Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds, UK.
J Natl Cancer Inst. 2012 Jan 4;104(1):56-66. doi: 10.1093/jnci/djr484. Epub 2011 Dec 2.
Multi-parameter genomic tests identify patients with early-stage breast cancer who are likely to derive little benefit from adjuvant chemotherapy. These tests can potentially spare patients the morbidity from unnecessary chemotherapy and reduce costs. However, the costs of the test must be balanced against the health benefits and cost savings produced. This economic evaluation compared genomic test-directed chemotherapy using the Oncotype DX 21-gene assay with chemotherapy for all eligible patients with lymph node-positive, estrogen receptor-positive early-stage breast cancer.
We performed a cost-utility analysis using a state transition model to calculate expected costs and benefits over the lifetime of a cohort of women with estrogen receptor-positive lymph node-positive breast cancer from a UK perspective. Recurrence rates for Oncotype DX-selected risk groups were derived from parametric survival models fitted to data from the Southwest Oncology Group 8814 trial. The primary outcome was the incremental cost-effectiveness ratio, expressed as the cost (in 2011 GBP) per quality-adjusted life-year (QALY). Confidence in the incremental cost-effectiveness ratio was expressed as a probability of cost-effectiveness and was calculated using Monte Carlo simulation. Model parameters were varied deterministically and probabilistically in sensitivity analysis. Value of information analysis was used to rank priorities for further research.
The incremental cost-effectiveness ratio for Oncotype DX-directed chemotherapy using a recurrence score cutoff of 18 was £5529 (US $8852) per QALY. The probability that test-directed chemotherapy is cost-effective was 0.61 at a willingness-to-pay threshold of £30 000 per QALY. Results were sensitive to the recurrence rate, long-term anthracycline-related cardiac toxicity, quality of life, test cost, and the time horizon. The highest priority for further research identified by value of information analysis is the recurrence rate in test-selected subgroups.
There is substantial uncertainty regarding the cost-effectiveness of Oncotype DX-directed chemotherapy. It is particularly important that future research studies to inform cost-effectiveness-based decisions collect long-term outcome data.
多参数基因组测试可识别出早期乳腺癌患者，这些患者从辅助化疗中获益甚少。这些测试可以避免患者遭受不必要的化疗的痛苦，并降低成本。然而，必须权衡测试成本与健康收益和成本节约。这项经济评估比较了使用 Oncotype DX 21 基因检测的基因组检测指导的化疗与所有淋巴结阳性、雌激素受体阳性早期乳腺癌患者的化疗。
我们使用状态转移模型进行了成本效益分析，以计算从英国的角度来看，一群雌激素受体阳性淋巴结阳性乳腺癌患者的终生预期成本和收益。Oncotype DX 选择的风险组的复发率是从对 Southwest Oncology Group 8814 试验数据拟合的参数生存模型中得出的。主要结果是增量成本效益比，用质量调整生命年（QALY）表示每单位成本（2011 年英镑）。增量成本效益比的置信度用成本效益概率表示，并使用蒙特卡罗模拟计算。在敏感性分析中，通过确定性和概率方法对模型参数进行了变化。信息价值分析用于对进一步研究的优先级进行排序。
使用复发评分截断值为 18 的 Oncotype DX 指导化疗的增量成本效益比为每 QALY5529 英镑（8852 美元）。在愿意支付每 QALY30000 英镑的阈值下，测试指导化疗具有成本效益的概率为 0.61。结果对复发率、长期蒽环类相关心脏毒性、生活质量、测试成本和时间范围敏感。信息价值分析确定的进一步研究的最高优先级是测试选择亚组中的复发率。
Oncotype DX 指导化疗的成本效益存在很大的不确定性。未来的研究应特别关注收集基于成本效益的决策的长期结果数据。
