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August 3, 2021JAMA145 citationsOpen Access

Interpreting the Results of Intention-to-Treat, Per-Protocol, and As-Treated Analyses of Clinical Trials

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VSValerie A. SmithUniversity College DublinCCCynthia J. CoffmanDuke UniversityMHMichael G. HudgensUniversity of North Carolina at Chapel Hill

Key Points

  • The aim is to explain how different analytical approaches affect the understanding of treatment effects in clinical trials.
  • Descriptive overview of intention-to-treat, per-protocol, and as-treated analysis methods.
  • Discussion on patient population variations across different analytical approaches.
  • Examination of implications for treatment effect interpretation.
  • Clarification of how treatment assignment impacts interpretation of clinical trial results.
  • Insight into choosing the appropriate analysis method for accurate outcome assessment.

Abstract

This JAMA Guide to Statistics and Methods describes how intention-to-treat, per-protocol, and as-treated approaches to analysis differ with regard to the patient population and treatment assignments and their implications for interpretation of treatment effects in clinical trials.

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Cite This Study

Smith et al. (2021) studied this question.

synapsesocial.com/papers/69dbe3e7f7e0c66ced836c4ahttps://doi.org/10.1001/jama.2021.2825
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