Table 5.
Power figures for the CLL trial based on the CAT-BUB design, the beta-binomial design with an efficacy test only (BB-EO), and the hierarchical beta-binomial design with an efficacy test followed by a toxicity test (BB-ET). Comparisons of θFC,E vs θF,E and θFC,T vs θF,T are characterized as being “equivalent” (=), or having “moderate” (>), “large” (≫), or “very large” (≫>) differences.
| Scenario | Probability of Final Conclusion | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| Beta-Binomial Designs | |||||||||
| Efficacy | Toxicity | CAT-BUB Design | Efficacy Only | Efficacy then Toxicity | |||||
| FC vs F | FC vs F | θU,FC–F | FC > F | F > FC | FC > F | F > FC | FC > F | F > FC | |
| 1.0: | = | = | 0.0 | 0.025 | 0.025 | 0.026 | 0.024 | 0.024 | 0.025 |
| 1.1: | = | > | −5.2 | 0.001 | 0.222 | 0.019 | 0.035 | 0.007 | 0.388 |
| 1.2: | = | ≫ | −11.9 | 0.000 | 0.782 | 0.012 | 0.047 | 0.005 | 0.982 |
|
| |||||||||
| 2.0: | > | = | 6.8 | 0.352 | 0.000 | 0.402 | 0.000 | 0.289 | 0.010 |
| 2.1: | > | > | 1.1 | 0.041 | 0.012 | 0.331 | 0.000 | 0.226 | 0.308 |
| 2.2: | > | ≫ | −6.5 | 0.000 | 0.314 | 0.278 | 0.000 | 0.173 | 0.818 |
|
| |||||||||
| 3.0: | ≫ | = | 13.5 | 0.903 | 0.000 | 0.910 | 0.000 | 0.846 | 0.002 |
| 3.1: | ≫ | > | 7.3 | 0.397 | 0.000 | 0.873 | 0.000 | 0.778 | 0.097 |
| 3.2: | ≫ | ≫ | −1.1 | 0.041 | 0.015 | 0.816 | 0.000 | 0.716 | 0.281 |
|
| |||||||||
| 4.0: | ≫> | = | 21.0 | 1.000 | 0.000 | 1.000 | 0.000 | 1.000 | 0.000 |
| 4.1: | ≫> | > | 14.2 | 0.917 | 0.000 | 1.000 | 0.000 | 0.999 | 0.001 |
| 4.2: | ≫> | ≫ | 5.0 | 0.201 | 0.001 | 0.999 | 0.000 | 0.997 | 0.003 |