OBJECTIVES: Treatment of acute myeloid leukemia (AML) has traditionally involved intensive chemotherapy (IC); thus, there remains an unmet need for approximately 50% of AML patients deemed unfit or ineligible for IC. This Canadian study evaluates the economic impact of venetoclax in combination with azacitidine (Ven+Aza) for the treatment of patients with newly diagnosed AML who are 75 years or older, or who have comorbidities that preclude IC.

METHODS: A lifetime partitioned survival model was developed to assess the cost-effectiveness of Ven+Aza compared to Aza alone. Health states included event-free survival, progressive/relapsed disease, and death. Efficacy parameters were based on the VIALE-A trial. Analyses were conducted from Ministry of Health (MoH) and societal perspectives. Cost components included initial and subsequent treatment, subsequent hematopoietic stem cell transplantation, management of adverse events, medical costs associated with health states (i.e., hospitalization, blood transfusion, and ongoing monitoring) and terminal care. From a societal perspective, costs associated with productivity loss were also considered.

RESULTS: Over a lifetime horizon, Ven+Aza was associated with a gain of 1.65 quality-adjusted life years (QALYs) compared to Aza alone. From a MoH perspective, Ven+Aza and Aza alone were associated with total costs of $204,305 and $82,333, respectively, resulting in an incremental cost-utility ratio (ICUR) of $73,841/QALY for Ven+Aza vs. Aza alone. From a societal perspective, Ven+Aza and Aza were associated with total costs of $177,510 and $77,955, respectively, resulting in an ICUR of $60,269/QALY. According to a willingness-to-pay threshold of $100,000/QALY, Ven+Aza was a cost-effective alternative compared to Aza alone in 98.8% and 99.7% of Monte Carlo simulations, from MoH and societal perspectives, respectively.

CONCLUSIONS: This economic evaluation demonstrates that, in comparison to Aza alone, Ven+Aza is a cost-effective strategy for the treatment of patients with newly diagnosed AML who are deemed unfit for IC.