Policy Analysis Inc. (PAI), 4 Davis Court, Brookline, MA, 02445, USA.
Amgen Inc., Thousand Oaks, CA, USA.
Pharmacoeconomics. 2019 Sep;37(9):1177-1193. doi: 10.1007/s40273-019-00812-6.
The TOWER and INO-VATE-ALL trials demonstrated the efficacy and safety of blinatumomab and inotuzumab ozogamicin (inotuzumab), respectively, versus standard-of-care (SOC) chemotherapy in adults with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia (ALL). The cost effectiveness of blinatumomab versus inotuzumab has not previously been examined.
Cost effectiveness of blinatumomab versus inotuzumab in R/R B-cell precursor ALL patients with one or no prior salvage therapy from a United States (US) payer perspective was estimated using a partitioned survival model. Health outcomes were estimated based on published aggregate data from INO-VATE-ALL and individual patient data from TOWER weighted to match patients in INO-VATE-ALL using matching adjusted indirect comparison (MAIC). Analyses were conducted using five approaches relating to use of anchored versus unanchored comparisons of health outcomes and, for the anchored comparisons, the reference treatment to which treatment effects on health outcomes were applied. Estimates from TOWER including the probabilities of complete remission and allogeneic stem-cell transplant (allo-SCT), overall and event-free survival, utilities, duration of therapy, and use of subsequent therapies were MAIC adjusted to match INO-VATE-ALL. Costs of treatment, adverse events, allo-SCT, subsequent therapies, and terminal care were from published sources. A 50-year time horizon and 3% annual discount rate were used.
Incremental costs for blinatumomab versus inotuzumab ranged from US$7023 to US$36,244, depending on the approach used for estimating relative effectiveness. Incremental quality-adjusted life-years (QALYs) ranged from 0.54 to 1.78. Cost effectiveness for blinatumomab versus inotuzumab ranged from US$4006 to US$20,737 per QALY gained.
Blinatumomab is estimated to be cost effective versus inotuzumab in R/R B-cell precursor ALL adults who have received one or no prior salvage therapy from a US payer perspective.
TOWER 和 INO-VATE-ALL 试验分别证明了blinatumomab 和 inotuzumab ozogamicin（inotuzumab）在接受过一种或无挽救性治疗的复发/难治性（R/R）B 细胞前体急性淋巴细胞白血病（ALL）成人患者中的疗效和安全性优于标准治疗（SOC）化疗。blinatumomab 与 inotuzumab 的成本效益此前尚未进行研究。
从美国（US）支付者的角度出发，使用分区生存模型估算blinatumomab 与 inotuzumab 在接受过一种或无挽救性治疗的 R/R B 细胞前体 ALL 患者中的成本效益。根据 INO-VATE-ALL 的汇总数据和 TOWER 的个体患者数据来估算健康结果，并使用匹配调整间接比较（MAIC）加权以匹配 INO-VATE-ALL 中的患者。使用五种方法进行分析，这些方法涉及健康结果的锚定与非锚定比较，以及对于锚定比较，将治疗效果应用于健康结果的参考治疗。TOWER 中的估计值包括完全缓解率和异基因造血干细胞移植（allo-SCT）、总生存率和无事件生存率、效用、治疗持续时间以及随后治疗的使用，均经过 MAIC 调整以匹配 INO-VATE-ALL。治疗、不良事件、allo-SCT、后续治疗和终末护理的成本均来自已发表的资源。使用 50 年时间范围和 3%的年度贴现率。
blinatumomab 与 inotuzumab 的增量成本在 US$7023 至 US$36,244 之间，具体取决于用于估计相对疗效的方法。增量质量调整生命年（QALY）在 0.54 至 1.78 之间。从 US 支付者的角度来看，blinatumomab 与 inotuzumab 的成本效益在每获得一个 QALY 时为 US$4006 至 US$20,737。
从美国支付者的角度来看，blinatumomab 与 inotuzumab 在接受过一种或无挽救性治疗的 R/R B 细胞前体 ALL 成人患者中具有成本效益。
