Pharmaceutical Outcomes and Policy, College of Pharmacy, University of Florida, Gainesville.
J Manag Care Spec Pharm. 2024 Sep;30(9):942-953. doi: 10.18553/jmcp.2024.30.9.942.
Abemaciclib was newly approved for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) high-risk early breast cancer (EBC). Clinical guidelines recommended abemaciclib as the first-line treatment for HR+/ HER2- EBC (early use) or HR+/ HER2- metastatic breast cancer (MBC) (delayed use).
To compare the cost-effectiveness of early vs delayed use of abemaciclib for treatment of HR+/HER2- high-risk EBC. Early use was defined as combined abemaciclib and endocrine therapy as first-line therapy for EBC, followed by treatment with fulvestrant for MBC. Delayed use was defined as endocrine therapy for EBC, followed by combined abemaciclib and fulvestrant therapy for MBC.
A 5-state model was developed to estimate lifetime costs, life-years (LYs), and quality-adjusted life-years (QALYs) of hypothetical patients with HR+/ HER2- EBC from a third-party US payer's perspective. Key clinical and safety data were derived from the monarchE and MONARCH 2 clinical trials. Costs, utilities, and disutility values of adverse events were obtained from the literature. We calculated the incremental cost-effectiveness ratio (ICER) of early vs delayed abemaciclib use and compared it with a willingness-to-pay (WTP) threshold of $100,000 per LY or QALY. Deterministic and probabilistic sensitivity analyses (PSAs) were performed to test the robustness of the base-case model.
Base-case analysis showed early use yielded 21.08 LYs and 17.93 QALYs for $586,213 and delayed use yielded 11.14 LYs and 9.38 QALYs for $157,576. The ICER of early vs delayed use was $43,136/LY and $50,104/QALY, which was cost-effective at the WTP threshold of $100,000. The PSA result indicated that a 94.6% likelihood of early use (vs delayed use) was cost-effective at the WTP threshold of $100,000 per QALY.
This study suggests that giving abemaciclib in the early stage rather than waiting until patients develop metastatic disease (current standard of care in MBC) is a cost-effective strategy.
阿贝西利新获批用于激素受体阳性（HR+）、人表皮生长因子受体 2 阴性（HER2-）高危早期乳腺癌（EBC）。临床指南推荐阿贝西利作为 HR+/HER2-EBC（早期使用）或 HR+/HER2-转移性乳腺癌（MBC）（延迟使用）的一线治疗药物。
比较 HR+/HER2-高危 EBC 早期与延迟使用阿贝西利的成本效益。早期使用定义为阿贝西利联合内分泌治疗作为 EBC 的一线治疗，随后用氟维司群治疗 MBC。延迟使用定义为 EBC 接受内分泌治疗，随后用阿贝西利联合氟维司群治疗 MBC。
从第三方美国支付者的角度，采用 5 状态模型来估算假设 HR+/HER2-EBC 患者的终生成本、生命年（LY）和质量调整生命年（QALY）。主要临床和安全性数据来自 monarchE 和 MONARCH 2 临床试验。成本、效用和不良事件的减效值均来自文献。我们计算了早期与延迟使用阿贝西利的增量成本效益比（ICER），并将其与 100,000 美元/LY 或 QALY 的支付意愿（WTP）阈值进行了比较。进行了确定性和概率敏感性分析（PSA）以检验基础模型的稳健性。
基础情况分析显示，早期使用可获得 21.08 LY 和 17.93 QALY，成本为 586,213 美元，而延迟使用可获得 11.14 LY 和 9.38 QALY，成本为 157,576 美元。早期与延迟使用的 ICER 分别为 43,136 美元/LY 和 50,104 美元/QALY，在 100,000 美元/WTP 阈值下具有成本效益。PSA 结果表明，早期使用（与延迟使用相比）具有 94.6%的可能性在 100,000 美元/QALY 的 WTP 阈值下具有成本效益。
这项研究表明，在疾病早期阶段（目前 MBC 的标准治疗）给予阿贝西利而非等到患者出现转移性疾病，是一种具有成本效益的策略。
