Russell-Smith T Alexander, Brockbank James, Mamolo Carla, Knight Christopher
Global Oncology, Pfizer Inc, New York, NY, USA.
Department of Health Economics, RTI Health Solutions, Manchester, UK.
Pharmacoecon Open. 2021 Dec;5(4):677-691. doi: 10.1007/s41669-021-00278-3. Epub 2021 Jun 28.
The phase III ALFA-0701 study demonstrated the efficacy and safety of gemtuzumab ozogamicin (GO) versus standard of care (SOC) chemotherapy (daunorubicin and cytarabine) for the treatment of adult patients with de novo CD33+ acute myeloid leukaemia (AML). This study analysed the cost-effectiveness of GO from the perspective of the UK health care payer.
A cohort state-transition model was developed to estimate direct health care costs and quality-adjusted life-years (QALYs) over a lifetime time horizon from AML diagnosis to death using monthly cycles. Data on complete remission, overall survival, relapse-free survival (RFS), haematopoietic stem-cell transplantation, and adverse events for GO plus SOC versus SOC were obtained from the ALFA-0701 study. Overall survival and RFS were extrapolated beyond the trial horizon using mixture cure models. Unit costs were obtained from standard national sources. Utilities were identified in a systematic literature review. Costs and outcomes were discounted at 3.5%. Analyses were performed for the base-case population, excluding patients with an unfavourable cytogenetic profile, and the overall population.
For the base-case and overall populations respectively, incremental per-patient costs (£13,456 and £14,773) and QALYs (0.99 and 0.68) for GO plus SOC versus SOC resulted in incremental cost-effectiveness ratios (ICERs) of £13,561 and £21,819 per QALY gained. The mean probabilistic ICERs were £14,217 and £23,245, respectively. Univariate sensitivity analyses supported the robustness of the results.
The ICERs for both populations met NICE's £20,000-£30,000 willingness-to-pay threshold for medicines and supported the current approval for GO.
III期ALFA - 0701研究证明了吉妥珠单抗奥唑米星（GO）与标准治疗（SOC）化疗（柔红霉素和阿糖胞苷）相比，在治疗初治CD33 +急性髓系白血病（AML）成年患者中的疗效和安全性。本研究从英国医疗保健支付者的角度分析了GO的成本效益。
建立了一个队列状态转换模型，以估计从AML诊断到死亡的终身时间范围内，使用月度周期的直接医疗保健成本和质量调整生命年（QALY）。GO联合SOC与SOC相比的完全缓解、总生存期、无复发生存期（RFS）、造血干细胞移植和不良事件的数据来自ALFA - 0701研究。使用混合治愈模型将总生存期和RFS外推至试验期之外。单位成本从标准的国家来源获取。通过系统的文献综述确定效用值。成本和结果按3.5%进行贴现。对基础病例人群（不包括细胞遗传学特征不良的患者）和总体人群进行了分析。
对于基础病例人群和总体人群，GO联合SOC与SOC相比，每位患者的增量成本分别为13,456英镑和14,773英镑，QALY分别为0.99和0.68，导致每获得一个QALY的增量成本效益比（ICER）分别为13,561英镑和21,819英镑。平均概率ICER分别为14,217英镑和23,245英镑。单因素敏感性分析支持了结果的稳健性。
两个群体的ICER均符合英国国家卫生与临床优化研究所（NICE）设定的20,000 - 30,000英镑的药品支付意愿阈值，并支持了GO目前的批准。
