The National Institute for Health and Care Excellence (NICE) in the UK has issued a Final Appraisal Determination (FAD) for Massachusetts-based pharmaceutical company Vertex’s Orkambi (lumacaftor / ivacaftor), as a new therapy for treating cystic fibrosis.
Though recognised as a new therapy with significant clinical benefits, NICE has not yet recommended it for use on the National Health Service (NHS) in England for people with cystic fibrosis who are aged 12 and older and with two copies of the F508del mutation.
Vertex senior vice-president and international general manager Simon Bedson said: "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."
Cystic fibrosis is a genetic disease that causes progressive damage to a number of organs throughout the body, thereby shortening the life of the affected patient.
Bedson added: "Vertex has submitted an access scheme to the Department of Health, and we urge the NHS and the Government to consider this proposal."
In the UK, nearly half of the people who suffer from cystic fibrosis die by their late 20’s.
Orkambi is the first available medicine that can treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation.
On the other hand, NICE has recommended American pharmaceutical company Bristol-Myers Squibb’s combination therapy of Opdivo (nivolumab) and Yervoy (ipilimumab) to treat NHS patients suffering from advanced skin cancer.
Image: ‘Clubbing’ of the fingers is a classic feature of Cystic Fibrosis, although not present in many patients. Photo: courtesy of Jerry Nick, M.D.