsden said: “This is a fantastic moment for many people with cystic fibrosis and their families – ending uncertainty and helping to ensure that everyone who can benefit is able to access these vital medicines – now and in the future.”