GilteritinibXospata
Treatment of patients with relapsed or refractory acute myeloid leukaemia (AML) with an FMS-like tyrosine kinase 3 (FLT3) mutation.
Decisions on record
- Meeting Mar 2024 Recommended Acute myeloid leukaemia (AML) no PSD
- Meeting Mar 2022 Recommended Acute myeloid leukaemia
- Meeting Nov 2021 Not recommended Acute myeloid leukaemia (AML)
Access path
- TGA registered · Xospata
TGA label narrower than the PBS population
- Nov 2021Recommended · restricted
vs salvage chemotherapy (low-intensity: low dose cytarabine or…
- Mar 2022Recommended · restricted
Comparator changed: salvage chemotherapy (low-intensity: low dose cytarabine or…
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended the Authority Required (telephone/online PBS Authorities system) listing of gilteritinib for the treatment of patients with relapsed or refractory acute myeloid leukaemia (AML) with an FMS-like tyrosine kinase 3 (FLT3) mutation.PSD · Mar 2022
5.2 The PBAC is satisfied that gilteritinib provides, for some patients, a significant improvement in efficacy over salvage chemotherapy.PSD · Mar 2022
4.12 As outlined in Table 2, the outstanding issues to be resolved in the resubmission were related to the economic model and financial estimates. In the resubmission’s base case economic model, a 3-year cure point was applied for all those alive (compared with 2 years in the previous submission’s base case). As per the November 2021 submission, these survivors are considered ‘cured’ in the sense that they have the best long-term survival of all patients.PSD · Mar 2022
4.13 In the two sensitivity analyses presented, the resubmission applied an alternate definition of those alive and relapse free and applied a cure point of 2 and 3 years respectively. The proportion alive and relapse free at 2 years was sourced from the September 2020 data cut of ADMIRAL, where 26 patients (10.5% of ITT population) on gilteritinib lived for 2 or more years without relapse, and no salvage chemotherapy patients were alive in sustained …PSD · Mar 2022
tolerability. Source: Table 1, p4 of the November 2021 submission.PSD · Mar 2022
FLAG-IDA; G-CSF, fludarabine, cytarabine, idarubicin; FLT3, FMS-like tyrosine kinase 3; HSCT, haematopoietic stem cell transplantation; MEC, mitoxantrone, etoposide, cytarabine.PSD · Mar 2022
4.3 The PBAC noted the comments received from Rare Cancers Australia and the Leukaemia Foundation, outlining the poor prognosis of this patient population and potential benefits associated with the oral formulation of gilteritinib. Both organisations were strongly supportive of the PBS listing.PSD · Mar 2022
4.17 The resubmission stated the sponsor was not able to provide a detailed risk sharing arrangement (RSA) in time for the early re-entry resubmission but was prepared to discuss and enter into an RSA with the Department.PSD · Mar 2022
Cost-effectiveness
Decision context
PopulationPatients with relapsed or refractory acute myeloid leukaemia (AML) with an FMS-like tyrosine kinase 3 (FLT3) mutation, including those with ECOG status of 0 to 2.
Risk sharingRisk Sharing Arrangement (RSA) with key elements including special pricing arrangement for gilteritinib, annual expenditure caps based on agreed net PBS/RPBS cost, and agreed rebates for exceeding the agreed annual expenditure caps.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2022 | Recommended · restricted | salvage chemotherapy (low intensity: low dose cytarabine or azacitidine; high intensity: MEC induction chemotherapy or F | $75k–95k | RCT · OS |
| Nov 2021 | Recommended · restricted | salvage chemotherapy (low-intensity: low dose cytarabine or azacitidine; high-intensity: MEC induction chemotherapy or F | — | RCT · OS |
Consumer voice
A health care professional and three organisations (Rare Cancers Australia, the Leukaemia Foundation, and the Australasian Leukaemia & Lymphoma Group) provided input supporting PBS listing of gilteritinib for relapsed/refractory FLT3 positive AML. They highlighted the poor prognosis of this patient population, the benefits of oral formulation, the potential to achieve remission and enable HSCT, an
the role of gilteritinib in clinical practice would be to achieve remission in relapsed/refractory FLT3 positive AML, to allow these patients to receive allogeneic hematopoietic stem cell transplantation (HSCT), and also to achieve the best curative outcomes through maintenance use in the post-transplant setting. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to later-line treatment and specifies ECOG 0-2 performance status; TGA label has no line or performance status restrictions.