PBAC's latest decision on Lumacaftor/ivacaftor: Not recommended (2019). Considered for Treatment of cystic fibrosis in patients aged 2 to 5 years who are homozygous for the F508del mutation in the CFTR gene.
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2019
Submissions
1
2019 → 2019
Eligible population
Children aged 2 to 5 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
Safety and pharmacokinetics
Pivotal trial size
60 patients
Key trials
Study 115
Comparator
best supportive care (BSC)
Economic model
CUA
ICER note
ICER values are redacted (marked as '''''''''''''''''''''') in the publicly available document; commercially sensitive.
Risk sharing
Risk-sharing arrangement in place — Risk sharing arrangement (RSA) under the current Deed of Agreement proposing a reduction in gross cost via a subsidisation cap, with intent to achieve a price of $'''''''''''' per patient per year (redacted). Similar arrangement to ivacaftor granules discussed.
Why PBAC said no
Reasons cited in the latest PSD: Non-comparative open-label trial design with no head-to-head comparison to BSC; small sample size (n=60 Part B); short duration (24 weeks) for a chronic disease; absence of comparator group prevented assessment of comparative efficacy/safety; uncertain comparative effectiveness versus deferring treatment to age 6 years; high risk of bias; significant risk that Government would pay higher amount per FTE patient than intended price.