Efficient design and inference for multistage randomized trials of individualized treatment policies.

ABSTRACT: Clinical demand for individualized "adaptive" treatment policies in diverse fields has spawned development of clinical trial methodology for their experimental evaluation via multistage designs, building upon methods intended for the analysis of naturalistically observed strategies. Because often there is no need to parametrically smooth multistage trial data (in contrast to observational data for adaptive strategies), it is possible to establish direct connections among different methodological approaches. We show by algebraic proof that the maximum likelihood (ML) and optimal semiparametric (SP) estimators of the population mean of the outcome of a treatment policy and its standard error are equal under certain experimental conditions. This result is used to develop a unified and efficie

SUBMITTER: Dawson R 

PROVIDER: S-EPMC3276275 | biostudies-literature | 2012 Jan

REPOSITORIES: biostudies-literature