ivacaftor (Kalydeco)

following a full submission considered under the ultra-orphan process:

ivacaftor (Kalydeco®) is not recommended for use within NHS Scotland.

Indication under review: for the treatment of patients with cystic fibrosis (CF) aged 18 years and older who have an R117H mutation in the CF transmembrane conductance regulator (CFTR) gene.

Ivacaftor, compared to placebo, significantly increased percent predicted forced expiratory volume in one second (ppFEV1) by 5.0% at 24 weeks in a subgroup of patients aged ≥18 years with CF and an R117H mutation of the CFTR gene. 

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present sufficiently robust clinical and economic analyses to gain acceptance by SMC. 

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.