Costs per patient achieving remission with venetoclax-based combinations in newly diagnosed patients with acute myeloid leukemia ineligible for intensive induction chemotherapy

BACKGROUND: Venetoclax, in combination with azacitidine, decitabine, or low-dose cytarabine (LDAC), received confirmatory approval in 2020 by the US Food and Drug Administration for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged 75 years or older or who are ineligible for intensive induction chemotherapy. The economic value associated with response to venetoclax combinations compared with other treatments for this patient population has not been comprehensively evaluated. OBJECTIVE: To assess the cost per patient achieving remission with venetoclax combination therapies, compared with other therapies for newly diagnosed patients with AML who are ineligible for intensive induction chemotherapy, from a US third-party payer perspective. METHODS: The analysis used treatment effect estimates (ie, complete remission [CR] +CR with incomplete blood count recovery [CRi]) from a network meta-analysis and annual cost estimates from a prior budget impact model. The model considered the total cost of care including the costs of drug and administration, adverse events, hospitalization, disease monitoring, blood transfusions, and subsequent AML management when patients discontinued active treatment. Costs per patient achieving CR+ CRi associated with venetoctax + azacitidine. venetoctax+ LDAC, azacitidine, decitabine, LDAC, and best supportive care (le, treatment given with the intent to maximize quality of life without specific antiteukemic intent, such as blood transfusion products and antibiotics) were calculated as the annual total cost of care per patient divided by the CR+ CRi rate. All costs were adjusted to 2020 US dollars. RESULTS: Venetoclax combination therapies were estimated to have substantially lower costs per patient achieving CR+ CRi (venetoctax + azacitidine: 5473,960; venetoclax+ LDAC: 5428,071) than alternative treatments. Azacitidine was estimated to have the the highest cost per patient achieving CR+ CRi (51,197,438), followed by best supportive care ($869,849), LDAC (5689,101), and decitabine ($594,074). A pairwise matrix of the difference between therapies estimated that both venetoctax+azacitidine and venetoclax+ LDAC were associated with significantly lower costs per patient achieving CR+ CRi than azacitidine (by 5723,477 and 5769,367, respectively) and LDAC (by $215,141 and $261,030; all P<0.05). CONCLUSIONS: From a US third-party payer perspective, venetoclax in combination with azacitidine or LDAC was estimated to be associated with a significantly lower cost per patient achieving CR+CRi than azacitidine or LDAC among newly diagnosed patients with AML ineligible for intensive chemotherapy.