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Using multiple outcomes in intervention studies: improving power while controlling type I errors
1Department of Experimental Psychology, University of Oxford, Oxford, Oxon, OX2 6GG, UK.
F1000Research
|November 9, 2023
Summary
Using multiple outcomes in clinical trials can improve efficiency. The Adjust NVar approach controls error rates, offering a better balance of power and type I error than single outcomes for studies with several correlated measures.
Area of Science:
- Biostatistics
- Clinical Trial Design
- Statistical Significance
Background:
- CONSORT guidelines recommend a single primary outcome to minimize false positives.
- Multiple outcomes can be used if the familywise error rate is controlled.
- Controlling error involves specifying a threshold (N) for significant outcomes based on their number and correlation.
Purpose of the Study:
- To explore an alternative to single primary outcomes in intervention studies.
- To develop a method for controlling familywise error rate with multiple outcomes.
- To assess the efficiency of using multiple correlated outcomes versus a single outcome.
Main Methods:
- Simulations used null-hypothesis significance testing with alpha = .05.
- Examined 2-12 outcome measures, correlations from 0 to .8, and effect sizes from 0 to .7.
- Developed the Adjust NVar approach, calculating minimum significant outcomes (MinNSig) to control the familywise error rate at 5%.
Main Results:
- The Adjust NVar approach demonstrated a more efficient trade-off between statistical power and type I error rate.
- This efficiency was observed when using three or more moderately intercorrelated outcome variables.
- Compared to single-outcome studies, Adjust NVar showed improved performance under specific conditions.
Conclusions:
- Employing a suite of moderately correlated outcome measures can be more efficient than a single primary outcome in intervention studies.
- This approach provides internal replication within a study.
- The Adjust NVar method can also be applied to evaluate existing intervention studies.
Keywords:
correlated outcomesfamilywise error rateinterventionmethodologymultiple comparisonspowerstatisticsMore Related Videos
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