个性化治疗规则开发的最优设计

Optimal designs for the development of personalized treatment rules

Scandinavian Journal of Statistics · 2022
被引 2
ABS 3

中文导读

研究了多臂平行组临床试验中,如何优化治疗分配以估计个性化治疗规则,发现两治疗组时最优分配仅取决于方差,三组以上则依赖协变量和回归系数。

Abstract

Abstract We study the design of multi‐armed parallel group clinical trials to estimate personalized treatment rules that identify the best treatment for a given patient with given covariates. Assuming that the outcomes in each treatment arm are given by a homoscedastic linear model, with possibly different variances between treatment arms, and that the trial subjects form a random sample from an unselected overall population, we optimize the (possibly randomized) treatment allocation allowing the allocation rates to depend on the covariates. We find that, for the case of two treatments, the approximately optimal allocation rule does not depend on the value of the covariates but only on the variances of the responses. In contrast, for the case of three treatments or more, the optimal treatment allocation does depend on the values of the covariates as well as the true regression coefficients. The methods are illustrated with a recently published dietary clinical trial.

临床试验设计个性化医疗统计学计量经济学