Table 3.
Operating characteristics for five designs in testing the marker effect, treatment effect, and marker by treatment interaction (marker predictive effect). The statistical power and overall response rate are shown in five scenarios.
| Scenario | Design | Marker Effect | Treatment Effect | MK × Interaction** | Overall Resp. Rate | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| In TX1* | In TX2 | Overall | In MK(−) | In MK(+) | Overall | In STR(−) | In STR(+) | STR(−) vs STR(+) | ||||
| 1 | Simple Rand. | 0.04 | 0.02 | 0.06 | 0.04 | 0.02 | 0.06 | 0.05 | 0.20 | |||
| Marker Stratified | 0.04 | 0.02 | 0.06 | 0.03 | 0.02 | 0.06 | 0.05 | 0.20 | ||||
| Marker Strategy | 0.03 | 0.00 | 0.03 | 0.03 | 0.00 | 0.03 | 0.05 | 0.05 | 0.05 | 0.03 | 0.20 | |
| Efficient Target | 0.04 | 0.20 | ||||||||||
| Bayesian AR | 0.03 | 0.02 | 0.05 | 0.02 | 0.04 | 0.06 | 0.03 | 0.20 | ||||
| 2 | Simple Rand. | 0.46 | 0.45 | 0.71 | 0.04 | 0.02 | 0.06 | 0.05 | 0.30 | |||
| Marker Stratified | 0.47 | 0.47 | 0.72 | 0.03 | 0.03 | 0.06 | 0.05 | 0.30 | ||||
| Marker Strategy | 0.40 | 0.28 | 0.56 | 0.03 | 0.02 | 0.04 | 0.06 | 0.46 | 0.05 | 0.04 | 0.30 | |
| Efficient Target | 0.05 | 0.05 | 0.40 | |||||||||
| Bayesian AR | 0.46 | 0.43 | 0.71 | 0.02 | 0.03 | 0.05 | 0.04 | 0.30 | ||||
| 3 | Simple Rand. | 0.04 | 0.02 | 0.06 | 0.46 | 0.45 | 0.70 | 0.05 | 0.30 | |||
| Marker Stratified | 0.04 | 0.02 | 0.06 | 0.46 | 0.47 | 0.72 | 0.04 | 0.30 | ||||
| Marker Strategy | 0.03 | 0.02 | 0.05 | 0.38 | 0.25 | 0.54 | 0.46 | 0.46 | 0.05 | 0.04 | 0.30 | |
| Efficient Target | 0.47 | 0.30 | ||||||||||
| Bayesian AR | 0.02 | 0.03 | 0.05 | 0.36 | 0.38 | 0.62 | 0.02 | 0.34 | ||||
| 4 | Simple Rand. | 0.04 | 0.95 | 0.95 | 0.04 | 0.95 | 0.95 | 0.64 | 0.30 | |||
| Marker Stratified | 0.04 | 0.95 | 0.95 | 0.03 | 0.94 | 0.95 | 0.62 | 0.30 | ||||
| Marker Strategy | 0.03 | 0.86 | 0.86 | 0.03 | 0.84 | 0.84 | 0.46 | 0.95 | 0.25 | 0.46 | 0.35 | |
| Efficient Target | 0.95 | 0.40 | ||||||||||
| Bayesian AR | 0.02 | 0.98 | 0.98 | 0.04 | 0.87 | 0.87 | 0.50 | 0.35 | ||||
| 5 | Simple Rand. | 0.54 | 0.95 | 0.98 | 0.15 | 0.75 | 0.79 | 0.08 | 0.30 | |||
| Marker Stratified | 0.54 | 0.95 | 0.98 | 0.15 | 0.74 | 0.78 | 0.08 | 0.30 | ||||
| Marker Strategy | 0.52 | 0.86 | 0.93 | 0.19 | 0.60 | 0.66 | 0.46 | 1.00 | 0.10 | 0.05 | 0.33 | |
| Efficient Target | 0.75 | 0.45 | ||||||||||
| Bayesian AR | 0.40 | 0.98 | 0.99 | 0.19 | 0.66 | 0.71 | 0.06 | 0.34 | ||||
: Testing whether marker is prognostic.
: Testing whether marker is predictive.
TX: Treatment; MK: Marker: STR: Strategy; Overall Resp. Rate: overall response rate.