Budget Impact Analysis of Revumenib for the Treatment of Relapsed or Refractory Acute Leukemias With a KMT2A Translocation in the United States
Author(s)
Ivo Abraham, PhD1, Pamela Martin, PhD2, Shailja Vaghela, MPH3, Timothy Klein, BS2, Eric Chow, PharmD4, Marie Rush, PharmD4, Robert Morlock, PhD5, Huan Huang, PhD4.
1Center for Health Outcomes and PharmacoEconomic Research, Department of Pharmacy Practice and Science, University of Arizona; Department of Pharmacy Practice and Science, College of Pharmacy, University of Arizona Cancer Center, Tucson, AZ, USA, 2Medical Decision Modeling, Indianapolis, IN, USA, 3HealthEcon Consulting, Inc., Ancaster, ON, Canada, 4Syndax Pharmaceuticals, Inc., Waltham, MA, USA, 5PROBRIDGE Solutions LLC, White Lake, MI, USA.
1Center for Health Outcomes and PharmacoEconomic Research, Department of Pharmacy Practice and Science, University of Arizona; Department of Pharmacy Practice and Science, College of Pharmacy, University of Arizona Cancer Center, Tucson, AZ, USA, 2Medical Decision Modeling, Indianapolis, IN, USA, 3HealthEcon Consulting, Inc., Ancaster, ON, Canada, 4Syndax Pharmaceuticals, Inc., Waltham, MA, USA, 5PROBRIDGE Solutions LLC, White Lake, MI, USA.
OBJECTIVES: Revumenib, a first-in-class, selective, oral menin inhibitor for the treatment of patients with relapsed/refractory (R/R) acute leukemias with a KMT2A translocation, including acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL), has demonstrated clinically meaningful responses and remissions. The aim of this study was to estimate the financial impact of adding revumenib for the treatment of adult patients (<65 years) with R/R acute leukemia with a KMT2A translocation onto the formulary of a hypothetical US health plan.
METHODS: A budget impact model (BIM) analyzed a formulary comprising one million members, with and without revumenib, from the perspective of US commercial third-party payers over a 3-year time horizon. Although no other targeted therapies were approved for the target population during BIM development, 11 other pharmacotherapies were included as model treatment options. Costs encompassed drug acquisition and administration, grade ≥3 adverse events (AEs), treatment-related supportive care and monitoring, subsequent salvage treatment, and end-of-life care. Hospitalization during treatment was considered as part of drug administration cost. Clinical data from pivotal trials informed the calculation of treatment, subsequent treatment, and end-of-life costs; AE rates were based on prescribing information and pivotal trials. All costs were adjusted to 2024 US dollars.
RESULTS: Approximately two adult patients with R/R KMT2A-rearranged (KMT2Ar) acute leukemias were estimated to be treatment eligible per year (AML, n=1.1; ALL, n=0.6). Compared to the formulary without revumenib, including revumenib was estimated approximately cost neutral, with an incremental per-member per month cost of -$0.0005. Revumenib was estimated to reduce the number of grade ≥3 AEs (0.2) over the 3-year period. Sensitivity and scenario analyses validated the robustness of the model.
CONCLUSIONS: Including revumenib on a formulary for adult patients with R/R KMT2Ar acute leukemias was approximately cost neutral and offers patients access to a targeted treatment with potential for improved clinical outcomes.
METHODS: A budget impact model (BIM) analyzed a formulary comprising one million members, with and without revumenib, from the perspective of US commercial third-party payers over a 3-year time horizon. Although no other targeted therapies were approved for the target population during BIM development, 11 other pharmacotherapies were included as model treatment options. Costs encompassed drug acquisition and administration, grade ≥3 adverse events (AEs), treatment-related supportive care and monitoring, subsequent salvage treatment, and end-of-life care. Hospitalization during treatment was considered as part of drug administration cost. Clinical data from pivotal trials informed the calculation of treatment, subsequent treatment, and end-of-life costs; AE rates were based on prescribing information and pivotal trials. All costs were adjusted to 2024 US dollars.
RESULTS: Approximately two adult patients with R/R KMT2A-rearranged (KMT2Ar) acute leukemias were estimated to be treatment eligible per year (AML, n=1.1; ALL, n=0.6). Compared to the formulary without revumenib, including revumenib was estimated approximately cost neutral, with an incremental per-member per month cost of -$0.0005. Revumenib was estimated to reduce the number of grade ≥3 AEs (0.2) over the 3-year period. Sensitivity and scenario analyses validated the robustness of the model.
CONCLUSIONS: Including revumenib on a formulary for adult patients with R/R KMT2Ar acute leukemias was approximately cost neutral and offers patients access to a targeted treatment with potential for improved clinical outcomes.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE254
Topic
Economic Evaluation
Topic Subcategory
Budget Impact Analysis
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases