Economic Evaluation of Azacitidine in Elderly Patients with Acute Myeloid Leukemia with High Blast Counts

Abstract

Background Azacitidine is an hypomethylating agent widely adopted for the treatment of acute myeloid leukaemia (AML) in patients who are ineligible for curative-intent chemotherapy. Patients with low bone marrow blast counts (< 30%) experience improved survival with azacitidine, but the benefits are significantly lower in patients with > 30% blasts in the bone marrow. As such, there is uncertainty around the economic benefit of azacitidine in patients with higher blast counts. Objective We present a cost-utility analysis of azacitidine in patients with AML with > 30% blasts to determine the economic value of azacitidine in this patient population from the perspective of a third-party payer. Methods A Markov model was developed with a time horizon of 25 months divided into 22 cycles of 35 days each. The cost utility of azacitidine was compared with that of conventional care regimens (which include best supportive care, low-dose cytarabine and induction chemotherapy). A Canadian public healthcare system perspective was selected. Results In the base case, the incremental cost per quality-adjusted life-year gained (incremental cost-effectiveness ratio [ICER]) for azacitidine compared with conventional care regimens was $Can160,438, year 2018 values. The estimated ICER was insensitive to a longer time horizon but sensitive to the cost of azacitidine and to assumptions relating to survival in both treatment regimens, although the ICER always remained greater than Can$80,000 in all scenarios. Conclusion Azacitidine is unlikely to be cost effective given that the estimated ICER exceeds the willingness to pay commonly used in the Canadian healthcare system.

JAK2 V617F as a Marker for Long-Term Disease Progression and Mortality in Polycythemia Vera and its Role in Economic Modeling

“…We thus had to rely on the KM data plots being correct and reflecting the actual study result. The digitizer tool we used has generated data into several other published modeling studies, [37][38][39][40] and we feel confident that the results that formed the basis of our model validation were reasonably reliable.…”

Cost-effectiveness of azacitidine and venetoclax in unfit patients with previously untreated acute myeloid leukemia

“…This economic evaluation comes on the heels of several others that have demonstrated persistently high ICERs for recently approved cancer treatments. [37][38][39][40][41][42] Although some high-cost therapies, such as the use of chimeric antigen receptor T-cell therapy in select B-cell malignancies, 43,44 have been shown to be cost-effective, other high-cost treatments that only marginally improve clinical outcomes and/or require continuous administration are generally of poor economic value. 41 Given the excitement surrounding the use of venetoclax in older, unfit patients with AML, 45,46 it is possible additional venetoclax combinations will be approved for AML treatment in the near future.…”