PBAC's latest decision on Tezacaftor with ivacaftor: Recommended with restriction (2019). Considered for Treatment of cystic fibrosis in patients aged 12 years or older who have at least one residual function (RF) mutation in the CFTR gene.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
3
first 2019
Submissions
3
2019 → 2019
Eligible population
Cystic fibrosis patients aged 12 years or older who have at least one residual function (RF) mutation in the CFTR gene.
Therapy area
Rare disease
Line of therapy
Not applicable
Evidence base
Single-arm
Primary endpoint
PFS
Key trials
Study 108
Comparator
best supportive care
Economic model
CUA
Eligible patients/year
10
ICER note
This was a minor resubmission that did not present an economic analysis. The March 2019 submission presented a cost-utility analysis, but specific ICER values are redacted (shown as '''''''''') in the public document. PBAC pragmatically considered the ICER would be no higher than that for lumacaftor with ivacaftor if pricing was comparable.
Risk sharing
Risk-sharing arrangement in place — Risk Sharing Arrangement (RSA) included in the Deed of Agreement for lumacaftor with ivacaftor. Subsidisation caps with reconciliation mechanism if Government expenditure per FTE patient exceeds redacted amount per patient per year.
Submission history
Mar 2019: Recommended with restriction · Authority Required
Mar 2019: Recommended with restriction · Authority Required
Nov 2019: Recommended with restriction · Authority Required