7 February 2017 - Subsequent to the deferral at the November 2016 PBAC meeting, the PBAC recommended the PBS listing of ivacaftor for treatment of cystic fibrosis in patients over the age of 2 years who have a G551D mutation or other class III gating mutations in the CFTR gene.

The PBAC noted that limited efficacy data in the 2-5 years age group was provided. However, the PBAC also noted the ethical difficulties associated with getting clinical trial data in this setting. The PBAC noted that observational data from the Ivacaftor Long Term Safety Study showed a statistically significant improvement in the relative risk (RR) of hospitalisation in patients aged <6 years (RR 0.68, 95%CI: 0.49-0.95). The PBAC recalled their earlier advice that the claim of superior efficacy over best supportive care in patients aged 2 to 5 years was biologically plausible and considered that, in this situation, the data provided was sufficient to support this claim.

Read PBAC recommendation for ivacaftor