GalsulfaseNAGLAZYME
Not recommended Rare disease
treatment of mucopolysaccharidosis type VI (MPS VI)
1
Submissions
2007–07
On the record
—
ICER range
Not modelled
Cost basis
Decision on record
- Meeting Jul 2007 Not recommended Mucopolysaccharidosis VI (MPS VI)
Access path
- TGA registered · NAGLAZYME
TGA label equal than the PBS population
- Jul 2007Recommended
vs placebo plus standard medical management
- PBS listing
RecommendedDeferredNot recommended
Cost-effectiveness
No numeric ICER or ICER band stated. Cost-consequences analysis presented instead of cost-effectiveness analysis with ICER. Document states 'treatment with galsulfase is associated with an unacceptably high incremental cost effectiveness ratio' but no specific ICER value is provided.
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 indicate that treatment with galsulfase is associated with an unacceptably high incremental cost effectiveness ratio. PBAC · 2007
ICER / price too high
Decision context
Populationpatients with a positive diagnosis of mucopolysaccharidosis type VI (MPS VI)
Why it was knocked back
- unacceptably high incremental cost-effectiveness ratio, limited evidence of longer-term effectiveness and impact on survival, failure to demonstrate significant advantages in secondary outcome measures of respiratory, cardiac and musculoskeletal function
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2007 | Recommended | placebo plus standard medical management | — | RCT · distance walked in 12 minutes at 24 weeks, rate at which stairs were climbed during the three minute stair climb at 24 weeks |
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both indicate treatment of MPS VI; PBAC's requirement for positive diagnosis is standard clinical practice inherent to the TGA label.