21. Causal Inference in Clinical Trials

  1. Carlo Berzuini,
  2. Philip Dawid and
  3. Luisa Bernardinelli
  1. Krista Fischer1,2 and
  2. Ian R. White1

Published Online: 25 JUN 2012

DOI: 10.1002/9781119945710.ch21

Causality: Statistical Perspectives and Applications

Causality: Statistical Perspectives and Applications

How to Cite

Fischer, K. and White, I. R. (2012) Causal Inference in Clinical Trials, in Causality: Statistical Perspectives and Applications (eds C. Berzuini, P. Dawid and L. Bernardinelli), John Wiley & Sons, Ltd, Chichester, UK. doi: 10.1002/9781119945710.ch21

Editor Information

  1. Statistical Laboratory, Centre for Mathematical Sciences, University of Cambridge, Cambridge, UK

Author Information

  1. 1

    MRC Biostatistics Unit, Cambridge, UK

  2. 2

    Estonian Genome Center, University of Tartu, Estonia

Publication History

  1. Published Online: 25 JUN 2012
  2. Published Print: 13 JUL 2012

Book Series:

  1. Wiley Series in Probability and Statistics

Book Series Editors:

  1. Walter A. Shewhart and
  2. Samuel S. Wilks

ISBN Information

Print ISBN: 9780470665565

Online ISBN: 9781119945710



  • causal inference in clinical trials;
  • causal effect of exposures, and unobserved confounding;
  • ideal randomized trial, a one-to-one correspondence;
  • ideal randomized trial, and protocol nonadherence;
  • causal models, in randomized trials, EPHT trial;
  • EPHT, effect of HT on average healthcare costs;
  • potential outcomes, counterfactual outcomes and effects;
  • crude (unadjusted) ITT analysis via potential outcomes;
  • adherence-adjusted ITT, treatment–effect heterogeneity at adherence levels;
  • SMM for ATT, principal stratification, parameters involved


This chapter contains sections titled:

  • Introduction

  • Causal effect of treatment in randomized trials

  • Estimation for a linear structural mean model

  • Alternative approaches for causal inference in randomized trials comparing experimental treatment with a control

  • Discussion

  • References