Taliglucerase AlfaElelyso
Long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.
Decisions on record
- Meeting Nov 2014 Not recommended 200 units injection, 1 vial Elelyso® Pfizer Australia Pty Ltd New listing (Major submission) Long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anaemia,
- Meeting Mar 2012 Not recommended Gaucher disease
Access path
- TGA registered · Elelyso
TGA label equal than the PBS population
- Mar 2012Not recommended
uncertain clinical effectiveness, lack of head-to-head comparative data, unknown equi-effective doses, unknown…
- Nov 2014Not recommended
equivalent comparative efficacy and safety to imiglucerase and velaglucerase alfa not established, lack of head-to-head…
From the public summary
7.1 The PBAC rejected the submission to list taliglucerase alfa on the PBS on the basis that equivalent comparative efficacy and safety to imiglucerase and velaglucerase alfa were not established. Although the PBAC accepted that taliglucerase alfa provides a clinical benefit to patients, the clinical claim that taliglucerase alfa provides equivalent health outcomes to imiglucerase and velaglucerase alfa was not accepted as the comparability of the results …PSD · Nov 2014
7.2 The PBAC recognised that imiglucerase and velaglucerase alfa are not listed on the PBS and have not been considered to be cost-effective for listing on the PBS but agreed that these two treatments were the appropriate comparators for the reasons outlined by the ESC. As velaglucerase alfa was now considered to be an appropriate additional comparator, it was noted that the resubmission presented clinical evidence reflecting this.PSD · Nov 2014
6.18 The resubmission presented a cost-minimisation analysis.PSD · Nov 2014
6.19 The evaluation questioned whether it was appropriate for taliglucerase alfa to be cost-minimised against products that have previously been considered non-cost- effective by the PBAC (Velaglucerase alfa Public Summary Document, November 2011/addendum March 2012).PSD · Nov 2014
6.16 The resubmission claimed that taliglucerase alfa achieves similar treatment efficacy outcomes to velaglucerase alfa and imiglucerase, and has a non-inferior safety and tolerability profile. The previous March 2012 submission made a similar claim regarding efficacy but made no claims regarding safety.PSD · Nov 2014
6.17 Based on the evidence provided in the resubmission, the ESC advised that there is a trend in the results that was suggestive of non-inferiority in efficacy and safety but that the resubmission’s claim was not robustly supported by the evidence presented.PSD · Nov 2014
Cost-effectiveness
Cost-minimisation analysis was not accepted because equivalent comparative efficacy and safety were not established.
The PBAC noted that the submission presented a simple non-comparative cost-per-outcome analysis with taliglucerase alfa treatment and given the high cost of taliglucerase, considered it unlikely that taliglucerase alfa would be considered cost-effective. PBAC · 2012
Decision context
PopulationAdult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anaemia, thrombocytopaenia.
Why it was knocked back
- equivalent comparative efficacy and safety to imiglucerase and velaglucerase alfa not established, lack of head-to-head direct randomised trial control data, differences in baseline values and outcome measurements limited comparability between trials, uncertain non-inferiority
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2014 | Not recommended | imiglucerase and velaglucerase alfa | — | RCT · Other |
| Mar 2012 | Not recommended | imiglucerase | — | RCT · Other |
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both require confirmed Type 1 Gaucher disease with identical clinical manifestations: splenomegaly, hepatomegaly, anaemia, or thrombocytopaenia.