Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the (a) effectiveness and (b) availability of the drug Trikafta for people with cystic fibrosis.

The Department has made no such assessment. Elexacaftor, tezacaftor and ivacaftor combination therapy (or triple therapy, marketed in the United States as Trikafta) does not yet have a marketing authorisation for use in the United Kingdom and the dates for licensing have not yet been confirmed.

The National Institute for Health and Care Excellence (NICE) is the independent body that makes recommendations for the National Health Service on whether new medicines represent a clinical and cost effective use of NHS resources. It is a condition of the agreement reached between NHS England, NHS Improvement and Vertex that the company will submit its full portfolio of cystic fibrosis medicines, including its new triple therapy to NICE for appraisal.

NICE has begun work for the appraisal and further information is available at the following link:

https://www.nice.org.uk/guidance/proposed/gid-ta10566

NICE will invite the company to provide an evidence submission immediately after the triple therapy has been formally referred to its work programme, expected in February.

Subject to licensing, in order to recommend the triple therapy for routine NHS funding, NICE will need to be assured that Vertex have priced it at a level that is fair to both the NHS and UK taxpayer. In the period leading up that, NICE will work extensively with the company and other stakeholders on putting the best possible evidence submission to its independent advisory committee.