Treatment to help Wakefield baby with cystic fibrosis live a longer and ‘normal’ life could be withdrawn

Kirsty Ginnelly, from Altofts, was expecting her son Oscar would be able to have “miracle drug” Kaftrio from the age of two, but she fears that draft guidance by the National Institute for Health and Care Excellence (NICE) means he will not receive the drug at all.

A person with cystic fibrosis taking Kaftrio could live until around 80.

The NHS says currently, about half of people with cystic fibrosis will live past the age of 40.

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.

"The prospect of Oscar starting the treatment aged two was a lifeline.

“He would have been able to go to nursery, not worry about catching bugs and live a relatively long life.

"But now I’ve thought ‘how many more Christmases will he have without this drug?’.

"He has an uncertain future now. I feel his future has been torn away by some people in the boardroom who feel his life is not cost effective.

"It’s not just my worry but parents across the country with children who are not yet on modulators like Kaftrio.”

People with cystic fibrosis who are already using the treatment will be able to continue.

It is currently recommended for children six and above.

NICE will publish its final recommendation next month.

The body’s draft guidance says: “The most likely cost-effectiveness estimates ... are above the range that NICE considers an acceptable use of NHS resources.”

The drug is manufactured by Vertex Pharmaceuticals.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said NICE’s recommendation was “disappointing news”.

He said: "It is important to emphasise that those already taking any of the modulator drugs are not affected by the NICE process because of the agreements already in place but this update creates uncertainty for those not yet on treatment.

"Vertex, NICE, and the NHS must now urgently work together to find a solution to make these treatments available for all those who could potentially benefit.

"We must never return to a situation where people with CF die far too young, knowing there’s a treatment that could change that.”

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.

"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.”

Vertex said it submitted 6,000 real world examples to NICE.

A spokesperson said: “We believe that the committee has not fully considered all of this real-world evidence and we will be providing additional information and analysis to be taken into account.

“We remain committed to working collaboratively with NICE and the NHS to ensure long term sustainable access for all eligible patients who could benefit from these medicines in the future.”