Cost-effectiveness analysis of azacitidine maintenance therapy in patients with acute myeloid leukemia

The QUAZAR AML-001 trial (NCT01757535) showed survival benefits with the maintenance treatment of oral azacitidine(CC-486) for acute myeloid leukemia(AML) in first complete remission. We conducted a cost-effectiveness analysis to explore the costs and benefits of oral azacitidine in AML.

We constructed a Markov model to evaluate the economic value of oral azacitidine. The time horizon was 10-years. The health utility scores and until prices of medical costs were acquired from previous studies and GoodRX. The transition probabilities were derived from the survival curves of the QUAZAR AML-001 study. Outcomes were measured in quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER).

Compared with placebo, oral azacitidine improved 0.39 QALY, with an increasing cost of $458,928.66. The ICER of oral azacitidine is $1,176,740.15(P < 0.05). Deterministic sensitivity analysis showed that the price of oral azacitidine has a significant impact on ICERs (P < 0.05). Probability sensitivity analysis showed that the probability of cost-effectiveness for oral azacitidine is 0.

In the United States, oral azacitidine is unlikely to be cost-effective for AML patients at current prices.

The trial is registered at ClinicalTrials.gov (CT.gov identifier: NCT01757535).