Patients with cystic fibrosis will not get 'routine' access to treatment with Vertex' Orkambi in England after the drug was ruled too expensive for NHS use.
The National Institute for Health and Care Excellence has now issued guidance to healthcare professionals not recommending Orkambi (lumacaftor/ivacaftor) on the NHS in England for people with CF ages 12 and older who have two copies of the F508del mutation.
According to Vertex, the Institute recognised the drug as a "valuable new therapy for managing cystic fibrosis" with significant clinical benefits as well as "wider benefits to society for people with cystic fibrosis and carers".
But it concluded that because of uncertainties over its impact on lung function - a key measure of improvement - particularly in the long-term, incremental cost-effectiveness ratio estimates for various scenarios fell well outside of what is normally considered value for money for the NHS.
However, the Cystic Fibrosis Trust argues that new long-term data not considered by NICE suggests Orkambi almost halves the rate of decline in lung function in people with CF over a two-year period, potentially adding years of life.
The charity's chief executive, Ed Owen, said the decision "demonstrates the weakness of the current NICE assessment process that the long-term value of the drug in slowing the decline in lung health is not sufficiently recognised".
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 (excluding VAT) per patient for every year of treatment (at list price).
The Scottish Medicines Consortium issued guidance in May also rejecting Orkambi in this setting.