Refaat Tamer, Choi Mehee, Gaber Germaine, Kiel Krystyna, Mehta Minesh, Gradishar William, Small William
Departments of *Radiation Oncology ∥Medicine-Hematology/Oncology, Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Feinberg School of Medicine §Department of Radiation Oncology, Rush University Medical Center, Chicago, IL †Department of Clinical Oncology ‡Clinical Research Center, Faculty of Medicine, Alexandria University, Alexandria, Egypt.
Am J Clin Oncol. 2014 Oct;37(5):480-5. doi: 10.1097/COC.0b013e31827e4e9a.
Metastatic breast cancer (MBC) remains an incurable disease despite advances in treatment modalities. In 2008, the FDA approved bevacizumab with paclitaxel for the initial treatment of HER2-negative MBC. The approval was then officially revoked by the FDA in November 2011. However, both the European Medicines Agency and NCCN still endorse bevacizumab for this indication. One of the greatest challenges facing health care worldwide is reconciling incremental clinical benefits with exponentially rising costs. This study aimed to assess the cost-effectiveness of bevacizumab with paclitaxel for HER2-negative MBC.
A Markov decision tree using Data 3.5 (TreeAge Software Inc.) was created for decision and cost-effectiveness analyses of using bevacizumab plus paclitaxel versus paclitaxel alone as first-line chemotherapy in HER2-negative MBC using efficacy and toxicity data from the E2100 study. The model was designed from the patient and payer perspectives and sensitivity analyses were run.
The marginal cost between paclitaxel alone versus bevacizumab and paclitaxel was 86k with a marginal efficacy of 0.369 quality-adjusted life-years and marginal cost effectiveness of 232,720.72 USD. The expected outcome value was 1.86 for bevacizumab and paclitaxel and 1.67 for paclitaxel alone. The combination was not cost effective and only a marginal survival advantage was observed.
This study demonstrates that, despite a significant progression-free survival advantage, the addition of bevacizumab to paclitaxel is not cost effective for the cohort of patients with HER2-negative MBC included in our analysis. Such data could be informative to policymakers who consider the health economics and incremental cost-effectiveness of medical therapies.
尽管治疗方式有所进步，但转移性乳腺癌（MBC）仍然是一种无法治愈的疾病。2008年，美国食品药品监督管理局（FDA）批准贝伐单抗联合紫杉醇用于HER2阴性MBC的初始治疗。然而，该批准于2011年11月被FDA正式撤销。尽管如此，欧洲药品管理局和美国国立综合癌症网络（NCCN）仍支持贝伐单抗用于该适应症。全球医疗保健面临的最大挑战之一是如何平衡逐渐增加的临床益处与呈指数级上升的成本。本研究旨在评估贝伐单抗联合紫杉醇治疗HER2阴性MBC的成本效益。
使用TreeAge Software Inc.公司的Data 3.5创建了一个马尔可夫决策树，以E2100研究的疗效和毒性数据为基础，对贝伐单抗联合紫杉醇与单纯紫杉醇作为HER2阴性MBC一线化疗方案进行决策和成本效益分析。该模型从患者和支付方的角度进行设计，并进行了敏感性分析。
单纯紫杉醇与贝伐单抗联合紫杉醇之间的边际成本为86,000美元，边际疗效为0.369个质量调整生命年，边际成本效益为232,720.72美元。贝伐单抗联合紫杉醇的预期结果值为1.86，单纯紫杉醇为1.67。联合治疗不具有成本效益，仅观察到边际生存优势。
本研究表明，尽管贝伐单抗联合紫杉醇具有显著的无进展生存优势，但对于我们分析中纳入的HER2阴性MBC患者队列，添加贝伐单抗并不具有成本效益。这些数据可能对考虑医疗疗法的健康经济学和增量成本效益的政策制定者具有参考价值。
