PBAC's latest decision on Galsulfase: Not recommended (2007). Considered for treatment of mucopolysaccharidosis type VI (MPS VI).
PBAC outcome
Not recommended
2007
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
1
first 2007
Submissions
1
2007 → 2007
Eligible population
patients with a positive diagnosis of mucopolysaccharidosis type VI (MPS VI)
Therapy area
Rare disease
Evidence base
RCT
Primary endpoint
distance walked in 12 minutes at 24 weeks, rate at which stairs were climbed during the three minute stair climb at 24 weeks
Pivotal trial size
19 patients
Key trials
Harmatz et al (2006), Harmatz et al (2005), Harmatz et al (2004)
Comparator
placebo plus standard medical management
Economic model
Not modelled
ICER note
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.
Why PBAC said no
Reasons cited in the latest PSD: 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