In the present paper, personalized treatment means choosing the best treatment for a patient while taking into account certain relevant personal covariate values. We study the design of trials whose goal is to find the best treatment for a given patient with given covariates. We assume that the subjects in the trial represent a random sample from the population, and consider the allocation, possibly with randomization, of these subjects to the different treatment groups in a way that depends on their covariates. We derive approximately optimal allocations, aiming to minimize expected regret, assuming that future patients will arrive from the same population as the trial subjects. We find that, for the case of two treatments, an approximately optimal allocation design 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 the optimal allocation design does depend on the covariates as we show for specific scenarios. Another finding is that the optimal allocation can vary a lot as a function of the sample size, and that randomized allocations are relevant for relatively small samples, and may not be needed for very large studies.
翻译:在本文件中,个性化治疗意味着选择病人的最佳治疗方法,同时考虑到某些相关的个人共变价值。我们研究试验的设计,其目的在于为特定病人找到有特定共变价值的最佳治疗方法。我们假定试验对象是来自人口的随机抽样,并考虑以取决于其共变的方式将这些治疗对象分给不同的治疗组,可能的话是随机分给不同治疗组。我们得到的分配大致是最佳的,目的是尽量减少预期的遗憾,假设未来的病人将来自与试验对象相同的人群。我们发现,就两种治疗而言,大约最佳的分配设计并不取决于共同变数的价值,而只取决于答复的差异。相反,就三种治疗而言,最佳分配设计取决于我们为具体情景所显示的共变数。另一个发现是,最佳的分配方法可以因抽样大小的函数而有很大差异,而且随机分配与相对较小的样本有关,可能不需要进行非常大的研究。