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Table 6 Simulation results evaluating Rule 2 when the threshold probability is set at 0.10

From: Evaluation of a multi-arm multi-stage Bayesian design for phase II drug selection trials – an example in hemato-oncology

Sample size

True benefit

Posterior mean estimate biases

Enrolled sample sizes

% Early stopping

 

dB

dC

pA

pB

pC

A

B

C

B

C

40

−0.20

0.00

0.0625

0.0585

−0.0123

30.6959

13.8404

29.9078

92.23 %

34.59 %

 

−0.15

0.00

0.0601

0.0311

−0.0120

31.2312

18.3044

29.5173

79.84 %

35.56 %

 

−0.10

0.00

0.0567

0.0107

−0.0119

32.3427

22.4957

29.6238

63.89 %

35.43 %

 

−0.05

0.00

0.0506

−0.0027

−0.0126

33.5772

26.5306

29.7194

48.34 %

35.68 %

 

0.00

0.00

0.0480

−0.0122

−0.0123

34.5352

29.3818

29.5852

35.89 %

35.74 %

 

0.05

0.00

0.0386

−0.0166

−0.0109

35.7946

32.2020

29.7191

25.14 %

35.03 %

 

0.10

0.00

0.0337

−0.0178

−0.0107

36.7805

34.2756

29.8086

17.75 %

34.52 %

 

0.15

0.00

0.0300

−0.0176

−0.0122

37.6091

35.9146

29.5542

12.22 %

35.64 %

 

0.20

0.00

0.0268

−0.0180

−0.0111

38.1120

36.8978

29.8112

8.92 %

34.91 %

100

−0.20

0.00

0.0704

0.0581

−0.0229

66.7855

15.4632

65.8818

99.60 %

42.83 %

 

−0.15

0.00

0.0673

0.0252

−0.0230

68.2876

23.4374

66.0635

96.28 %

42.43 %

 

−0.10

0.00

0.0601

0.0007

−0.0229

71.7312

36.8674

66.5112

84.36 %

42.43 %

 

−0.05

0.00

0.0571

−0.0160

−0.0231

75.3970

51.5977

65.8379

64.18 %

43.02 %

 

0.00

0.00

0.0467

−0.0237

−0.0240

81.2572

66.4872

65.8151

42.25 %

42.95 %

 

0.05

0.00

0.0379

−0.0255

−0.0233

86.5612

76.7592

66.9099

27.22 %

41.95 %

 

0.10

0.00

0.0338

−0.0241

−0.0242

89.8261

83.4433

64.7309

18.46 %

43.92 %

 

0.15

0.00

0.0279

−0.0229

−0.0232

92.3335

88.1690

66.3140

12.66 %

42.48 %

 

0.20

0.00

0.0234

−0.0208

−0.0245

94.1323

91.5214

65.5106

8.98 %

43.34 %

  1. pA, pB and pC refer to the posterior means of response probability in arms A, B and C, respectively; Δ=0. Bold data refer to the null hypothesis of absence of any treatment difference (dB=dC=0), or to the alternative hypothesis of an expected true 0.15 benefit of treatment B (dB=0.15)