Final Nice appraisal says no to cystic fibrosis drug, Orkambi

pharmafile | June 17, 2016 | News story | Medical Communications NICE, Orkambi, Vertex Pharma, cystic fibrosis, recommendation, regulation 

The National Institute for Health and Care Excellence (Nice) has issued a final appraisal determination rejecting cystic fibrosis drug, Orkambi (lumacaftor/ivacaftor), which is developed by Vertex Pharmaceuticals.

Despite describing it as a “valuable new therapy for managing cystic fibrosis”, the drug will not be made available to patients on the NHS in England. The drug is indicated for people with cystic fibrosis aged 12 and over who have two copies of the F508del mutation.

Cystic fibrosis is a genetic disease that causes progressive damage to a number of organs throughout the body. Approximately, half of those with the disease in the UK die in their late 20’s. Vertex describes Orkambi as the “first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation.”

Nice did not recommend the drug in initial draft guidance. At the time, the independent appraisal committee concluded that “the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources.” They indicated that its cost of £104,000 per year per patient was excessive and that this cost would be exacerbated by most patients remaining on currently-available treatments while taking Orkambi.

Vertex, however, criticised the process that Nice uses to evaluate drugs for rare diseases and expressed concerns that eligible patients in England would not be able to access this potentially life-saving medicine.

Simon Bedson, senior VP at Vertex, says: “As we’ve said for many months, the single technology appraisal process is not appropriate for assessing medicines, such as Orkambi, for rare diseases like cystic fibrosis. As a result of applying the wrong appraisal process, approximately 2,700 people in England who could benefit from Orkambi are being forced to continue waiting for access.

“Vertex has submitted an access scheme to the Department of Health, and we urge the NHS and the government to consider this proposal. We are committed to working with all patients to find a solution in order to make Orkambi available to all eligible parties in England as quickly as possible, just as we did with Kalydeco (ivacaftor).”

Sean Murray

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