Department of Oncology, Xiangya Hospital, Central South University, Changsha, 410008, Hunan, China.
Department of Medical Oncology, The Sixth Affiliated Hospital of Sun Yat-Sen University, Guangzhou, 510655, China.
Adv Ther. 2021 Mar;38(3):1650-1659. doi: 10.1007/s12325-021-01627-8. Epub 2021 Feb 10.
Recently the phase 3 BEACON trial showed that the combination of encorafenib, cetuximab, and binimetinib versus cetuximab and irinotecan/FOLFIRI improved overall survival in pre-treated patients with metastatic colorectal cancer (mCRC) with BRAF V600E mutation. However, whether the benefits of these therapies justify their high costs has not been estimated in the USA. The purpose of this study was to evaluate the cost-effectiveness of BEC (binimetinib, encorafenib, and cetuximab), EC (encorafenib and cetuximab), and CI/CF (cetuximab with irinotecan or FOLFIRI) in patients with BRAF V600E-mutated mCRC after first- and second-line therapy.
A Markov model was constructed to determine the costs and effects of BEC, EC, and CI/CF on the basis of BEACON trial outcomes data. Health outcomes were measured in life years (LYs), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). Deterministic and probabilistic sensitivity analyses characterized parameters influencing cost-effectiveness. Subgroup analyses were conducted as well.
The QALYs gained in BEC, EC, and CI/CF were 0.62, 0.54, and 0.40, respectively. BEC resulted in ICERs of $883,895.73/QALY and $1,646,846.14/QALY versus CI/CF and EC, respectively. Compared with CI/CF, the ICER was $435,449.88/QALY in EC. The most sensitive parameters in the comparison among the three arms were the utilities of progressive disease and progression-free survival. Probabilistic sensitivity analyses showed that the probability of BEC and EC being cost-effective was 0%. In subgroup analyses, the ICER remained above the willingness-to-pay threshold of $150,000 per QALY.
BEC and EC were not cost-effective regimens for patients with pre-treated mCRC with BRAF V600E mutation.
最近,3 期 BEACON 试验表明,与西妥昔单抗和伊立替康/FOLFIRI 相比,encorafenib、cetuximab 和 binimetinib 的联合治疗可改善 BRAF V600E 突变的转移性结直肠癌(mCRC)患者经前期治疗后的总生存期。然而,这些治疗方法的益处是否超过其高昂的成本,尚未在美国进行评估。本研究旨在评估 BEC(binimetinib、encorafenib 和 cetuximab)、EC(encorafenib 和 cetuximab)和 CI/CF(西妥昔单抗联合伊立替康或 FOLFIRI)在接受过一线和二线治疗的 BRAF V600E 突变 mCRC 患者中的成本效益。
基于 BEACON 试验结果数据,使用 Markov 模型来确定 BEC、EC 和 CI/CF 的成本和效果。健康结果以生命年(LYs)、质量调整生命年(QALYs)和增量成本效益比(ICERs)来衡量。确定性和概率敏感性分析描述了影响成本效益的参数。此外还进行了亚组分析。
BEC、EC 和 CI/CF 获得的 QALYs 分别为 0.62、0.54 和 0.40。与 CI/CF 和 EC 相比,BEC 的 ICER 分别为 883895.73 美元/QALY 和 1646846.14 美元/QALY。与 CI/CF 相比,EC 的 ICER 为 435449.88 美元/QALY。三种治疗方案比较中最敏感的参数是进展性疾病和无进展生存期的效用。概率敏感性分析表明,BEC 和 EC 具有成本效益的概率均为 0%。在亚组分析中,ICER 仍高于每 QALY 150000 美元的意愿支付阈值。
对于 BRAF V600E 突变的经前期 mCRC 患者,BEC 和 EC 不是有效的治疗方案。