NICE denies NHS patients access to Vertex's Orkambi

“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.”

“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,” said Simon Bedson, Senior Vice President and International General Manager at Vertex. “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,” continued Mr Bedson. “We are committed to working with all parties to find a solution in order to make OrkambiI available to all eligible patients in England as quickly as possible, just as we did with Kalydeko (ivacaftor).”

CF is a life-shortening genetic disease that causes progressive damage to a number of organs throughout the body. In the UK, approximately half of those with CF die by their late 20s. Orkambi is the first medicine to treat the underlying cause of CF in people with two copies of the F508del mutation.