Optimal Designs of Two-Phase Studies.

Optimal Designs of Two-Phase Studies.
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两阶段研究的最优设计。

DOI:
10.1080/01621459.2019.1671200
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发表时间:
2020
影响因子:
3.7
通讯作者:
Lin DY
Lin DY
中科院分区:
数学1区
文献类型:
--
作者:
Tao R;Zeng D;Lin DY

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两阶段设计是一种具有成本效益的抽样策略,用于在某些协变量过于昂贵而无法在所有研究受试者中测量时评估协变量对结局的影响。在这种设计下,在第一阶段中对所有受试者测量结果和廉价协变量,并且第一阶段信息用于选择受试者以在第二阶段中测量昂贵协变量。以往对两阶段研究的研究主要集中在推理过程,而不是设计方面。我们研究了两阶段研究的设计效率,通过估计昂贵协变量回归系数的半参数效率界限来衡量。我们考虑一般的两阶段研究,其中结果变量可以是连续的、离散的或删失的,并且第二阶段采样可以以任何方式依赖于第一阶段数据。我们开发了最优或近似最优的两阶段设计,这可以大大提高效率比现有的设计。我们通过广泛的模拟研究和两项大型医学研究证明了新设计相对于现有设计的改进。
The two-phase design is a cost-effective sampling strategy to evaluate the effects of covariates on an outcome when certain covariates are too expensive to be measured on all study subjects. Under such a design, the outcome and inexpensive covariates are measured on all subjects in the first phase and the first-phase information is used to select subjects for measurements of expensive covariates in the second phase. Previous research on two-phase studies has focused largely on the inference procedures rather than the design aspects. We investigate the design efficiency of the two-phase study, as measured by the semiparametric efficiency bound for estimating the regression coefficients of expensive covariates. We consider general two-phase studies, where the outcome variable can be continuous, discrete, or censored, and the second-phase sampling can depend on the first-phase data in any manner. We develop optimal or approximately optimal two-phase designs, which can be substantially more efficient than the existing designs. We demonstrate the improvements of the new designs over the existing ones through extensive simulation studies and two large medical studies.
DOI: 10.1111/biom.12013
发表时间: 2013-06
期刊: Biometrics
影响因子: 1.9
作者:
Schildcrout JS;Garbett SP;Heagerty PJ
通讯作者: Heagerty PJ
DOI: 10.1038/nature13595
发表时间: 2014-07-24
期刊: NATURE
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发表时间: 1998-01-01
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发表时间: 1997-01-01
期刊: JOURNAL OF THE ROYAL STATISTICAL SOCIETY SERIES B-METHODOLOGICAL
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