IdursulfaseElaprase
Not recommended Rare diseaseAuthority RequiredNot applicable line
Treatment of Hunter syndrome (Mucopolysaccharidosis II, MPS II) where diagnosis is confirmed by documented deficiency of iduronate sulphatase enzyme activity.
2
Submissions
2007–07
On the record
$15k–45k
ICER range
2 sourced ICERs · 2007
ICER stated
Cost basis
Decision on record
- Meeting Nov 2007 Not recommended Hunter syndrome (a genetic disorder also known as MPS II))
Access path
- TGA registered · Elaprase
- Nov 2007Recommended
vs placebo
- Nov 2007Recommended
- PBS listing · Authority Required
RecommendedDeferredNot recommended
Cost-effectiveness
ICER expressed as incremental cost per additional metre walked in 6MWT; doubts noted over meaningfulness of this ratio. Not a conventional QALY-based ICER.
The Committee agreed that it was appropriate for the submission not to present a modelled economic evaluation but considered that the results of the preliminary economic evaluation indicated that treatment with idursulfase was associated with an unacceptably high incremental cost effectiveness ratio. PBAC · 2007
ICER uncertainICER / price too highPrice cut / RSA neededSurrogate endpoint
Decision context
PopulationPatients with Hunter syndrome (MPS II) confirmed by documented deficiency of iduronate sulphatase enzyme activity, excluding those with severe CNS involvement.
Why it was knocked back
- unacceptably high incremental cost-effectiveness ratio
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2007 | Recommended | placebo | $15k–45k | RCT · 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) |
| Nov 2007 | Recommended | placebo | $15k–45k | RCT |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| TKT024 | Ph 2, PHASE3 | 94 | Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 | completed |