PBAC's latest decision on Ivacaftor: Recommended with restriction (2025). Considered for Treatment of cystic fibrosis in patients aged 1 to 4 months with either a G551D mutation or other gating (Class III) mutation in the CFTR gene on at least 1 allele, or at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
10
first 2013
Submissions
10
2013 → 2025
Eligible population
Cystic fibrosis patients aged 1 to less than 4 months with either G551D mutation or other gating (Class III) mutation in the CFTR gene on at least 1 allele, or at least one mutation in the CFTR gene responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
Therapy area
Rare disease
Line of therapy
Any
Evidence base
Single-arm
Primary endpoint
Safety
Pivotal trial size
38 patients
Key trials
VX15-770-124 Cohort 8, VX15-770-126
Comparator
best supportive care
Economic model
Not modelled
ICER note
No economic evaluation was presented in the submission; listing was requested on the basis of equity and clinical need.
ICER (historical)
$60k/QALY–$80k/QALY across 3 submissions (2013–2015) — the latest submission carried no numeric base case. No single PSD states this combined range; see source PSDs.