Table 3.
Operating characteristics of the various approaches for selecting initial design for the oncology trial using two-stage Bayesian CRM. The bolded columns represent the true maximum tolerated doses.
| Method | Initial design | Proportion of recommendation | N̄ | DLT Proportion | |||||
|---|---|---|---|---|---|---|---|---|---|
| Dose level | MTD below level 1 | 1 | 2 | 3 | 4 | 5 | |||
| pr(DLT) | 0.25 | 0.35 | 0.50 | 0.65 | 0.80 | ||||
| 3+3 Design | 0.44 | 0.36 | 0.18 | 0.02 | 0.00 | 0.00 | 9 | 0.40 | |
| General | (1,1,2,2,18) | 0.06 | 0.64 | 0.26 | 0.04 | 0.00 | 0.00 | 24 | 0.36 |
| Tailored | (2,2,2,2,16) | 0.06 | 0.60 | 0.29 | 0.05 | 0.00 | 0.00 | 24 | 0.35 |
| Fixed design 1 | (1,1,1,1,20) | 0.06 | 0.54 | 0.35 | 0.05 | 0.00 | 0.00 | 24 | 0.37 |
| Fixed design 2 | (3,3,3,3,12) | 0.06 | 0.54 | 0.35 | 0.04 | 0.00 | 0.00 | 24 | 0.35 |
|
| |||||||||
| pr(DLT) | 0.15 | 0.25 | 0.40 | 0.55 | 0.70 | ||||
| 3+3 Design | 0.21 | 0.34 | 0.33 | 0.11 | 0.01 | 0.00 | 11 | 0.30 | |
| General | (1,1,2,2,18) | 0.02 | 0.27 | 0.48 | 0.21 | 0.02 | 0.00 | 24 | 0.29 |
| Tailored | (2,2,2,2,16) | 0.02 | 0.22 | 0.52 | 0.22 | 0.02 | 0.00 | 24 | 0.28 |
| Fixed design 1 | (1,1,1,1,20) | 0.02 | 0.18 | 0.57 | 0.22 | 0.01 | 0.00 | 24 | 0.30 |
| Fixed design 3 | (3,3,3,3,12) | 0.02 | 0.19 | 0.56 | 0.21 | 0.01 | 0.00 | 24 | 0.27 |
|
| |||||||||
| pr(DLT) | 0.10 | 0.15 | 0.25 | 0.40 | 0.55 | ||||
| 3+3 Design | 0.10 | 0.19 | 0.30 | 0.30 | 0.10 | 0.02 | 14 | 0.23 | |
| General | (1,1,2,2,18) | 0.01 | 0.04 | 0.27 | 0.46 | 0.21 | 0.02 | 24 | 0.25 |
| Tailored | (2,2,2,2,16) | 0.01 | 0.03 | 0.27 | 0.47 | 0.20 | 0.02 | 24 | 0.24 |
| Fixed design 1 | (1,1,1,1,20) | 0.01 | 0.02 | 0.25 | 0.54 | 0.17 | 0.01 | 24 | 0.25 |
| Fixed design 2 | (3,3,3,3,12) | 0.01 | 0.02 | 0.28 | 0.52 | 0.16 | 0.01 | 24 | 0.22 |
|
| |||||||||
| pr(DLT) | 0.03 | 0.07 | 0.15 | 0.25 | 0.40 | ||||
| 3+3 Design | 0.01 | 0.06 | 0.17 | 0.36 | 0.29 | 0.10 | 18 | 0.16 | |
| General | (1,1,2,2,18) | 0.00 | 0.00 | 0.03 | 0.27 | 0.47 | 0.23 | 24 | 0.22 |
| Tailored | (2,2,2,2,16) | 0.00 | 0.00 | 0.03 | 0.28 | 0.48 | 0.21 | 24 | 0.20 |
| Fixed design 1 | (1,1,1,1,20) | 0.00 | 0.00 | 0.03 | 0.34 | 0.51 | 0.13 | 24 | 0.21 |
| Fixed design 2 | (3,3,3,3,12) | 0.00 | 0.00 | 0.03 | 0.37 | 0.47 | 0.14 | 24 | 0.17 |
|
| |||||||||
| pr(DLT) | 0.01 | 0.03 | 0.07 | 0.15 | 0.25 | ||||
| 3+3 Design | 0.00 | 0.01 | 0.05 | 0.17 | 0.32 | 0.46 | 19 | 0.11 | |
| General | (1,1,2,2,18) | 0.00 | 0.00 | 0.00 | 0.04 | 0.27 | 0.69 | 24 | 0.17 |
| Tailored | (2,2,2,2,16) | 0.00 | 0.00 | 0.00 | 0.04 | 0.31 | 0.65 | 24 | 0.15 |
| Fixed design 1 | (1,1,1,1,20) | 0.00 | 0.00 | 0.00 | 0.06 | 0.39 | 0.56 | 24 | 0.17 |
| Fixed design 2 | (3,3,3,3,12) | 0.00 | 0.00 | 0.00 | 0.07 | 0.42 | 0.51 | 24 | 0.12 |