PBAC's latest decision on Elexacaftor/tezacaftor/ivacaftor: Recommended with restriction (2022). Considered for Treatment of cystic fibrosis in patients aged 6 to 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
3
first 2021
Submissions
3
2021 → 2022
Eligible population
Children aged 6 to 11 years with cystic fibrosis who have at least one F508del mutation in the CFTR gene, across six genotype subpopulations (F/F, F/RF, F/G, F/MF, F/R117H, F/not yet characterised).
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
Other
Key trials
Study 1061
Comparator
lumacaftor/ivacaftor (F/F population), ivacaftor (F/G population), best supportive care (F/MF, F/RF, F/R117H, F/not yet characterised populations)
Economic model
CEA
ICER note
No ICER stated in the document. Economic model based on cost-effectiveness and cost-comparison analyses, but numeric ICER values not published in this public summary.
Submission history
Mar 2021: Recommended with restriction · Authority Required
Dec 2021: Recommended with restriction · Authority Required
Nov 2022: Recommended with restriction · Authority Required