Ivacaftor/tezacaftor/elexacaftor (Kaftrio®) in combination with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This Rapid Review assessment pertains to the subpopulation of patients aged 6 to 11 years with cystic fibrosis and who are heterozygous for the F508del mutation and either a minimal function (MF) mutation, or an unknown mutation in the CFTR gene.
|NCPE Assessment Process||Complete|
|Rapid review commissioned||12/04/2022|
|Rapid review completed||10/05/2022|
|Rapid Review outcome||A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of ivacaftor/tezacaftor/elexacaftor (Kaftrio®) with a regimen of ivacaftor (Kalydeco®) compared with the current standard of care.|