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A three-outcome design for randomized comparative phase II clinical trials.
1Eli Lilly and Company, Indianapolis, IN 46285, USA. hongsh@lilly.com
Statistics in Medicine
|January 31, 2007
Summary
This study introduces a three-outcome design for phase II oncology trials, enabling formal statistical comparisons with reduced sample sizes. This approach accounts for uncertainty, aiding clinical judgment in randomized comparative studies.
Area of Science:
- Clinical Trials Methodology
- Biostatistics
- Oncology Research
Background:
- Randomized designs are recommended for phase II oncology trials to mitigate patient selection bias.
- Formal statistical comparisons in these trials are often limited by sample size constraints.
Purpose of the Study:
- To present a sample size reduction approach for randomized comparative phase II oncology trials.
- To extend the three-outcome design for single-arm trials to a comparative setting.
Main Methods:
- The study extends Sargent et al.'s three-outcome design to randomized comparative trials.
- It incorporates a third 'grey area' outcome for inconclusive hypothesis testing results.
- Statistical formulations for single-stage and two-stage trials are provided.
Main Results:
- The three-outcome design facilitates formal statistical comparison with smaller sample sizes compared to standard two-outcome designs.
- Sample sizes are tabulated for typical clinical scenarios.
Conclusions:
- The proposed three-outcome design offers a statistically valid method for sample size reduction in phase II oncology trials.
- It balances statistical rigor with the need for clinical judgment in decision-making.
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