NICE issues final draft guidance on multiple technology appraisal (MTA), ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor– ivacaftor for treating cystic fibrosis.
Final draft guidance for the MTA, ivacaftor–tezacaftor–elexacaftor (Kaftrio), tezacaftor–ivacaftor (Symkevi) and lumacaftor– ivacaftor (Orkambi) has been published by the National Institute for Health and Care Excellence (NICE). There is now a three-week period for appeal, after which final guidance will be published.
The Scottish Medicines Consortium (SMC) is collaborating with NICE on this guidance. SMC has had direct input into the decision-making committee that produced the final draft guidance.
The final draft guidance recommends:
- ivacaftor–tezacaftor–elexacaftor (Kaftrio) plus ivacaftor alone is recommended within its marketing authorisation, as an option for treating cystic fibrosis (CF) in people 2 years and over who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.
- tezacaftor–ivacaftor (Symkevi) plus ivacaftor alone is recommended, within its marketing authorisation, for treating CF in people 6 years and over who have:
- 2 copies of the CFTR gene with F508del mutations or
- a copy of the CFTR gene with an F508del mutation and a copy of the CFTR gene with 1 of the mutations listed in section 2.2 of NICE final draft guidance.
- lumacaftor– ivacaftor (Orkambi) is recommended, within its marketing authorisation, for treating CF in people 1 year and over who have 2 copies of the CFTR gene with F508del mutations.
At the time of publication of this final draft guidance, NHS National Procurement in Scotland has not been able to confirm and agree with Vertex the commercial arrangements necessary to deliver these recommendations for the NHS in Scotland. Discussions between NHS National Procurement and Vertex continue. If an agreement is reached, the advice will have the same status for health board consideration as other Scottish Medicines Consortium advice on new medicines.
While the MTA is underway the existing arrangements in Scotland and England for access to these medicines will continue. This will be the case for people starting or already taking these treatments.
You can read more about the final draft guidance recommendations here: