ASYMPTOTIC INFERENCE WITH RESPONSE-ADAPTIVE TREATMENT ALLOCATION DESIGNS
成果类型：
Article
署名作者：
ROSENBERGER, WF
刊物名称：
ANNALS OF STATISTICS
ISSN/ISSBN：
0090-5364
DOI：
10.1214/aos/1176349412
发表日期：
1993
页码：
2098-2107
关键词：
摘要：
A response-adaptive treatment allocation design for a clinical trial attempts to place the majority of patients on the treatment that appears more successful, based on the responses of patients already treated. One example of such a design is the randomized play-the-winner rule developed by Wei and Durham, which randomizes the treatment assignment probabilities according to the outcomes of treatments previously assigned. For a trial with dichotomous treatment responses and a randomized play-the-winner assignment scheme, exact small sample permutation tests of the hypothesis of equal treatment effects and large sample tests based on a population model have previously been developed. We present a large sample permutation test statistic for this case; under certain conditions on the sequence of responses, the test statistic is shown to be asymptotically normal. For a trial with a continuous response variable, we develop a rank-based analog of the randomized play-the-winner assignment scheme. Simulation evidence in both cases suggests that a normal approximation to the test statistic works well for moderate-sized trials, with some conservatism in the extreme tails.