Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what the timescale is for NICE to make a decision on Orkambi.

In July 2016 the National Institute for Health and Care Excellence (NICE) published guidance on Orkambi for treating cystic fibrosis in people 12 years and older who are homozygous for the F508del mutation. This guidance is scheduled for review this year (2019). If NICE updates the existing recommendations, and the company participates, updated guidance for these patients is expected late 2020/early 2021.

In March 2019, NICE received Ministerial referral to appraise Orkambi in people aged between 2 and 11 years old who are homozygous for the F508del mutation. NICE is in the process of inviting the company and other stakeholders to participate in this appraisal. If the company participates, NICE expects to be able to come to a decision on the use of Orkambi for this group of children in the autumn of 2020.

The Secretary of State for Health wrote to Vertex in August offering a further meeting again with Vertex, as soon as possible, to consider what the barriers are to an agreement and how the situation can be resolved. We continue to urge Vertex to accept NHS England’s generous offer. In the absence of an agreement NHS England and Improvement continue to explore other options.