PBAC's latest decision on Velaglucerase alfa: Noted (2012). Considered for treatment of type 1 Gaucher disease in a patient who meets certain criteria, as per the Life Saving Drugs Program (LSDP).
PBAC outcome
Noted
2012
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
2
first 2011
Submissions
2
2011 → 2012
Eligible population
paediatric and adult patients with type 1 Gaucher disease associated with at least one of the following clinical manifestations: anaemia, thrombocytopaenia, hepato-splenomegaly
Therapy area
Rare disease
Evidence base
RCT | Single-arm
Primary endpoint
OS | PFS | DFS | ORR | QoL | Surrogate
Key trials
HGT-039, TKT-032, TKT-034, TKT-025
Comparator
imiglucerase
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis conducted; no numeric ICER calculated. Document states submission presented 'a cost-minimisation analysis of velaglucerase compared to imiglucerase' with claimed dose relativity of 1 U/kg to 1 U/kg and estimated nil financial cost to PBS.