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

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. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s41669-019-00180-z) contains supplementary material, which is available to authorized users.