A miracle-drug for cystic fibrosis patients could be axed from the NHS due to its huge cost, as Rhys Williams reports
Teenager Aidan Long has cystic fibrosis, but it’s not holding him back.
Ever since the 14-year-old has been taking the life-changing drug, Kaftrio, eating and drinking became less painful, and breathing is far easier. Last year he even completed a triathlon.
But Kaftrio’s future in the UK is now in the balance, as the medicines watchdog National Institute for Care Excellence (NICE) say it is too expensive to be prescribed on the NHS.
Kaftrio is manufactured by American pharmaceutical company Vertex.
The list price for a packet of 56 tablets costs £8,346, meaning NHS treatment could amount to more than £180,000 per year for each patient, an amount some say is “unjustifiable.”
Vertex insists the list price does not reflect the final reimbursed price agreed with the NHS.
Should Kaftrio be withdrawn next March, only those already taking the drug will be able to continue.
That means toddlers like Bertie Wozencroft, who is too young to take the medicine now, face having the “miracle drug” cruelly snatched away before they can even have it prescribed.
His mum, Kate, told me waiting for a decision was “brutal”, particularly as Bertie turns two-years-old in March, the same month NICE is due to make a decision.
“I think the cost is extraordinary, and it’s awful how much money the NHS has to pay for it,” says Kate.
She added: “But it’s known as a miracle drug and it has such benefits for the patient - they can come off other medications they’re on, they have less hospitals stays, they have less physio.”
Pharmacologist Dr Andrew Hill says Kaftrio maker Vertex needs to explain why the drug costs so much despite the manufacturing cost being cheaper
Vertex says it is committed to working with the NHS to ensure long term access to its Cystic Fibrosis medicines, but said the price reflects their clinical value and benefits to patients and healthcare systems.
In a statement, NICE says it’s evaluating the “cost-effectiveness” of cystic fibrosis medicines, and that it’s continuing to work with Vertex, the NHS and the Cystic Fibrosis Trust to deliver the best outcome.
Across the country toddlers like Bertie now facing the frightening possibility of having a miracle drug cruelly snatched away.
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