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. Author manuscript; available in PMC: 2019 Nov 18.
Published in final edited form as: J Am Stat Assoc. 2018 Jun 28;113(523):1016–1027. doi: 10.1080/01621459.2017.1383260

Table 2:

True immune response, toxicity, efficacy probabilities, and utility at each dose, and selection percentage and the average number of patients treated at each dose level under the proposed design and the EffTox design. The boldface numbers are the target doses.

dose level

1 2 3 4 5

Scenario 1
E(YI ) 2.0 2.9 4.5 5.7 6.3
πT 0.03 0.04 0.1 0.17 0.23
E,1, πE,2) (0.46, 0.11) (0.50, 0.13) (0.35, 0.07) (0.16, 0.02) (0.08, 0.01)
Utility 30.9 34.6 26.2 13.1 8
Selection % (proposed) 0.282 0.649 0.069 0 0
# of patients 16.0 16.9 13.9 9.0 4.8
Selection % (EffTox) 0.476 0.521 0.001 0 0.002
# of patients 17.4 16.3 13.1 8.2 4.4
Scenario 2
E(YI) 2.0 2.9 4.5 5.7 6.3
πT 0.03 0.07 0.19 0.35 0.49
E,1, πE,2) (0.28, 0.14) (0.31, 0.17) (0.31, 0.18) (0.28, 0.14) (0.25, 0.11)
Utility 24.2 27.8 30.6 23.8 17.7
Selection % (proposed) 0.117 0.094 0.736 0.047 0.006
# of patients 13.0 14.0 14.6 10.9 7.4
Selection % (EffTox) 0.212 0.602 0.183 0.001 0.002
# of patients 14.2 15.2 14.4 10.1 6.2
Scenario 3
E(YI ) 2.0 2.9 4.5 5.7 6.3
πT 0.07 0.09 0.12 0.3 0.49
E,1, πE,2) (0.16, 0.04) (0.17, 0.05) (0.22, 0.07) (0.25, 0.10) (0.27, 0.12)
Utility 9.8 11.9 19.7 22.1 20.1
Selection % (proposed) 0.028 0.005 0.29 0.572 0.057
# of patients 9.5 9.8 15.0 15.1 9.2
Selection % (EffTox) 0.143 0.103 0.254 0.255 0.203
# of patients 11.1 12.0 13.0 12.9 9.8
Scenario 4
E(YI ) 2.0 2.9 4.5 5.7 6.3
πT 0.16 0.16 0.16 0.16 0.16
E,1, πE,2) (0.21, 0.06) (0.26, 0.09) (0.34, 0.17) (0.36, 0.20) (0.37, 0.22)
Utility 12.0 17.7 32.8 40.2 42.3
Selection % (proposed) 0.000 0.000 0.006 0.257 0.735
# of patients 7.9 8.7 12.4 15.3 15.6
Selection % (EffTox) 0.008 0.004 0.046 0.207 0.733
# of patients 9.3 10.6 12.4 13.4 14.3
Scenario 5
E(YI ) 2.6 3.4 3.9 4.3 4.6
πT 0.05 0.12 0.2 0.28 0.36
E,1, πE,2) (0.15, 0.09) (0.22, 0.15) (0.24, 0.17) (0.23, 0.16) (0.21, 0.15)
Utility 13.8 21.9 24.5 23.7 20.6
Selection % (proposed) 0.024 0.036 0.431 0.42 0.077
# of patients 11.6 12.8 13.0 12.3 10.0
Selection % (EffTox) 0.002 0.258 0.711 0.017 0.002
# of patients 11.4 14.7 14.4 11.2 7.8
Scenario 6
E(YI ) 2.6 3.4 3.9 4.3 4.6
πT 0.01 0.04 0.09 0.14 0.2
E,1, πE,2) (0.24, 0.06) (0.31, 0.10) (0.34, 0.12) (0.35, 0.13) (0.36, 0.13)
Utility 17.3 24.9 28.3 29.6 29.4
Selection % (proposed) 0.016 0.001 0.098 0.401 0.483
# of patients 10.4 11.1 12.1 13.1 13.2
Selection % (EffTox) 0.024 0.094 0.43 0.321 0.129
# of patients 10.3 12.1 12.9 12.7 11.8
Scenario 7
E(YI ) 2.6 3.4 3.9 4.3 4.6
πT 0.16 0.16 0.16 0.16 0.16
E,1, πE,2) (0.16, 0.37) (0.16, 0.37) (0.16, 0.37) (0.16, 0.37) (0.16, 0.37)
Utility 32.5 34.3 36.9 38.5 39.7
Selection % (proposed) 0.175 0.021 0.038 0.091 0.675
# of patients 11.9 11.8 12.2 11.9 12.2
Selection % (EffTox) 0.351 0.086 0.146 0.07 0.347
# of patients 13.2 12.0 11.4 11.5 11.9
Scenario 8
E(YI ) 2.5 3.1 3.5 3.8 3.9
πT 0.72 0.78 0.84 0.88 0.91
E,1, πE,2) (0.06, 0.04) (0.08, 0.06) (0.09, 0.06) (0.09, 0.07) (0.10, 0.08)
Utility 2.4 3.5 4.1 4.7 5.2
Selection % (proposed) 0 0 0 0 0
# of patients 4.3 3.2 3.1 1.7 0.7
Selection % (EffTox) 0 0 0 0 0
# of patients 4.1 3.0 2.7 2.0 0.8

πE,1 = Pr(YE = 1), and πE,2 = Pr(YE = 2). 24