Table 2 Description of the simulation models used to mimic DDP segments
Parameter | Example 1 Phase II trial followed by a phase III trial | Example 2 Phase I dose-finding trial followed by a phase II trial | Example 3 Phase II enrichment trial followed by a phase III RCT in the selected subpopulation |
|---|---|---|---|
Sample size of the first trial | 20–140 patients | 10–80 patients | 20–140 patients |
SOC ORR | 0.40 | 0.40 | 0.40 |
Hypothetical treatment effect used to choose the sample size for the second trial | Estimated from the phase II trial | 0.20 | Estimated from the phase II trial |
Type I error rate of the second trial | 0.05 | 0.05 | 0.05 |
Targeted power of the second trial | 0.90 | 0.80 | 0.90 |
Sample size of the second trial | Computed using the results of the phase II trial. Maximum size: 400 patients | Calculated to achieve the desired power. Trial size fixed: 42 patients | Computed using the results of the phase II trial. Maximum size: 400 patients |