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Table 4 Recommendations for incorporating clinical significance into randomized controlled trial design and interpretation

From: Clinical significance in pediatric oncology randomized controlled treatment trials: a systematic review

Recommendationsa
1. Conduct a comprehensive review of the literature to identify the MCID. If the RCT is completely novel, use preliminary pilot data to inform the MCID
2. Perform a sample size calculation using a delta value that is based on the MCID. If the sample size is not feasible given resource constraints, adjust the delta value to increase the sample size to a value that is still clinically meaningful
3. When reporting the results of an RCT ensure the following are reported in the sample size calculation:
• Type 1 error (α value)
One- or two-sided p value
• Type 2 error (β value)
At least 80% is recommended
• Estimated controlled value and justification
• Estimated experimental value and justification
• Delta value in absolute terms and justification of treatment effect
• Explicitly identify primary outcome when multiple outcomes are being investigated
4. Calculate and report confidence intervals for the experimental and controlled values as well as the treatment effect
5. Interpret the treatment effect and its confidence interval in relation to the MCID and place weight on conclusions based on the precision determined by the confidence interval
6. Ensure conclusions reflect the quality of the trial based on the recommendations of the CONSORT Statement
  1. MCID minimally clinically important difference, RCT randomized control trial, CONSORT Consolidated Standards of Reporting Trials
  2. aRecommendations adapted from and informed by Cook et al. [24], Moher et al. [11], and Koynova et al. [25]