Kalydeco is a transformational treatment that has been appraised and confirmed as having a significant clinical benefit for people with cystic fibrosis over the age of six with the G551D gene mutation.
It is vital that a fair and affordable arrangement is reached soon between the NHS and Vertex, the drug's manufacturers, to ensure all who would benefit from the drug receive it as soon as possible at a reasonable cost to the NHS.
We welcome the work of NHS commissioners and advisors in England to accelerate the system of approval, and their recognition of the drug's clinical benefit. We also welcome the work continuing in Scotland, Wales and Northern Ireland to appraise the treatment.
We therefore call on both Vertex and the NHS to recognise, when they meet this month, that they have an unprecedented opportunity to make available a treatment which has the potential truly to change the lives and outlooks of patients with cystic fibrosis in England who carry the G551D mutation.
We call upon Vertex to recognise the financial pressures on the NHS and to demonstrate an ability to deliver innovative and cutting-edge treatments at a price which is affordable. We call upon the NHS to demonstrate that it has the will and capability to make such treatments available to patients on an equal and speedy basis.
Cystic Fibrosis Trust
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