Vertex seeks deal after NICE rejects CF drug Orkambi

NICE has rejected Vertex’s cystic fibrosis drug, Orkambi (lumacaftor+ivacaftor) for routine funding on the National Health Service in England – but the company has said it will fight to get the drug to patients.

In final guidance, the cost-effectiveness body rejected Orkambi in people aged 12 and over who have two copies of the F508del mutation.

Its £104,000 per year per patient price tag was too much for the NHS, said NICE, adding that even after a price reduction, the incremental cost-effectiveness ratio is around £218,000 per quality adjusted life year for Orkambi plus standard of care compared with standard of care alone.

Although NICE recognised Orkambi’s value in managing the disease, and wider benefits to society for people with cystic fibrosis, and their carers, it said the drug was too expensive for the NHS.

However Simon Bedson, senior vice president and international general manager at Vertex, said the company had submitted a patient access scheme to NICE.

This could provide rapid access for patients while managing the budget impact to the NHS, said Bedson, who said the request is “under active review”.

Bedson said: “We are committed to continuing to work with the Department of Health and NICE and are optimistic that we can find a solution if all parties can work together in the coming months.”

But he argued that NICE’s single appraisal process is not appropriate for rare disease medicines such as Orkambi, adding that Vertex has raised the issue repeatedly with NICE, ministers and the Department of Health.

Bedson said he hoped the Accelerated Access Review, which was commissioned by former life sciences minister, George Freeman, will provide a route for innovative treatments like Orkambi to reach NHS patients.

The Scottish Medicines Consortium is also reviewing Orkambi and Vertex is working on another access scheme for patients north of the border. It is also in discussions with authorities in Wales and Northern Ireland.

Last week, NICE said that England's NHS should fund PTC Therapeutics' Duchenne muscular dystrophy drug, Translarna (ataluren), after the manufacturer offered a managed access scheme.