SMC publishes Collaborative Advice Documents for ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis.
The Scottish Medicines Consortium (SMC) collaborated with the National Institute for Health and Care Excellence (NICE) on TA988; ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis.
SMC has published Collaborative Advice Documents for ivacaftor-tezacaftor-elexacaftor (Kaftrio), tezacaftor-ivacaftor (Symkevi) and lumacaftor- ivacaftor (Orkambi). The recommendations are aligned with NICE Final Guidance and are as follows:
- ivacaftor-tezacaftor-elexacaftor (Kaftrio) (SMC2713) is accepted for use in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508delmutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- tezacaftor-ivacaftor (Symkevi) (SMC2711) is accepted for use in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T
- lumacaftor-ivacaftor (Orkambi) (SMC2712) is accepted for use treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508delmutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene).
The advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS / list price that is equivalent or lower.
SMC Chairman Scott Muir said: “We are pleased that people in Scotland who are living with cystic fibrosis will continue to have access to these treatments, along with those who are diagnosed with cystic fibrosis in the future”.
You can read NICE Final Guidance here: https://www.nice.org.uk/guidance/ta988