Hope For Harley: Luton toddler with cystic fibrosis starts treatment ahead of NICE’s drug funding decision

Harley sat with his mum using his nebuliser. Picture: Tony Margiocchi

But his mum says she feels selfish as scores of other children might miss out on the medication

A toddler from Luton living with cystic fibrosis has started treatment, just weeks before the National Institute for Health and Care Excellence (NICE) is set to publish its recommendations about keeping the funding for the drugs.

Harley Whiting, a 23-month-old boy, was diagnosed with the hereditary disorder when he was born in 2022. His family had worried about his future when NICE recommended, in November, that no new patients get the life-changing drug Kaftrio because of its cost to the NHS. NICE had said that the recommendation was not final – and could change.

Harley has now started taking Orkambi, a drug used to treat cystic fibrosis. His mum, Bethany said: “He started Orkambi a month ago, he should start Kaftrio in April after he's had some blood tests. He’s been doing really well healthwise.”

Bethany and her husband, Adam, were devastated when they heard their son might miss out on the medication. 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. Next month, Harley turns two.

Bethany said: “It is so close now [to NICE’s recommendation announcement] and because he’s on Orkambi, it actually makes it a lot easier for him to start it. It definitely is a weight that’s been lifted.”

But if the draft guidance is finalised, other children may never get the chance to access these drugs. The draft guidance explained that funding for new patients would be withdrawn, but those currently using the medications can continue to use them.

Kaftrio costs £8,346 for a packet of 56 tablets. With two tablets a day, NHS treatment could tally up to more than £180,000 per year.

In a statement, NICE said: “Vertex has confirmed that they have commenced commercial discussions with NHS England with the goal of securing long-term access arrangements for any patients eligible for their cystic fibrosis treatments in the future.

“They confirm that any patients currently on Orkambi, Symkevi or Kaftrio or anyone who may start on one of the medicines prior to the expiry of the current access agreements will continue to have access now and going forward irrespective of the outcome of this process and subsequent NICE final recommendations.”

His mum said: “It's a massive relief, knowing that he'll be able to start this medication that has made a difference in so many lives already.”

But Bethany says she feels for the children who may not get these ‘miracle’ drugs.

She explained: “It's positive for us. But then there's going to be other families that haven't even started Orkambi. And if the guidance changes then so many families who haven't even had a child yet with CF, that’s going to impact them massively.

“It’s quite hard knowing that parents are going to go through such a horrible kind of journey if this medication is taken away, and it's almost like you feel selfish because your child gets it.”

A petition calling for continued funding for the drugs has already got over 70,000 signatures, and 100,000 would mean the issue is debated in Parliament.

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

NICE could not confirm when the full guidance will be published.