articleEpidemiologyJan 2, 2026HYBRID OA

The Ideal Trial: Defining Causal Estimands that Balance Relevance and Feasibility in Target Trial Emulations and Actual Randomized Trials

The University of Melbourne · Murdoch Children's Research Institute

PubMed
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Abstract

Causal inference is the goal of randomized trials and many observational studies. The first step in a formal causal inference framework is to define the causal estimand, and in both types of study this can be done mathematically or, equivalently, by specifying an ideal trial: a hypothetical perfect randomized experiment (with representative sample, perfect adherence, etc). The target trial framework is increasingly used for causal inference in observational studies, but clarity is lacking in how a target trial should be specified and how it relates to an ideal trial. Here, we review the mathematical and ideal trial approaches to defining a causal estimand, highlighting their equivalence and the need to balance…

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Topics & keywords

Keywords
  • Causal inference
  • Observational study
  • Relevance (law)
  • Randomized controlled trial
  • Inference
  • Ideal (ethics)
  • Causal model
  • CLARITY
UN Sustainable Development Goals
  • Good health and well-being
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