Estimation of multivariate treatment effects in contaminated clinical trials

Pharm Stat. 2022 May;21(3):535-565. doi: 10.1002/pst.2185. Epub 2021 Dec 23.

Abstract

The paper addresses estimating and testing treatment effects with multivariate outcomes in clinical trials where imperfect diagnostic devices are used to assign subjects to treatment groups. The paper focuses on the pre-post design and proposes two novel methods for estimating and testing treatment effects. In addition, methods for sample size and power calculations are developed. The methods are compared with each other and with a traditional method in a simulation study. The new methods show significant advantages in terms of power, coverage probability, and required sample size. The application of the methods is illustrated with data from electroencephalogram (EEG) recordings of alcoholic and control subjects.

Keywords: EM algorithm; F-approximation; finite mixture; method of moments; sample size determination.

MeSH terms

  • Clinical Trials as Topic
  • Computer Simulation
  • Humans
  • Probability
  • Research Design*
  • Sample Size