A pseudo-response approach to constructing confidence intervals for the subset of patients expected to benefit from a new treatment.

ABSTRACT: In precision medicine, there is much interest in estimating the expected-to-benefit (EB) subset, i.e. the subset of patients who are expected to benefit from a new treatment based on a collection of baseline characteristics. There are many statistical methods for estimating the EB subset, most of which produce a 'point estimate' without a confidence statement to address uncertainty. Confidence intervals for the EB subset have been defined only recently, and their construction is a new area for methodological research. This article proposes a pseudo-response approach to EB subset estimation and confidence interval construction. Compared to existing methods, the pseudo-response approach allows us to focus on modelling a conditional treatment effect function (as opposed to the conditional mea

SUBMITTER: Liu W 

PROVIDER: S-EPMC11090399 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature