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Daunorubicin With Cytarabine (Liposomal)

Recommended mixed outcome HaematologyAuthority RequiredFirst-line line 💬 consumer voice

Treatment of adults with newly diagnosed therapy-related acute myeloid leukaemia (t-AML) or acute myeloid leukaemia with myelodysplasia-related changes (AML-MRC).

1
Submissions
2023–23
On the record
Redacted
ICER range
commercial-in-confidence
Redacted
Cost basis

Decisions on record

5 decisions

The committee reached more than one outcome for this medicine at its most recent sitting — different indications were decided differently. The header reflects the least favourable of them; all are listed below.

  • Meeting Mar 2024 Withdrawn Acute myeloid leukaemia no PSD
  • Meeting Mar 2024 Recommended Acute myeloid leukaemia no PSD
  • Meeting Nov 2023 Recommended Therapy-related acute myeloid leukaemia or acute myeloid leukaemia with myelodysplasia-related changes
  • Meeting Jul 2023 Not recommended Therapy-related acute myeloid leukaemia (t-AML) and acute myeloid leukaemia with myelodysplasia-related changes (AML-MRC)
  • Meeting Jul 2022 Withdrawn Therapy-related acute myeloid leukaemia and acute myeloid leukaemia with myelodysplasia-related changes no PSD

From the public summary

Verbatim · PSD text · may span indications
PBAC outcome
7.1 The PBAC did not recommend liposomal daunorubicin and cytarabine for the treatment of therapy-related acute myeloid leukaemia (t-AML) or acute myeloid leukaemia with myelodysplasia-related changes (AML-MRC).PSD · Jul 2023
7.2 The primary reason for this outcome was due to the economic evaluation.PSD · Jul 2023
Economic analysis
6.64 The submission presented a modelled economic evaluation comparing treatment with liposomal daunorubicin and cytarabine versus idarubicin and cytarabine, in patients with newly diagnosed t-AML and AML-MRC. The model was based on the liposomal daunorubicin and cytarabine and daunorubicin and cytarabine arms of Study 301, 24PSD · Jul 2023
Table 10: Key components of the economic evaluation Component Summary Treatments Liposomal daunorubicin and cytarabine (LDC) vs idarubicin and cytarabine (IC). 25 years in the model base case versus a median follow-up of 5 years in Study 301. Average age Time horizon of patients at model entry was 67.7 years. Outcomes Quality-adjusted life years; life years.PSD · Jul 2023
Clinical claim
compared to idarubicin + cytarabine. Source: Table 1-1, p4 of the submissionPSD · Jul 2023
6.57 The submission described liposomal daunorubicin and cytarabine as superior in terms of efficacy compared to daunorubicin and cytarabine, as a proxy for idarubicin and cytarabine. The ESC noted that the data supported superior efficacy when comparing liposomal daunorubicin and cytarabine versus daunorubicin and cytarabine.PSD · Jul 2023
Consumer comments
6.2 The PBAC noted and welcomed the input from health care professionals (13) and organisations (2) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with liposomal daunorubicin and cytarabine including the improved response rate and overall survival, particularly in patients who subsequently underwent a HSCT.PSD · Jul 2023
6.3 The PBAC noted the advice received from Rare Cancers Australia and the Leukaemia Foundation, both which supported the submission and noted the unmet need for alternative and effective AML treatments.PSD · Jul 2023

Cost-effectiveness

ICER not stated in the public summary document; economic analysis referenced but values not disclosed.

Decision context

PopulationAdults aged 18+ years with newly diagnosed therapy-related acute myeloid leukaemia or acute myeloid leukaemia with myelodysplasia-related changes, WHO performance status ≤2, without prior induction chemotherapy, and deemed unsuitable for midostaurin treatment.

Submission history

1 entries
DecidedOutcomeComparatorICEREvidence
Jul 2023 Not recommended idarubicin and cytarabine (7+3 regimen for induction; 5+2 regimen for second induction and consolidation) RCT · OS

Consumer voice

Jul 2023

Health care professionals and consumer organisations submitted comments supporting liposomal daunorubicin and cytarabine treatment, highlighting improved response rates, overall survival benefits, and shorter administration times, while noting access barriers in rural settings due to short expiration times after compounding.

The comments described a range of benefits of treatment with liposomal daunorubicin and cytarabine including the improved response rate and overall survival, particularly in patients who subsequently underwent a HSCT. Consumer comments · PSD
survival benefittreatment burdenunmet needaccess barriers

Similar precedents

By decision profile

Regulatory · TGA

Label narrower than PBS population — PBAC restricted to WHO PS ≤2, no prior induction chemotherapy, and unsuitable for midostaurin; TGA label has no such restrictions.