Velaglucerase AlfaVpriv
Not recommended Rare diseaseNot applicableFirst-line line
Long-term enzyme replacement therapy for paediatric and adult patients with Type 1 Gaucher disease associated with at least one of the following clinical manifestations: anaemia, thrombocytopaenia, hepato-splenomegaly.
3
Submissions
2 resub
2011–12
On the record
—
ICER range
Cost-min
Cost basis
Decisions on record
- Meeting Mar 2012 Not recommended Long-term enzyme replacement therapy for paediatric and adult patients with Type 1 Gaucher disease associated with at least one of the following clinical manifestations: anaemia, thrombocytopaenia, hepato-splenomegaly.
- Meeting Nov 2011 Deferred Type 1 Gaucher disease
Access path
- TGA registered · Vpriv
TGA label narrower than the PBS population
- Nov 2011Deferred
vs imiglucerase
- Mar 2012Deferred
- Mar 2012Deferred
RecommendedDeferredNot recommended
Cost-effectiveness
Cost-minimisation analysis presented; no ICER calculated. Submission claimed net cost saving based on 1:1 dose ratio equivalence.
The PBAC noted that due to the probable high cost of velaglucerase (even if at a significant discount to imiglucerase), it would not be considered cost effective for listing on the PBS and hence proceeded to consider the possible inclusion of velaglucerase on the LSDP. PBAC · 2012
ICER / price too highEconomic model disputed
Decision context
PopulationPaediatric and adult patients with Type 1 Gaucher disease associated with anaemia, thrombocytopaenia, and/or hepato-splenomegaly.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2012 | Deferred | imiglucerase | — | RCT | Single-arm · OS | PFS | DFS | ORR | QoL | Surrogate |
| Mar 2012 | Deferred | imiglucerase | — | RCT · Hb |
| Nov 2011 | Deferred | imiglucerase | — | RCT | Single-arm · OS | PFS | DFS | ORR | QoL | Surrogate |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients meeting Life Saving Drugs Program criteria; TGA label has no such administrative restriction.