Eytan Stein1, Jipan Xie2, Emilie Duchesneau3, Subrata Bhattacharyya4, Umakanth Vudumula4, Briana Ndife5, Gaetano Bonifacio5, Annie Guerin6, Nanxin Li3, George Joseph5. 1. Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY, 10065, USA. 2. Analysis Group, Inc., 333 South Hope Street, 27th Floor, Los Angeles, CA, 90071, USA. Jipan.Xie@analysisgroup.com. 3. Analysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA, 02199, USA. 4. Novartis Healthcare Private Limited, Hyderabad, India. 5. Novartis Pharmaceuticals Corporation, 1 Health Plaza, East Hanover, NJ, 07936, USA. 6. Analysis Group, Inc., 1000 de la Gauchetière Ouest, Bureau 1200, Montréal, QC, H3B4W5, Canada.
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.
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.
Authors: Jay P Patel; Mithat Gönen; Maria E Figueroa; Hugo Fernandez; Zhuoxin Sun; Janis Racevskis; Pieter Van Vlierberghe; Igor Dolgalev; Sabrena Thomas; Olga Aminova; Kety Huberman; Janice Cheng; Agnes Viale; Nicholas D Socci; Adriana Heguy; Athena Cherry; Gail Vance; Rodney R Higgins; Rhett P Ketterling; Robert E Gallagher; Mark Litzow; Marcel R M van den Brink; Hillard M Lazarus; Jacob M Rowe; Selina Luger; Adolfo Ferrando; Elisabeth Paietta; Martin S Tallman; Ari Melnick; Omar Abdel-Wahab; Ross L Levine Journal: N Engl J Med Date: 2012-03-14 Impact factor: 91.245
Authors: Dimitri A Breems; Wim L J Van Putten; Peter C Huijgens; Gert J Ossenkoppele; Gregor E G Verhoef; Leo F Verdonck; Edo Vellenga; Georgine E De Greef; Emanuel Jacky; Johannes Van der Lelie; Marc A Boogaerts; Bob Löwenberg Journal: J Clin Oncol Date: 2005-01-04 Impact factor: 44.544
Authors: Anna Forsythe; Patricia S Brandt; Mike Dolph; Sachin Patel; Adrian Paul J Rabe; Gabriel Tremblay Journal: Clinicoecon Outcomes Res Date: 2018-01-25
Authors: Eytan M Stein; Min Yang; Annie Guerin; Wei Gao; Philip Galebach; Cheryl Q Xiang; Subrata Bhattacharyya; Gaetano Bonifacio; George J Joseph Journal: Health Qual Life Outcomes Date: 2018-09-21 Impact factor: 3.186