Faculty of Medicine, University of Calgary, Health Research Innovation Centre, 3280 Hospital Dr NW, Room 3C56, Calgary, AB T2N 4Z6.
Am J Manag Care. 2011 May;17 Suppl 5 Developing(0 5 0):SP61-70.
Decision makers must make decisions without complete information. That uncertainty can be decreased when economic evaluations use local data and can be quantified by considering the variability of all model inputs concurrently per international evaluation guidelines. It is unclear how these recommendations have been implemented in evaluations of targeted cancer therapy. By using economic evaluations of adjuvant trastuzumab, we have assessed the extent to which decision support recommendations were adopted.
Systematic review.
Published economic evaluations of adjuvant trastuzumab treatment in early-stage breast cancer were examined as an established example of targeted therapy. Canadian, United Kingdom, and US economic evaluation guidelines were reviewed to establish extraction criteria. Extraction characterized the use of effectiveness evidence and local data sources for model parameters, sensitivity analysis methods (scenario, univariate, multivariate, and probabilistic), and uncertainty representation (ie, cost-effectiveness plane, scatterplot, confidence ellipses, tornado diagrams, cost-effectiveness acceptability curve).
Fifteen economic evaluations of adjuvant trastuzumab were identified in the literature. Local data were used to estimate costs (15 of 15) and utilities rarely (2 of 15) but not trastuzumab efficacy. Univariate sensitivity analysis was most common (12 of 15), whereas probabilistic analysis was less frequent (10 of 15). Two-thirds of all studies provided visual representation of results and decision uncertainty.
Authors of adjuvant trastuzumab economic evaluations rarely use local data beyond costs. Quantification of uncertainty and its representation also fell short of guideline recommendations. This review demonstrates that economic evaluations of adjuvant trastuzumab, as an example of targeted cancer therapy, can be improved for decision-making support.
决策者必须在不完全了解信息的情况下做出决策。当经济评估使用当地数据并根据国际评估指南同时考虑所有模型输入的变异性来量化不确定性时，不确定性可以降低。尚不清楚这些建议在靶向癌症治疗的评估中是如何实施的。通过使用曲妥珠单抗辅助治疗的经济评估，我们评估了支持决策的建议被采用的程度。
系统评价。
检查了早期乳腺癌曲妥珠单抗辅助治疗的已发表经济评估，作为靶向治疗的既定范例。审查了加拿大、英国和美国的经济评估指南，以确定提取标准。提取特征在于模型参数的有效性证据和本地数据源的使用、敏感性分析方法（情景、单变量、多变量和概率）以及不确定性表示（即成本效果平面、散点图、置信椭圆、龙卷图、成本效果可接受性曲线）。
文献中确定了 15 项曲妥珠单抗辅助治疗的经济评估。当地数据用于估计成本（15 项中的 15 项），很少用于估计效用（15 项中的 2 项），但不用于估计曲妥珠单抗疗效。最常见的是单变量敏感性分析（15 项中的 12 项），而概率分析则不太常见（15 项中的 10 项）。三分之二的研究都提供了结果和决策不确定性的直观表示。
曲妥珠单抗辅助治疗经济评估的作者很少使用成本以外的本地数据。不确定性的量化及其表示也不符合指南建议。本综述表明，作为靶向癌症治疗范例的曲妥珠单抗辅助治疗的经济评估可以在支持决策方面得到改进。
