Astellas Pharma Global Development, Inc, Northbrook, IL, USA.
Analysis Group, Inc, Boston, MA, USA.
J Med Econ. 2021 Jan-Dec;24(1):19-28. doi: 10.1080/13696998.2020.1851698.
To estimate the economic impact of the introduction of gilteritinib for the treatment of relapsed/refractory (R/R) mutation-positive ( ) acute myeloid leukemia (AML) from a US payer's perspective.
A budget impact model (BIM) was developed to evaluate the 3-year total budgetary impact of treating adults with R/R AML eligible for gilteritinib in a hypothetical US health plan. Total costs (drugs/administration, hospitalization, monitoring, adverse events, transfusions, subsequent hematopoietic stem cell transplantation, post-progression, and testing) were estimated before and after gilteritinib entry. The budget impact was the total cost difference between the two scenarios. The target population size and cost inputs were based on public data or published literature, drug market share was informed by market research data, and the model included recommended treatments for R/R AML per clinical guidelines. Deterministic sensitivity analyses (DSAs) and scenario analyses varying key model inputs and assumptions were conducted to test for robustness.
In a hypothetical health plan with 1 million members, 20.9 adults with R/R AML were estimated to be eligible for gilteritinib. Of these, it was assumed 30.0% would be treated with gilteritinib in Year 1 following gilteritinib entry, increasing the total plan budget by $663,795 and the per-member-per-month (PMPM) cost by $0.055. In Years 2-3, the market share of gilteritinib increased to 45.0%, increasing the total plan budget impact by $1,078,371 and $1,087,230, and the PMPM cost by $0.090 and $0.091, respectively. The model results remained robust in DSAs and scenario analyses, with the largest impact observed when the projected uptake of gilteritinib was changed.
The results of this BIM are contingent upon the model's assumptions and inputs.
Adding gilteritinib to the formulary for the treatment of adults with R/R AML had a minimal budget impact from a US payer's perspective.
从美国支付者的角度评估吉瑞替尼治疗复发/难治性（R/R）突变阳性（）急性髓系白血病（AML）的引入所产生的经济影响。
开发了一个预算影响模型（BIM），以评估在假设的美国健康计划中，治疗有资格使用吉瑞替尼的 R/R 急性髓系白血病成年患者的 3 年总预算影响。在吉瑞替尼进入之前和之后，估算了总费用（药物/管理、住院、监测、不良事件、输血、后续造血干细胞移植、进展后和检测）。预算影响是两种方案之间的总成本差异。目标人群规模和成本投入基于公共数据或已发表的文献，药物市场份额来自市场研究数据，模型包括临床指南建议的 R/R 急性髓系白血病的治疗方法。进行了确定性敏感性分析（DSA）和情景分析，以测试关键模型输入和假设的变化，以检验模型的稳健性。
在一个拥有 100 万成员的假设性健康计划中，预计有 20.9 名 R/R 急性髓系白血病患者有资格使用吉瑞替尼。假设在吉瑞替尼进入后的第 1 年，有 30.0%的患者接受吉瑞替尼治疗，这将使计划总预算增加 663,795 美元，每位成员每月（PMPM）成本增加 0.055 美元。在第 2-3 年，吉瑞替尼的市场份额增加到 45.0%，使计划总预算影响增加 1,078,371 美元和 1,087,230 美元，PMPM 成本增加 0.090 美元和 0.091 美元。在 DSA 和情景分析中，模型结果仍然稳健，当预测的吉瑞替尼使用率发生变化时，观察到的影响最大。
本 BIM 的结果取决于模型的假设和投入。
从美国支付者的角度来看，将吉瑞替尼纳入治疗复发/难治性 急性髓系白血病成人的药物清单对预算的影响很小。
