Mamolo C, Welch V, Walter RB, Cappelleri JC, Brockbank J, Cawson M, Knight C, Wilson M. Budget impact analysis of gemtuzumab ozogamicin for the treatment of CD33-positive acute myeloid leukemia. Pharmacoeconomics. 2021 Jan;39(1):121-31. doi: 10.1007/s40273-020-00976-6.

BACKGROUND: Gemtuzumab ozogamicin (GO) was approved in 2017 in the United States for the treatment of adults with newly diagnosed CD33-positive (CD33+) acute myeloid leukemia (AML) and adults and pediatric patients with CD33+ relapsed/refractory (R/R) AML.

OBJECTIVE: To estimate the budgetary impact of introducing GO to a 1-million-member US health plan over a 5-year period.

METHODS: We developed models to estimate the impact of introducing GO in combination with conventional induction chemotherapy or as monotherapy for newly diagnosed AML and as monotherapy for R/R AML. Models were built using data on drug costs and treatment-related outcomes obtained from published clinical trials and other publicly available sources. Results were reported on a per member/per year and per member/per month (PMPM) basis.

RESULTS: Base-case results of the newly diagnosed models indicated net PMPM costs (US$) were low (<$0.003) over a 5-year period in the monotherapy and combination settings. Cost savings were observed in years 3–5 in the combination therapy setting due to fewer relapses and transplants in members treated with GO. Scenario analyses examining a 20% higher and lower uptake of GO also demonstrated cost savings in years 3–5 in the combination therapy setting and low net PMPM costs (<$0.004) in the monotherapy setting. Base-case results of the R/R model showed that GO was associated with increased net costs, but the overall budget impact was negligible (<$0.004 net PMPM cost). Scenario analyses examining a 20% higher and lower treatment uptake for GO and the lowest price per unit across the formulation of each treatment option also resulted in increased net costs, although the incremental PMPM cost was <$0.005 across all years and scenarios.

CONCLUSION: The introduction of GO for newly diagnosed and R/R AML would have a minimal impact on the budget of a US health plan and could result in cost savings in the combination therapy setting for newly diagnosed AML.

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