Estimating the optimal individualized treatment rule from a cost-effectiveness perspective.
Estimating the optimal individualized treatment rule from a cost-effectiveness perspective.
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作者：
Xu Y;Greene TH;Bress AP;Sauer BC;Bellows BK;Zhang Y;Weintraub WS;Moran AE;Shen J
最佳个体化治疗规则（ITR）根据受试者特征提供定制化治疗建议，以根据精准医学的目标最大限度地提高临床获益。因此，人们越来越有兴趣开发统计工具，以估计最佳的ITR在循证研究。从卫生经济学的角度来看，决策者考虑卫生收益和干预措施的增量成本之间的权衡，以确定优先事项和分配资源。然而，大多数关于ITR的工作都侧重于最大限度地提高处理效果，而不考虑成本。在本文中，我们共同考虑疗效和成本对治疗决策的影响，并在复合结果设置下定义ITR，以便我们通过直接优化确定最具成本效益的ITR，该ITR可解释个体水平的异质性。特别是，我们提出了一个基于决策树的统计学习算法，使用一个基于净货币利益的奖励，以提供最佳ITR的非参数估计。我们提供了几种方法来估计作为主题特征的函数的ITR的奖励。我们提出了每种方法的优点和缺点，并通过比较它们在模拟研究中的表现提供了实用的指导方针。我们通过评估收缩压干预试验（SPRINT）研究的强化血压控制的预计15年个性化成本效益，说明了我们模拟的最佳方法。
Optimal individualized treatment rules (ITRs) provide customized treatment recommendations based on subject characteristics to maximize clinical benefit in accordance with the objectives in precision medicine. As a result, there is growing interest in developing statistical tools for estimating optimal ITRs in evidence-based research. In health economic perspectives, policy makers consider the tradeoff between health gains and incremental costs of interventions to set priorities and allocate resources. However, most work on ITRs has focused on maximizing the effectiveness of treatment without considering costs. In this paper, we jointly consider the impact of effectiveness and cost on treatment decisions and define ITRs under a composite-outcome setting, so that we identify the most cost-effective ITR that accounts for individual-level heterogeneity through direct optimization. In particular, we propose a decision-tree–based statistical learning algorithm that uses a net-monetary-benefit–based reward to provide nonparametric estimations of the optimal ITR. We provide several approaches to estimating the reward underlying the ITR as a function of subject characteristics. We present the strengths and weaknesses of each approach and provide practical guidelines by comparing their performance in simulation studies. We illustrate the top-performing approach from our simulations by evaluating the projected 15-year personalized cost-effectiveness of the intensive blood pressure control of the Systolic Blood Pressure Intervention Trial (SPRINT) study.
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影响因子：
24
作者：
Anderson, Jeffrey L.;Heidenreich, Paul A.;Shaw, Leslee J.
通讯作者：
Shaw, Leslee J.
影响因子：
15.8
作者：
Basu S;Sussman JB;Rigdon J;Steimle L;Denton BT;Hayward RA
通讯作者：
Hayward RA
影响因子：
24
作者：
Kohli-Lynch, Ciaran N.;Bellows, Brandon K.;Moran, Andrew E.
通讯作者：
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影响因子：
1.9
作者：
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通讯作者：
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影响因子：
2.3
作者：
Li, Jiaqi;Vachani, Anil;Mitra, Nandita
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Mitra, Nandita