Division of Hematology/Oncology, University of Miami Sylvester Comprehensive Cancer Center, Miami, FL, USA.
Breast Cancer Res Treat. 2012 Apr;132(2):747-51. doi: 10.1007/s10549-011-1919-y. Epub 2011 Dec 27.
Bevacizumab in combination with chemotherapy increases progression-free survival (PFS), but not overall survival when compared to chemotherapy alone in the treatment of metastatic breast cancer (MBC). Recently in November, 2011 the Food and drug administration revoked approval of bevacizumab in combination with paclitaxel for the treatment of MBC. The European Medicines Agency, in contrast, maintained its approval of bevacizumab in MBC. While neither agency considers health economics in their decision-making process, one of the greatest challenges in oncology practice today is to reconcile hard-won small incremental clinical benefits with exponentially rising costs. To inform policy-makers in the US, this study aimed to assess the cost-effectiveness of bevacizumab/paclitaxel in MBC, from a payer perspective. We created a decision analytical model using efficacy and adverse events data from the ECOG 2100 trial. Health utilities were derived from available literature. Costs were obtained from the Center for Medicare Services Drug Payment Table and Physician Fee Schedule and are represented in 2010 US dollars. Quality-adjusted life-years (QALY) and incremental cost-effectiveness ratio (ICER) were calculated. Sensitivity analyses were performed. Bevacizumab added 0.49 years of PFS and 0.135 QALY with an incremental cost of $100,300, and therefore a cost of $204,000 per year of PFS gained and an ICER of $745,000 per QALY. The main drivers of the model were drug acquisition cost, PFS, and health utility values. Using a threshold of $150,000/QALY, drug price would have to be reduced by nearly 80% or alternatively PFS increased by 10 months to make bevacizumab cost-effective. The results of the model were robust in sensitivity analyses. Bevacizumab plus paclitaxel is not cost-effective in treating MBC. Value-based pricing and the development of biomarkers to improve patient selection are needed to better define the role of the drug in this population.
贝伐珠单抗联合化疗可延长转移性乳腺癌（MBC）患者的无进展生存期（PFS），但与单独化疗相比，不能延长总生存期。2011 年 11 月，食品和药物管理局撤销了贝伐珠单抗联合紫杉醇治疗 MBC 的批准。相比之下，欧洲药品管理局维持了贝伐珠单抗在 MBC 中的批准。虽然这两个机构在决策过程中都不考虑卫生经济学，但目前肿瘤学实践中最大的挑战之一是协调来之不易的微小临床获益与指数级上升的成本。为了向美国的决策者提供信息，本研究旨在从支付者的角度评估贝伐珠单抗/紫杉醇治疗 MBC 的成本效益。我们使用 ECOG 2100 试验的疗效和不良事件数据创建了一个决策分析模型。健康效用值来自现有文献。成本来自医疗保险服务中心药物支付表和医师费用表，并以 2010 年的美元表示。计算了质量调整生命年（QALY）和增量成本效益比（ICER）。进行了敏感性分析。贝伐珠单抗使 PFS 延长了 0.49 年，QALY 增加了 0.135，增量成本为 100300 美元，因此，每年每获得 1 个 PFS 的成本为 204000 美元，ICER 为 745000 美元/QALY。模型的主要驱动因素是药物采购成本、PFS 和健康效用值。如果以 15 万美元/QALY 为阈值，药物价格必须降低近 80%，或者 PFS 增加 10 个月，才能使贝伐珠单抗具有成本效益。模型的结果在敏感性分析中是稳健的。贝伐珠单抗联合紫杉醇治疗 MBC 并不具有成本效益。需要基于价值的定价和开发生物标志物来改善患者选择，以更好地确定该药在该人群中的作用。
