Compliance subsampling designs for comparative research: estimation and optimal planning.
Compliance subsampling designs for comparative research: estimation and optimal planning.
复制标题
用于比较研究的合规二次抽样设计：估计和最优规划。
DOI：
10.1111/j.0006-341x.2001.00899.x
复制
发表时间：
2001
期刊：
影响因子：
--
通讯作者：
Baker,SG
中科院分区：
文献类型：
--
作者：
Frangakis,CE;Baker,SG
对于治疗不依从性的研究，最近开发了分析，以更好地估计治疗疗效。然而，测量依从性数据的优势和成本对研究设计有影响，尚未系统地探讨。为了估计更好的治疗效果与较低的成本，我们提出了一类新的依从性子抽样（CSS）设计，受试者被分配治疗后，依从性行为的测量，只有亚组的受试者。允许子样本的大小与治疗分配、分配概率、总样本量、结局和依从性的预期分布以及研究的成本参数相关。CSS设计方法涉及到先前的工作（i）在两阶段设计中，协变量是二次抽样和（ii）因果推理，因为二次抽样后随机化的合规行为是不是真正的协变量的利益。对于每个CSS设计，我们开发了有效的估计治疗效果下的二进制结果和全或无观察到的遵守。然后，我们得出一个最小的成本CSS设计，达到所需的精度估计治疗效果。我们比较的CSS设计的属性，在研究中的患者选择在生命的尽头医疗保健的传统协议。
For studies with treatment noncompliance, analyses have been developed recently to better estimate treatment efficacy. However, the advantage and cost of measuring compliance data have implications on the study design that have not been as systematically explored. In order to estimate better treatment efficacy with lower cost, we propose a new class of compliance subsampling (CSS) designs where, after subjects are assigned treatment, compliance behavior is measured for only subgroups of subjects. The sizes of the subsamples are allowed to relate to the treatment assignment, the assignment probability, the total sample size, the anticipated distributions of outcome and compliance, and the cost parameters of the study. The CSS design methods relate to prior work (i) on two-phase designs in which a covariate is subsampled and (ii) on causal inference because the subsampled postrandomization compliance behavior is not the true covariate of interest. For each CSS design, we develop efficient estimation of treatment efficacy under binary outcome and all-or-none observed compliance. Then we derive a minimal cost CSS design that achieves a required precision for estimating treatment efficacy. We compare the properties of the CSS design to those of conventional protocols in a study of patient choices for medical care at the end of life.