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August 3, 2021JAMA144 citationsOpen Access

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

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VSValerie A. SmithCCCynthia J. CoffmanMHMichael G. Hudgens

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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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Beyond the intention-to-treat in comparative effectiveness research2011 · 541 citations
  2. 2Future Directions in Clinical Trials and Intention-To-Treat Analysis: Fulfilling Admirable Intentions Through the Right Questions2024 · 7 citations
  3. 3Intention-to-treat concept: A review2011 · 1,980 citations
  4. 4IMPROVING INFERENCES FROM RANDOMIZED TRIALS: USING PER-PROTOCOL ANALYSES TO ESTIMATE EFFECTS OF HIV TREATMENT2026
  5. 5Intention‐to‐treat analyses and missing outcome data: A tutorial2024