Abraham Ivo, Martin Pam, Vaghela Shailja, Klein Tim, Chow Eric, Rush Marie, Morlock Robert, Huang Huan
University of Arizona, Center for Health Outcomes and PharmacoEconomic Research and Department of Pharmacy Practice and Science, Ken Coit College of Pharmacy, University of Arizona, and University of Arizona Cancer Center, Tucson, AZ.
Medical Decision Modeling, Indianapolis, IN.
J Manag Care Spec Pharm. 2025 Jul;31(7):680-693. doi: 10.18553/jmcp.2025.25027. Epub 2025 Jun 3.
Acute leukemias (ALs), including acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), are heterogeneous diseases characterized by different phenotypic, genetic, and molecular alterations that can guide treatment decisions. ALs harboring lysine methyltransferase 2A gene translocation (), previously known as mixed-lineage leukemia, are associated with high rates of relapsed or refractory (R/R) disease. Revumenib, a first-in-class oral menin inhibitor, has shown improved clinical outcomes in patients with R/R ALs.
To estimate, using a budget impact model (BIM), the financial impact of introducing revumenib for the treatment of adult patients with R/R ALs on the formulary of a hypothetical US 1-million-member commercial health plan.
The BIM compared scenarios with or without revumenib and the resulting impact on commercial US third-party payers over a 3-year time horizon. Although no other therapies specifically targeted for R/R ALs were approved during BIM development, 11 additional pharmacotherapies for R/R ALs (5 for AML and 6 for ALL, not including revumenib) were included as treatment options in the model. Clinical data included adverse event (AE) rates, duration of treatment, time to subsequent treatment, and survival outcomes. Cost inputs (USD 2024) included in the model comprised drug acquisition and administration, grade 3 or greater AEs, treatment-related supportive care and monitoring, subsequent treatment, and end-of-life costs. The differential cost per member per month (PMPM) was estimated. One-way sensitivity analyses varying the costs of drug acquisition and toxicity by ±20% and scenario analyses varying uptake of revumenib and epidemiology inputs, as well as excluding costs related to supportive care and posttreatment discontinuation, were performed.
An estimated 1.7 adult patients (AML, 1.1; ALL, 0.6) were treatment eligible annually. Estimated 3-year total plan costs without and with revumenib were $2,146,564 and $2,126,919, respectively, for savings of -$19,646. Including revumenib was estimated to yield a differential PMPM cost of -$0.0005 over 3 years. The total number of grade 3 or greater AEs was lower over 3 years (10.82 vs 10.99, respectively) in the plan with revumenib vs without. Sensitivity and scenario analyses validated the robustness of the model.
The BIM demonstrated that including revumenib in a formulary for adult patients with R/R ALs was approximately cost neutral, offering patients access to a targeted treatment with potential for improved clinical outcomes.
急性白血病（AL），包括急性髓系白血病（AML）和急性淋巴细胞白血病（ALL），是异质性疾病，其特征在于不同的表型、基因和分子改变，这些改变可指导治疗决策。携带赖氨酸甲基转移酶2A基因易位（）的AL，以前称为混合谱系白血病，与高复发率或难治性（R/R）疾病相关。瑞武尼布是一种一流的口服Menin抑制剂，已在R/R AL患者中显示出改善的临床结果。
使用预算影响模型（BIM）估计在美国一个拥有100万成员的假设商业健康计划的处方中引入瑞武尼布治疗成年R/R AL患者的财务影响。
BIM比较了有或没有瑞武尼布的情景以及在3年时间范围内对美国商业第三方支付者的影响。虽然在BIM开发期间没有其他专门针对R/R AL的疗法被批准，但模型中包括了11种额外的R/R AL药物疗法（5种用于AML，6种用于ALL，不包括瑞武尼布）作为治疗选择。临床数据包括不良事件（AE）发生率、治疗持续时间、后续治疗时间和生存结果。模型中包括的成本投入（2024美元）包括药物采购和给药、3级或更高级别的AE、治疗相关的支持性护理和监测、后续治疗以及临终成本。估计了每位成员每月的差异成本（PMPM）。进行了单向敏感性分析，将药物采购成本和毒性成本分别变化±20%，以及情景分析，改变瑞武尼布的采用率和流行病学输入，以及排除与支持性护理和治疗后停药相关的成本。
估计每年有1.7名成年患者（AML，1.1；ALL，0.6）符合治疗条件。估计在没有瑞武尼布和有瑞武尼布的情况下，3年的计划总成本分别为2,146,564美元和2,126,919美元，节省了-19,646美元。包括瑞武尼布估计在3年内产生的差异PMPM成本为-0.0005美元。在有瑞武尼布的计划中，3年内3级或更高级别AE的总数低于没有瑞武尼布的计划（分别为10.82对10.99）。敏感性和情景分析验证了模型的稳健性。
BIM表明，在成年R/R AL患者的处方中包括瑞武尼布几乎是成本中性的，为患者提供了获得有改善临床结果潜力的靶向治疗的机会。
