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Orkambi deemed not suitable for NHS use

Published on 27/07/16 at 11:40am

The National Institute for Health and Care Excellence (NICE) has issued its recommendation that the cystic fibrosis drug Orkambi (lumacaftor/ivacaftor) manufactured by Vertex Pharma should not be used on the National Health Service (NHS) in England for people with CF ages 12 and older who have two copies of the F508del mutation. The decision comes despite its earlier recognition of the drug as a “valuable new therapy for managing cystic fibrosis” with “wider benefits to society for people with cystic fibrosis and carers of people with cystic fibrosis”.

It is estimated that 2,700 people in England could benefit from the drug that do not currently have a medicine to treat the underlying cause of their disease.

“We are disappointed that NICE has not recommended Orkambi; however, this is not the end of the process,” commented Simon Bedson, senior vice president and international general manager at Vertex. “Vertex submitted a patient access scheme last month, which would provide rapid access to Orkambi for all eligible patients whilst simultaneously managing the budget impact to the NHS, and this submission is under active review. We are committed to continuing to work with the Department of Health and NICE and are optimistic that we can find a solution if all parties can work together in the coming months.”

“If medicines for rare diseases are going to be made available to the people who can benefit from them in this country, the current appraisal process must be adapted to be appropriate for assessing these types of medicines,” continued Bedson. “The UK Government recently stated that it is ‘actively looking at what we can do to accelerate the pathway for innovative treatments to reach NHS patients, including those with rare diseases like CF’. Collaborating with all parties to find a solution to make ORKAMBI available, just as they did with Kalydeco in 2012, is an opportunity for them to deliver on this goal.”

We caught up with Vertex Pharma vice president Simon Lem who echoed Bedson’s statements with his thoughts on NICE’s approval process earlier this year; read the full interview here.

Elsewhere in the UK, Vertex is working to make another submission with a proposed access scheme in Scotland as advised by the Scottish Medicines Consortium, with work proceeding in Northern Ireland and Wales in order to make Orkambi available ‘as quickly as possible’.

Matt Fellows

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