Ingress-Health, Rotterdam, the Netherlands.
Pfizer Canada Inc, Quebec, Canada.
J Med Econ. 2021 Jan-Dec;24(1):150-161. doi: 10.1080/13696998.2021.1875743.
The clinical efficacy and safety of DAURISMO (glasdegib) combined with low-dose cytarabine (LDAC) were demonstrated in the BRIGHT AML 1003 study among newly diagnosed acute myeloid leukemia patients who are not eligible to receive intensive chemotherapy. This study aims to evaluate its cost-effectiveness versus LDAC alone and azacitidine from a Canadian payer perspective.
A partitioned-survival model was developed with three health states: progression-free survival (PFS), relapse/progression and death. Clinical inputs were obtained from the BRIGHT AML 1003 study for glasdegib and LDAC, and from the two trial publications and indirect treatment comparison for azacitidine. Drug acquisition/administration, disease management, adverse event and end-of-life costs were considered. All costs were measured in Canadian dollars. Cost-effectiveness of glasdegib + LDAC was assessed against LDAC alone in main population, and against azacitidine by bone marrow blasts (BMB). A weighted average ICER was calculated to represent the current treatment use of Canadian clinical practice. The reference-case analysis was conducted probabilistically, and numerous probabilistic scenario analyses were conducted.
The incremental cost-effectiveness ratios (ICERs) compared to LDAC alone was CAD $177,065 (a mean gain of 0.41 QALYs and an incremental cost of CAD $72,695), to azacitidine in 20-30% and >30% BMB group were CAD $178,201 (a mean gain of 0.34 QALYs and an incremental cost of CAD $59,889) and dominant (a mean gain of 0.28 QALYs while reducing costs by CAD $7,856) respectively, resulting in a weighted average ICER of CAD $81,310 per QALY.
Though uncertainties remain with the generated PFS curve, the derived azacitidine curves, administration and vial wastage, the model has been built under the best available evidence and relied on clinical opinions where there were data gaps. The weighted average ICER suggests that glasdegib + LDAC is cost-effective at a CAD $100,000 willingness-to-pay threshold.
在 BRIGHT AML 1003 研究中，新诊断的不符合强化化疗条件的急性髓系白血病患者接受地西他滨（LDAC）联合达雷妥尤单抗（glasdegib）的临床疗效和安全性已得到证实。本研究旨在从加拿大支付者的角度评估glasdegib+LDAC 与单独 LDAC 和阿扎胞苷相比的成本效益。
采用三状态分区生存模型：无进展生存期（PFS）、复发/进展和死亡。glasdegib 和 LDAC 的临床数据来自 BRIGHT AML 1003 研究，阿扎胞苷的数据来自两项试验出版物和间接治疗比较。药物获取/管理、疾病管理、不良事件和生命终末期成本均被考虑在内。所有成本均以加元计量。glasdegib+LDAC 的成本效益在主要人群中与单独 LDAC 进行比较，并与骨髓原始细胞（BMB）<20%、20-30%和>30%的阿扎胞苷进行比较。使用加权平均增量成本效果比（ICER）来表示加拿大临床实践中的当前治疗方法。参考案例分析采用概率法进行，还进行了大量概率情景分析。
与单独 LDAC 相比，glasdegib+LDAC 的增量成本效果比（ICER）分别为 CAD$177065（平均增加 0.41 个 QALY，增量成本为 CAD$72695）、BMB<20%、20-30%和>30%的阿扎胞苷为 CAD$178201（平均增加 0.34 个 QALY，增量成本为 CAD$59889）和占优（平均增加 0.28 个 QALY，同时降低 CAD$7856），加权平均 ICER 为每 QALY CAD$81310。
尽管生成的 PFS 曲线存在不确定性，以及阿扎胞苷曲线、给药和小瓶损耗的不确定性，但该模型是根据现有最佳证据构建的，并在数据空白处依赖临床意见。加权平均 ICER 表明，glasdegib+LDAC 在加元 10 万的意愿支付阈值下具有成本效益。
