AzacitidineAzacitidine-Teva
Maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieve complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction chemotherapy with or without consolidation treatment, and who are not candidates for, including those who choose not to proceed to, haematopoietic stem cell transplantation (HSCT).
Decisions on record
- Meeting Sep 2022 Recommended Acute myeloid leukaemia maintenance therapy in patients achieving complete remission who are not candidates for haematopoietic stem cell transplantation no PSD
- Meeting Jul 2022 Not recommended Acute myeloid leukaemia maintenance therapy in patients not candidates for haematopoietic stem cell transplantation
- Meeting Mar 2011 Recommended Myelodysplastic syndrome no PSD
- Meeting Jul 2009 Deferred Myelodysplastic syndrome Chronic myelomonocytic leukaemia Acute myeloid leukaemia
Access path
- TGA registered · Azacitidine-Teva
TGA label narrower than the PBS population
- Jul 2009Recommended
vs best supportive care (BSC) which included low dose…
- Jul 2022Recommended · restricted
Comparator changed: best supportive care (BSC) which included low dose cytarabine and…
- PBS listing · Authority Required
From the public summary
The PBAC did not recommend oral azacitidine as maintenance therapy in patients with acute myeloid leukaemia (AML) who achieve complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction chemotherapy with or without consolidation treatment, and who are not candidates for, including those who choose not to proceed to, haematopoietic stem cell transplantation (HSCT).PSD · Jul 2022
The PBAC considered that there was a high unmet need for effective therapies to treat this condition.PSD · Jul 2022
The economic evaluation presented was a cost-utility analysis. Health benefits were reported as RFS years gained, life years (LYs) gained, and quality adjusted life years (QALYs) gained. The key aspects of the economic evaluation are summarised in Table 11. 19PSD · Jul 2022
Outcomes Quality adjusted life years, life years, relapse-free life years Methods used to Partitioned survival approach, incorporating a cohort expected value analysis. generate results Health states Relapse free (on treatment and off treatment substates), relapse, death.PSD · Jul 2022
On the basis of the direct evidence from the QUAZAR trial, the submission claimed that oral azacitidine was superior in terms of effectiveness and inferior in terms of safety compared with BSC. The clinical claim of effectiveness was based on OS, and the clinical claim of safety was based on comparison of all TEAEs, frequently reported TEAEs (any Grade and Grade 3-4) and haematologic adverse events.PSD · Jul 2022
The ESC considered that the clinical claims were supported by the results presented in the submission, noting that: 18PSD · Jul 2022
The PBAC noted and welcomed the input from individuals (2) and organisations (2) via the Consumer Comments facility on the PBS website. Comments from individuals and South East Myeloma Support expressed support for the listing of oral azacitidine.PSD · Jul 2022
The PBAC noted the advice received from the Leukaemia Foundation. The PBAC specifically noted the advice that the use of oral azacitidine as maintenance therapy for certain patients with AML may improve overall survival and appears to have very acceptable tolerability. The Leukaemia Foundation also noted the unmet need for effective AML maintenance therapies. The PBAC noted that this advice was supportive of the evidence provided in the submission.PSD · Jul 2022
Cost-effectiveness
ICER value is commercially sensitive and not published in the PSD.
The PBAC concluded that both the base-case ICER and the revised ICER were highly uncertain as the ICER was very sensitive to utilities, duration of therapy and wastage related to vials. PBAC · 2009
Decision context
PopulationAdult patients with acute myeloid leukaemia who achieved CR or CRi following induction chemotherapy with or without consolidation treatment, and who are not candidates for or choose not to proceed to haematopoietic stem cell transplantation.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2022 | Recommended · restricted | best supportive care (BSC) or no maintenance therapy | — | RCT · OS |
| Jul 2009 | Recommended | best supportive care (BSC) which included low dose cytarabine and standard chemotherapy | $45k–75k | RCT, Single-arm · OS |
Consumer voice
Four consumer sources (2 individuals and 2 organisations) supported listing of oral azacitidine. The Leukaemia Foundation noted that oral azacitidine as maintenance therapy for certain AML patients may improve overall survival with acceptable tolerability, and highlighted the unmet need for effective AML maintenance therapies.
The use of oral azacitidine as maintenance therapy for certain patients with AML may improve overall survival and appears to have very acceptable tolerability. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC explicitly excludes candidates for HSCT; TGA label does not specify HSCT eligibility status.