In July 2016, the National Institute for Health and Care Excellence (NICE), the independent body that develops guidance on drugs and treatments for clinical and cost effectiveness for the National Health Service in England, was unable to recommend Orkambi as a cost effective use of NHS resources. NICE concluded that, although clinically significant for managing cystic fibrosis, the longer term outcomes and benefits were not sufficient to justify its considerable costs. Orkambi is not therefore routinely available to NHS patients with cystic fibrosis.
Since then there has been a constructive dialogue underway between the company, NHS England and NICE, including discussion hosted through NICE’s confidential ‘Office for Market Access’, although NICE is yet to receive any fresh proposals from Vertex, the company that manufactures Orkambi. Both NHS England and NICE have been consistent in advice to Vertex, that progress can only be made by working through NICE’s appraisal processes and the existing commercial framework.
Any funding decisions in the absence of positive NICE technology appraisal guidance should be made by NHS commissioners based on an assessment of the available evidence and on the basis of an individual patient’s clinical circumstances.