An outcome model approach to transporting a randomized controlled trial results to a target population.

Journal: Journal of the American Medical Informatics Association : JAMIA
Published Date: May 1, 2019

Abstract

OBJECTIVE: Participants enrolled into randomized controlled trials (RCTs) often do not reflect real-world populations. Previous research in how best to transport RCT results to target populations has focused on weighting RCT data to look like the target data. Simulation work, however, has suggested that an outcome model approach may be preferable. Here, we describe such an approach using source data from the 2 × 2 factorial NAVIGATOR (Nateglinide And Valsartan in Impaired Glucose Tolerance Outcomes Research) trial, which evaluated the impact of valsartan and nateglinide on cardiovascular outcomes and new-onset diabetes in a prediabetic population.

Authors

  • Benjamin A Goldstein
    Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina.
  • Matthew Phelan
    Center for Predictive Medicine, Duke Clinical Research Institute, Durham, North Carolina.
  • Neha J Pagidipati
    Center for Predictive Medicine, Duke Clinical Research Institute, Durham, North Carolina.
  • Rury R Holman
    Endocrinology and Metabolism, University of Oxford, Oxford, United Kingdom.
  • Michael J Pencina
    Duke Clinical Research Institute, Department of Biostatistics and Bioinformatics, Duke University, Durham, North Carolina.
  • Elizabeth A Stuart
    Department of Mental Health, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD 21205.

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