Jensen Ivar S, Wu Elizabeth, Sacks Naomi C, Cyr Philip L, Chung Karen C
Director, Evidence Strategy & Generation, Precision Xtract, Boston, MA.
Health Economist, Evidence Strategy & Generation, Precision Xtract.
Am Health Drug Benefits. 2018 Oct;11(7):380-386.
Current national estimates for acute myelogenous leukemia (AML) indicate this disease accounts for 1.1% of new cancer diagnoses and 1.8% of cancer deaths in the United States. The 5-year overall survival rate for patients with AML was 27.4% between 2008 and 2014. The standard induction for patients with AML includes cytarabine, infused for 7 days, with 3 once-daily injections of an anthracycline, such as daunorubicin, known as the 7+3 regimen. Daunorubicin plus cytarabine liposomal encapsulation for injection was approved in the United States in 2017 for adults with newly diagnosed therapy-related AML (tAML) or AML with myelodysplasia-related changes (AML-MRC).
To estimate the annual budget impact of introducing daunorubicin-cytarabine liposome as induction treatment for patients with tAML or AML-MRC in the United States over a 3-year period.
The model consisted of a simple decision analytic framework for a 1- to 3-year period. We used an incidence-based approach to estimate the annual number of patients newly diagnosed with tAML or AML-MRC in a hypothetical 1-million-member plan. Patients were allocated to 2 groups based on when daunorubicin-cytarabine liposome became available, with the base-case group allocated to the 7+3 regimen, and another group allocated to daunorubicin-cytarabine liposome treatment. The incidence of AML was estimated as 4.3 per 100,000 people. Efficacy measures included the proportion of complete responders, proportion of patients who had undergone transplantation, and survival at 180 and 365 days. Inpatient drug and hospitalization costs were based on diagnosis-related group rates, and outpatient drug costs on wholesale acquisition costs.
Based on this hypothetical 1-million-member health plan, 15.1 members would receive intensive induction for newly diagnosed tAML or AML-MRC annually. Increasing the use of daunorubicin-cytarabine liposome (assumption of year 1, 20%; year 2, 50%; year 3, 80%) resulted in a 3-year incremental cumulative budget impact of $72,041 (1.7% increase for patients with tAML or AML-MRC), with a per-member per-month cost of $0.0032 at year 3. Over a 3-year period, the use of daunorubicin-cytarabine liposome would result in an estimated increase in the number of patients with a complete response to therapy by 2.72 (23.1%), which would lead to an incremental cost decrease of $179,956 per responding patient compared with the use of the 7+3 regimen in the base-case group.
Based on these results, induction treatment with daunorubicin-cytarabine liposome for patients with tAML or AML-MRC instead of the 7+3 regimen may have a limited economic impact on the budget of commercial health plans and may result in cost offsets, particularly in patients who respond to therapy.
目前美国全国范围内对急性髓系白血病（AML）的估计表明，该疾病占新癌症诊断病例的1.1%，占癌症死亡病例的1.8%。2008年至2014年期间，AML患者的5年总生存率为27.4%。AML患者的标准诱导治疗包括连续7天输注阿糖胞苷，并每日一次注射3次蒽环类药物，如柔红霉素，即所谓的7+3方案。注射用柔红霉素加阿糖胞苷脂质体于2017年在美国获批用于新诊断的治疗相关AML（tAML）或伴有骨髓增生异常相关改变的AML（AML-MRC）成人患者。
评估在美国3年期间引入柔红霉素-阿糖胞苷脂质体作为tAML或AML-MRC患者诱导治疗的年度预算影响。
该模型由一个为期1至3年的简单决策分析框架组成。我们采用基于发病率的方法来估计在一个假设的100万成员计划中每年新诊断为tAML或AML-MRC的患者数量。根据柔红霉素-阿糖胞苷脂质体何时可用，将患者分为两组，基础病例组采用7+3方案，另一组采用柔红霉素-阿糖胞苷脂质体治疗。AML的发病率估计为每10万人中有4.3例。疗效指标包括完全缓解者的比例、接受移植的患者比例以及180天和365天的生存率。住院药物和住院费用基于诊断相关组费率，门诊药物费用基于批发采购成本。
基于这个假设的100万成员健康计划，每年有15.1名成员将接受新诊断tAML或AML-MRC的强化诱导治疗。增加柔红霉素-阿糖胞苷脂质体的使用（假设第１年为20%，第2年为50%，第3年为80%）导致3年累计预算增量影响为72,041美元（tAML或AML-MRC患者增加1.7%），第3年每位成员每月成本为0.0032美元。在3年期间，使用柔红霉素-阿糖胞苷脂质体估计将使治疗完全缓解的患者数量增加2.72例（23.1%），与基础病例组使用7+3方案相比，每位缓解患者的成本将增量降低179,956美元。
基于这些结果，用柔红霉素-阿糖胞苷脂质体替代7+3方案对tAML或AML-MRC患者进行诱导治疗，对商业健康计划预算的经济影响可能有限，并且可能导致成本抵消，特别是在对治疗有反应的患者中。
