Placebo Response as a Latent Characteristic: Application to Analysis of Sequential Parallel Comparison Design Studies

Published

Journal Article

© 2018, © 2018 American Statistical Association. In clinical trials, placebo response can affect the inference about efficacy of the studied treatment. It is important to have a robust way to classify trial subjects with respect to their response to placebo. Simple, criterion-based classification may lead to classification error and bias the inference. The uncertainty about placebo response characteristic has to be factored into the treatment effect estimation. We propose a novel approach that views the placebo response as a latent characteristic and the study sample as an unlabeled mixture of “placebo responders” and “placebo nonresponders.” The likelihood-based methodology is used to estimate the treatment effect corrected for placebo response as defined within sequential parallel comparison design.

Full Text

Duke Authors

Cited Authors

  • Rybin, D; Lew, R; Pencina, MJ; Fava, M; Doros, G

Published Date

  • October 2, 2018

Published In

Volume / Issue

  • 113 / 524

Start / End Page

  • 1411 - 1430

Electronic International Standard Serial Number (EISSN)

  • 1537-274X

International Standard Serial Number (ISSN)

  • 0162-1459

Digital Object Identifier (DOI)

  • 10.1080/01621459.2017.1375930

Citation Source

  • Scopus