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

Journal Article (Journal Article)

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