NCIC Clinical Trials Group, Kingston, ON, Canada.
J Natl Cancer Inst. 2010 Mar 3;102(5):298-306. doi: 10.1093/jnci/djp518. Epub 2010 Feb 16.
BACKGROUND: The NCIC Clinical Trials Group conducted the BR.21 trial, a randomized placebo-controlled trial of erlotinib (an epidermal growth factor receptor tyrosine kinase inhibitor) in patients with previously treated advanced non-small cell lung cancer. This trial accrued patients between August 14, 2001, and January 31, 2003, and found that overall survival and quality of life were improved in the erlotinib arm than in the placebo arm. However, funding restrictions limit access to erlotinib in many countries. We undertook an economic analysis of erlotinib treatment in this trial and explored different molecular and clinical predictors of outcome to determine the cost-effectiveness of treating various populations with erlotinib. METHODS: Resource utilization was determined from individual patient data in the BR.21 trial database. The trial recruited 731 patients (488 in the erlotinib arm and 243 in the placebo arm). Costs arising from erlotinib treatment, diagnostic tests, outpatient visits, acute hospitalization, adverse events, lung cancer-related concomitant medications, transfusions, and radiation therapy were captured. The incremental cost-effectiveness ratio was calculated as the ratio of incremental cost (in 2007 Canadian dollars) to incremental effectiveness (life-years gained). In exploratory analyses, we evaluated the benefits of treatment in selected subgroups to determine the impact on the incremental cost-effectiveness ratio. RESULTS: The incremental cost-effectiveness ratio for erlotinib treatment in the BR.21 trial population was $94,638 per life-year gained (95% confidence interval = $52,359 to $429,148). The major drivers of cost-effectiveness included the magnitude of survival benefit and erlotinib cost. Subgroup analyses revealed that erlotinib may be more cost-effective in never-smokers or patients with high EGFR gene copy number. CONCLUSION: With an incremental cost-effectiveness ratio of $94 638 per life-year gained, erlotinib treatment for patients with previously treated advanced non-small cell lung cancer is marginally cost-effective. The use of molecular predictors of benefit for targeted agents may help identify more or less cost-effective subgroups for treatment.
背景：NCIC 临床研究组开展了 BR.21 试验，这是一项针对已接受治疗的晚期非小细胞肺癌患者的厄洛替尼（表皮生长因子受体酪氨酸激酶抑制剂）随机安慰剂对照试验。该试验于 2001 年 8 月 14 日至 2003 年 1 月 31 日期间入组患者，结果发现厄洛替尼组的总生存期和生活质量均优于安慰剂组。然而，资金限制限制了许多国家获得厄洛替尼的机会。我们对该试验中的厄洛替尼治疗进行了经济分析，并探讨了不同的分子和临床预后预测因素，以确定用厄洛替尼治疗各种人群的成本效益。
方法：从 BR.21 试验数据库中的个体患者数据中确定资源利用情况。该试验招募了 731 名患者（厄洛替尼组 488 名，安慰剂组 243 名）。记录了厄洛替尼治疗、诊断测试、门诊就诊、急性住院、不良事件、肺癌相关伴随药物、输血和放射治疗所产生的费用。增量成本效益比计算为增量成本（2007 年加拿大元）与增量效果（获得的生命年）之比。在探索性分析中，我们评估了选定亚组的治疗益处，以确定对增量成本效益比的影响。
结果：BR.21 试验人群中厄洛替尼治疗的增量成本效益比为每获得 1 个生命年需花费 94638 加元（95%置信区间=52359 至 429148 加元）。成本效益的主要驱动因素包括生存获益的大小和厄洛替尼的成本。亚组分析显示，厄洛替尼在从不吸烟者或 EGFR 基因拷贝数高的患者中可能更具成本效益。
结论：厄洛替尼治疗既往治疗的晚期非小细胞肺癌的增量成本效益比为每获得 1 个生命年需花费 94638 加元，略具有成本效益。对于靶向药物，使用获益的分子预测因子可能有助于确定更具成本效益或成本效益较低的治疗亚组。
J Natl Cancer Inst. 2010-2-16
Expert Rev Pharmacoecon Outcomes Res. 2012-8
Health Technol Assess. 2009-6
J Natl Cancer Inst. 2010-3-3
Health Technol Assess. 2010-10
Expert Rev Pharmacoecon Outcomes Res. 2009-10
Curr Oncol. 2019-4-1
Pharmacoeconomics. 2019-5
World J Oncol. 2013-10
Zotero 插件