PBAC's latest decision on Elosulfase alfa: Not recommended (2016). Considered for Treatment of patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A Syndrome).
PBAC outcome
Not recommended
2016
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
2
first 2014
Submissions
2
2014 → 2016
Eligible population
Patients with mucopolysaccharidosis type IVA (Morquio A Syndrome) aged 5 years and over with baseline 6-minute walk test distance of 30–325 metres, as per the MOR-004 trial population; the re-submission proposed inclusion of all children under age 5 years without continuation criteria until age 5.
placebo in combination with standard medical management
Economic model
CUA
ICER note
ICER stated as 'more than $200,000/QALY' but exact bounds not specified; reported as redacted/commercially sensitive in the cost table.
Risk sharing
Risk-sharing arrangement in place — Pay-for-performance arrangements proposal with a cap at redacted vials per injection.
Why PBAC said no
Reasons cited in the latest PSD: unclear clinically significant clinical benefit compared to placebo, unreliable and unacceptably high ICER (more than $200,000/QALY), uncertain clinical trial population applicability, concerns about duration of economic evaluation, cost of adverse events excluded from analysis, unreliable utility values