The seven-month-old baby’s family are pleading for health chiefs to reverse draft guidance that said the wonder drugs are too costly for new and future patients.
His father Dan said: “Dexter is smiley and happy and a wonderful boy and we just want him to have as normal a life as possible.
“Dexter could live a life unlimited by his genetic condition. He didn’t ask for this.”
Before 2019, there was no effective CF treatment on the NHS.
But in October of that year, after a campaign by the Daily Express, breakthrough drugs by US giant Vertex were made available to UK patients. Its newer CF wonder pill Kaftrio was later added.
The group of medications have transformed patients’ lives. But a month ago, NHS treatment approval group the National Institute for Health and Care Excellence gave initial draft guidance that Vertex’s drugs are too costly for future patients.
This means babies under one could be denied Vertex drugs when they are medically ready for them. Nice’s final decision on the matter is expected in the new year.
So, for now, any baby CF sufferers – and any sufferers not yet born – face missing out on Kaftrio, hailed as “almost a cure” for the cruel condition.
Yesterday, Dan told us that as Nice now ponders its final decision, he hopes officials realise how vital these drugs are.
The construction worker, of Selby, North Yorks, said: “We were shocked and gutted when we heard Nice’s initial decision, but there’s a long way to go yet and we hope they see sense.
“Kaftrio is an amazing drug and we are thankful to all those brilliant scientists who helped create it. And we’re not naive – we accept it’s only fair they are financially rewarded for them, but it has to be a fair price too.
“We have been doing our bit to raise awareness about how life-changing these drugs are and how necessary they are too.
“We are now praying that everyone at Nice listens to all the powerful arguments in favour of Vertex’s drugs and changes their decision in the new year.”
He and his wife Naomi, who also have a 3-year-old son Parker, who does not have CF, were already aware of the condition as Dan’s half-sister Millie, 22, also has it.
So far, Dexter has been growing strong and healthy and – apart from a brief bronchiolitis bout that required antibiotics – he has avoided any nasty lung bugs.
When he turns one in May, he will be old enough to start Vertex’s most basic starter drug Orkambi.
NHSE confirmed that, regardless of Nice’s decision, any CF patient already currently taking Orkambi can move on to Kaftrio when aged two – the minimum age you must be to take the wonder drug. So Dexter’s parents are hoping that Nice either reverses its initial guidance or takes until May to decide when the tot will be old enough to start on Orkambi.
CF is caused by a faulty gene which controls the movement of salt and water in and out of cells. It means sticky mucus clogs up lungs and bowels, making it hard to breathe and digest food.
Once a new treatment has a licence, drug appraisal bodies weigh up the manufacturer’s price against clinical data and recommend whether or not the NHS should make it available.
The health service is legally obliged to fund drugs recommended by these bodies. However, it also works the other way. If a drug is not recommended, then the NHS is not obliged to fund it.
Yet, after a campaign by the Cystic Fibrosis Trust, families and the Daily Express, NHSE announced in October 2019 it had agreed a rare interim access agreement for Vertex’s CF drugs.
That was on the proviso that Nice would appraise the drugs while patients were already taking them. But last month Nice dropped its bombshell draft guidance, saying despite the Vertex drugs being clinically effective treatments, their “list price” was too high for full-scale, long-term NHS use.
Its report said of the Vertex drugs: “Even when considering the condition’s severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates are above the range that Nice considers an acceptable use of NHS resources. So, they are not recommended.”
That leaves around 2,000 toddlers and babies either too young to yet start on the medication, or awaiting approval to start, who now remain in limbo.
Nice met again on Thursday for a second consultation, after the Cystic Fibrosis Trust, patient groups and Vertex themselves submitted extra evidence to reverse the decision.
The CF Trust said: “We’re expecting an update from Nice next week, which we don’t expect to be a final outcome, but the next stages in the process, which could continue well into 2024.”
Helen Knight, director of medicines evaluation at Nice, said: “We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure taxpayers continue to get value for money after interim access.
“We are continuing to work collaboratively with the company, NHS England and other stakeholders to deliver the best outcome both for people with cystic fibrosis and for the wider NHS.”
The Department of Health said: “It is vital patients have access to new and innovative medicines but the NHS must use its budget fairly.”
Last night, Vertex told us: “Vertex is committed to working collaboratively with the NHS and Nice to secure long-term access for any new patients who may benefit in the future following the conclusion of the Nice evaluation.”