Kang Dong-Won, Lynn Patricio B, Wang Li, Zhou Shouhao, Shen Chan
Department of Surgery, Penn State College of Medicine, The Pennsylvania State University, 500 University Drive, H151, Hershey, PA, 17033-0850, USA.
Department of Public Health Sciences, Penn State College of Medicine, Hershey, PA, USA.
Pharmacoecon Open. 2025 Jan;9(1):93-101. doi: 10.1007/s41669-024-00529-z. Epub 2024 Oct 8.
The FRESCO-2 trial established the efficacy and safety of fruquintinib in patients with refractory metastatic colorectal cancer. However, its cost-effectiveness in the US context is not well documented.
This study evaluates the cost-effectiveness of fruquintinib versus placebo for this patient population from the perspective of US payers.
We developed a partitioned survival model on the basis of patient-level data reconstructed from the survival curves of the FRESCO-2 trial. Parametric estimation was conducted to estimate long-term clinical outcomes and medical costs over a lifetime horizon. Cost inputs and utilities were sourced from public data and previous literature. We used a discount rate of 3.0% per year for both clinical outcomes and costs. We adopted an incremental cost-effectiveness ratio (ICER) threshold of US$100,000 per quality-adjusted life-year (QALY) gained. We performed sensitivity and scenario analyses to examine the robustness of cost-effectiveness results.
Fruquintinib treatment resulted in incremental gains of 0.108 life years (LYs) and 0.073 QALYs compared with the placebo, at an additional cost of US$112,294, primarily driven by medication expenses. The ICER for fruquintinib versus placebo was calculated at US$1,037,855 per LY and US$1,546,619 per QALY gained, exceeding the predefined cost-effectiveness threshold. The cost-effectiveness results were robust across all sensitivity and scenario analyses.
Despite the survival benefit, fruquintinib was not cost-effective compared with the placebo in patients with refractory metastatic colorectal cancer in the US setting, on the basis of the conventional willingness-to-pay threshold. Our findings may provide a basis for informing the pricing and reimbursement decisions regarding fruquintinib.
FRESCO-2试验确立了呋喹替尼在难治性转移性结直肠癌患者中的疗效和安全性。然而，其在美国背景下的成本效益尚无充分记录。
本研究从美国医保支付方的角度评估呋喹替尼与安慰剂相比对该患者群体的成本效益。
我们基于从FRESCO-2试验生存曲线重建的患者层面数据开发了一个分段生存模型。进行参数估计以估计终身范围内的长期临床结局和医疗成本。成本投入和效用值来自公开数据和既往文献。我们对临床结局和成本均采用每年3.0%的贴现率。我们采用每获得一个质量调整生命年（QALY）100,000美元的增量成本效益比（ICER）阈值。我们进行了敏感性分析和情景分析以检验成本效益结果的稳健性。
与安慰剂相比，呋喹替尼治疗带来了0.108个生命年（LYs）和0.073个QALYs的增量收益，额外成本为112,294美元，主要由药物费用驱动。呋喹替尼与安慰剂相比的ICER计算为每增加1个LY为1,037,855美元，每获得1个QALY为1,546,619美元，超过了预先定义的成本效益阈值。成本效益结果在所有敏感性分析和情景分析中均稳健。
尽管有生存获益，但在美国背景下，基于传统的支付意愿阈值，呋喹替尼与安慰剂相比在难治性转移性结直肠癌患者中不具有成本效益。我们的研究结果可为呋喹替尼的定价和报销决策提供依据。
