'Don't take this miracle drug from our children' - parents' plea over possible withdrawal of 'life changer'

Little Bertie Wozencraft is 19 months old and has cystic fibrosis, a genetic condition which sees a sticky mucus build up in his lungs and digestive system.

His parents, Kate, 30, and George, 35, who live in Rhayader, in Mid Wales, have to be on constant guard against possible infections and they take him to hospital for regular appointments.

"He's a wild child," said Kate. "A typical boy, full of fun and full of energy and feeling good in himself at the moment."

But on the brink of being eligible when he reaches his second birthday in March for a "miracle drug" called Kaftrio, Kate, a practice nurse in Rhayader, fears it could be taken away before Bertie's birthday.

The National Institute for Health and Care Excellence (NICE) has recommended that the NHS withdraws the availability of CF modulators including Kaftrio which are a life-saving medication for the condition.

Anyone on the drug at the moment will be staying on it but those aged under two or yet to be born could see the opportunity taken away.

"Anyone of course wants the best for their children but I am not doing this just for Bertie," she said.

"It's also for those who have not been born yet. This is discrimination against young children."

Each year's treatment costs something like £150,000 which isn't remotely possible for the Wozencrafts to pay for privately.

Kate, originally from Presteigne, says: "Having cystic fibrosis is not cost free without the drugs because of the hospital appointments we have to make, and the time off we have to take. When Bertie goes in for antibiotics he has to have a hospital bed for seven days.

"The fact that NICE is even recommending this is disgusting. Without the drugs, people with CF are expected to live into their 30s, but with the drug which works straight away on the symptoms of CF, they can live a normal, healthy life.

"Who has the right to say you are not worth it? I think they are discriminating against young children, and I don't know how you can target them. The drug is there, and being used so they are putting a price tag on all kids' lives."

Kate says she is fighting not only for her own son but the children and yet-to-be-born children of others.

Campaigners have launched a petition to get the issue debated in Parliament.

The Cystic Fibrosis Trust is calling for the drug companies, NICE and the NHS to find a solution.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said: "NICE’s initial recommendation that the modulator drugs Kaftrio, Orkambi and Symkevi are highly effective for people with cystic fibrosis, but just too expensive to be available on the NHS, is disappointing news.

"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."

A spokesperson for NICE stressed that the decision in its draft guidance is not final. The issue is still in consultation and they added that the final recommendations will not affect those already receiving these medications.

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.

“The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions. This is so that we have all the relevant information to accurately capture the value of these effective medicines when the committee makes its final decision.

“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."

An independent NICE committee is appraising the clinical and cost effectiveness of these medicines as a four-year data collection agreement between NICE, NHS England, and Vertex Pharmaceuticals, is to conclude this year.