Elexacaftor, tezacaftor and ivacaftor fixed dose combination therapy for treating cystic fibrosis with the F508del mutation [ID1661]
Discontinued
Reference number: GID-TA10566
The interim data collection agreement for Vertex’s cystic fibrosis modulator therapies has been updated to include elexacaftor, tezacaftor and ivacaftor (https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data-collection-agreement) following the announcement of a 4-year deal agreed by NHS England & NHS and Vertex (https://www.england.nhs.uk/2020/06/nhs-patients-among-first-in-europe-to-benefit-from-landmark-deal-for-cystic-fibrosis-treatment/) . The updated interim data collection agreement enables eligible patients continued access to Vertex’s cystic fibrosis modulator therapies while further data is collected to inform a future NICE technology appraisal. In addition, future therapeutic indications (for ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor or ivacaftor/tezacaftor/elexacaftor) that receive a marketing authorisation from the Medicines and Healthcare products Regulatory Agency or the European Medicines Agency (applicable to the United Kingdom) during the term of the interim data collection agreement will automatically be considered part of the agreement and formalised through a contract addendum.
NICE is currently unable to make a recommendation about the use of elexacaftor, tezacaftor and ivacaftor combination therapy in the NHS. In line with the updated interim data collection agreement the company has confirmed that it will provide an evidence submission towards the end of the interim access agreement initiating the NICE appraisal. NICE will contact stakeholders about participating in the new appraisal in due course.