Cost Effectiveness of Midostaurin in the Treatment of Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia in the United States

Eytan Stein, Jipan Xie (), Emilie Duchesneau, Subrata Bhattacharyya, Umakanth Vudumula, Briana Ndife, Gaetano Bonifacio, Annie Guerin, Nanxin Li and George Joseph
Additional contact information
Eytan Stein: Memorial Sloan Kettering Cancer Center
Jipan Xie: Analysis Group, Inc.
Emilie Duchesneau: Analysis Group, Inc.
Subrata Bhattacharyya: Novartis Healthcare Private Limited
Umakanth Vudumula: Novartis Healthcare Private Limited
Briana Ndife: Novartis Pharmaceuticals Corporation
Gaetano Bonifacio: Novartis Pharmaceuticals Corporation
Annie Guerin: Analysis Group, Inc.
Nanxin Li: Analysis Group, Inc.
George Joseph: Novartis Pharmaceuticals Corporation

PharmacoEconomics, 2019, vol. 37, issue 2, No 8, 239-253

Abstract: Abstract Objectives The aim of this study was to assess the cost effectiveness of midostaurin + cytarabine + daunorubicin (midostaurin arm) versus placebo + cytarabine + daunorubicin (placebo arm) in the treatment of adult patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML) who are eligible for standard cytarabine + daunorubicin chemotherapy, from a US third-party payer perspective. Methods A lifetime partitioned survival model with four health states (active disease, complete remission [CR], relapse, and death) was constructed. Efficacy inputs (time to CR or death, time to relapse or death, and overall survival) were estimated using data from the RATIFY trial (NCT00651261). Costs (inflated to 2016 US dollars) included treatment, drug monitoring, stem cell transplantation (SCT), adverse events costs, and medical costs associated with health states. Incremental costs per quality-adjusted life-year (QALY) and life-year (LY) gained were estimated. Deterministic (DSA) and probabilistic sensitivity analyses (and PSA) were performed to assess model robustness. Results In the base case, patients in the midostaurin arm incurred higher total direct costs over a lifetime compared with the placebo arm ($4,043,470 vs. $3,959,741), resulting in an incremental cost of $83,729; however, the midostaurin arm had better effectiveness, with 1.59 more LYs and 1.37 more QALYs. These led to a base-case incremental cost-effectiveness ratio (ICER) of $52,596 per LY, or $61,167 per QALY. Results were robust in the DSA. In the PSA, the probability of the midostaurin arm being cost-effective compared with the placebo arm was 65.9%, at a willingness to pay of $150,000/QALY. Conclusions This analysis suggests that midostaurin is a cost-effective treatment for adult patients with newly diagnosed FLT3-mutated AML, from a US third-party payer perspective.

Date: 2019
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DOI: 10.1007/s40273-018-0732-4

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