Patients with cystic fibrosis will not be getting access to Vertex' Orkambi on the NHS in England Wales, after the National Institute for Health and Care Excellence stuck with its prior decision that the drug does not offer value for money.
The Institute has concluded that, compared to the current standard of care, the benefit Orkambi offers does not justify its "considerable cost".
It claims that, while the drug has been shown to reduce instances where people with cystic fibrosis are admitted to hospital, the benefits to lung function - a key measure of improvement - appear modest in the short term whilst the long-term benefits remain uncertain.
During the consultation process the Cystic Fibrosis Trust suggested that additional real-world data could be collected to better demonstrate that Orkambi does offer significant benefits in the long-term.
"However the committee concluded that if the drug were to be made available to patients on this basis, the company would have to provide Orkambi to the NHS in a cost effective way. Vertex Pharmaceuticals did not put this proposal forward to the NICE appraisal committee to consider," the Institute said.
Around 2,750 patients in England, with a specific genetic defect known as the F508del mutation, would be eligible for treatment with Orkambi, which costs £104,000 per patient for every year of treatment.
"We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high," said Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation.
"We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it."
If there are no appeals to the draft guidelines, final guidance will be published in July.
The Scottish Medicines Consortium issued guidance in May which does not recommend Orkambi.