PBAC's latest decision on Eliglustat: Recommended with restriction (2015). Considered for Treatment of Gaucher disease type 1 (GD1) in adult patients aged ≥18 years with at least one GD1-related disease manifestation (skeletal disease, haematological complications, or gastrointestinal complications due to enlarged liver or spleen).
PBAC outcome
Recommended with restriction
Restricted
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
1
first 2015
Submissions
1
2015 → 2015
Eligible population
Adults aged ≥18 years with confirmed diagnosis of Gaucher disease type 1 and at least one GD1-related disease manifestation (splenomegaly, hepatomegaly, anaemia, thrombocytopenia, or skeletal disease) who are CYP2D6 poor, intermediate or extensive metabolisers.
Therapy area
Metabolic
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Maintenance of response in composite outcome (haemoglobin level, platelet count, spleen volume and liver volume)
Pivotal trial size
199 patients
Key trials
ENCORE, ENGAGE
Comparator
enzyme replacement therapy (imiglucerase or velaglucerase-alfa)
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis; no ICER calculated as this was a cost-minimisation comparison against ERT.