Pragmatic randomized trials are designed to support clinical decision-making rather than regulatory approval. The features of pragmatic trials (e.g., typical patients in typical care settings, active treatment strategies as comparators, unconcealed assignment, long-term outcomes) make their results more relevant for patients, clinicians, and other stakeholders. However, these features also introduce an increased risk of non-adherence and loss to follow-up, which reduce the value of the usual intention-to-treat analysis.
This document proposes a preliminary set of guidelines for more informative causal inference in pragmatic trials. We hope that a revised version of these draft guidelines will help guide the design and analysis of future pragmatic trials. We are therefore soliciting feedback to ensure that the content and citations of the revised guidelines reflect the best practices for estimation of causal effects in pragmatic trials.
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The development of these guidelines was supported through a Patient Centered Outcomes Research Institute (PCORI) award (ME-1503-8119). All statements in this report, including its findings and conclusions, are solely those of the authors and do not necessarily represent the views of the Patient-Centered Outcomes Research Institute (PCORI), its Board of Governors or Methodology Committee.