PBAC's latest decision on Arsenic trioxide: Not recommended (2015). Considered for First-line treatment of acute promyelocytic leukaemia (APL) in combination with all-trans retinoic acid (ATRA) with or without chemotherapy, for both induction of remission and consolidation therapy.
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
—
ICER not stated
Submissions
2
first 2009
Submissions
2
2009 → 2015
Eligible population
Newly diagnosed patients with previously untreated acute promyelocytic leukaemia (APL), characterised by t(15:17) translocation or PML/RAR-α gene expression, treated with ATO+ATRA±chemotherapy
Therapy area
Haematology
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Event-free survival
Pivotal trial size
156 patients
Key trials
Lo-Coco 2013, APML4, APML3, Shen 2004
Comparator
ATRA+chemotherapy (APML3 regimen used in economic model)
Economic model
CEA
ICER note
ICER not stated in public text; economic evaluation presented but document does not provide numeric ICER value
ICER (historical)
$45k/QALY–$75k/QALY in an earlier submission (2009) — the latest submission carried no numeric base case. No single PSD states this combined range; see source PSDs.
Why PBAC said no
Reasons cited in the latest PSD: uncertain effectiveness in high-risk patients (single-arm studies with high risk of bias), comparator regimen (APML3) did not represent current Australian clinical practice, potential inappropriate initiation of ATO in PML/RAR-α negative patients, insufficient evidence regarding maximum duration of induction therapy, regimens in APML4 not simply addition of ATO to APML3
Submission history
Mar 2009: Recommended with restriction · Authority Required — ICER $75k/QALY