Global sensitivity analysis for repeated measures studies with informative drop-out: A semi-parametric approach

Biometrics. 2018 Mar;74(1):207-219. doi: 10.1111/biom.12729. Epub 2017 May 23.

Abstract

In practice, both testable and untestable assumptions are generally required to draw inference about the mean outcome measured at the final scheduled visit in a repeated measures study with drop-out. Scharfstein et al. (2014) proposed a sensitivity analysis methodology to determine the robustness of conclusions within a class of untestable assumptions. In their approach, the untestable and testable assumptions were guaranteed to be compatible; their testable assumptions were based on a fully parametric model for the distribution of the observable data. While convenient, these parametric assumptions have proven especially restrictive in empirical research. Here, we relax their distributional assumptions and provide a more flexible, semi-parametric approach. We illustrate our proposal in the context of a randomized trial for evaluating a treatment of schizoaffective disorder.

Keywords: Bootstrap; Cross-validation; Exponential tilting; Identifiability; Jackknife; One-step estimator; Plug-in estimator; Selection bias.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Humans
  • Models, Statistical*
  • Psychotic Disorders / therapy
  • Randomized Controlled Trials as Topic
  • Reproducibility of Results
  • Research Design* / statistics & numerical data
  • Statistical Distributions*
  • Treatment Outcome*