PBAC's latest decision on Idursulfase: Not recommended (2007). Considered for treatment of Hunter syndrome (MPS II).
PBAC outcome
Not recommended
2007
ICER (AUD/QALY)
$45k/QALY
Range: $15k/QALY–$45k/QALY
Submissions
1
first 2007
Submissions
1
2007 → 2007
Eligible population
patients with Hunter syndrome (MPS II) where the diagnosis has been confirmed by documented deficiency of iduronate sulphatase enzyme activity
Therapy area
Rare disease
Evidence base
RCT
Primary endpoint
Two-component composite score, combining the ranked changes from baseline to week 53 for percentage predicted forced vital capacity (FVC) and six-minute walk test (6MWT)
Pivotal trial size
64 patients
Key trials
TKT024
Comparator
placebo
Economic model
CUA
ICER note
ICER expressed as incremental cost per additional metre walked in 6MWT; PBAC noted doubts over whether this was a meaningful ratio and that the 6MWT was a surrogate measure.
Why PBAC said no
Reasons cited in the latest PSD: unacceptably high cost-effectiveness ratio, unclear clinical relevance of surrogate outcome (6MWT), unknown longer-term effectiveness and toxicity, unknown impact on survival and disease progression, price higher than comparator laronidase for MPS I