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Table 4 Simulation results for dropping treatment arms based on the second rule (R2) and the absolute bias for such arms in the estimated treatment effect at the time of dropping decision – all priors on pk (k=A,B,C) are non-informative beta(1,1) priors, when decision threshold is set at 0.05

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

Average sample sizes

% Early stopping

n

dB

dC

pA

pB

pC

A

B

C

B

C

40

−0.20

0.00

0.0446

0.0410

−0.0036

34.7688

19.0005

34.1757

83.18 %

20.90 %

 

−0.15

0.00

0.0407

0.0196

−0.0049

35.4230

23.9111

34.1465

65.96 %

21.12 %

 

−0.10

0.00

0.0384

0.0076

−0.0045

36.0178

28.2528

33.9857

47.31 %

21.51 %

 

−0.05

0.00

0.0325

0.0004

−0.0039

36.8988

31.7086

34.2757

31.84 %

20.67 %

 

0.00

0.00

0.0289

−0.0046

−0.0034

37.5750

34.3312

34.2628

20.68 %

20.55 %

 

0.05

0.00

0.0262

−0.0053

−0.0046

38.1404

35.9159

34.2368

13.61 %

21.01 %

 

0.10

0.00

0.0212

−0.0068

−0.0038

38.6097

37.1804

34.2661

8.79 %

20.81 %

 

0.15

0.00

0.0184

−0.0079

−0.0043

39.0075

38.0515

34.3098

5.79

20.69 %

 

0.20

0.00

0.0163

−0.0098

−0.0051

39.2281

38.5864

34.2477

4.01 %

20.85 %

100

−0.20

0.00

0.0458

0.0362

−0.0144

80.7525

22.0750

80.0766

99.05 %

26.36 %

 

−0.15

0.00

0.0427

0.0103

−0.0153

81.9307

34.6011

79.9595

91.50 %

26.58 %

 

−0.10

0.00

0.0418

−0.0063

−0.0152

83.5055

51.2404

79.0897

71.48 %

27.30 %

 

−0.05

0.00

0.0344

−0.0142

−0.0153

87.6493

67.8997

80.1142

46.17 %

26.49 %

 

0.00

0.00

0.0277

−0.0161

−0.0157

90.8189

79.7603

79.7672

26.62 %

26.82 %

 

0.05

0.00

0.0247

−0.0149

−0.0154

93.1423

86.5031

79.4970

15.96 %

26.65 %

 

0.10

0.00

0.0202

−0.0130

−0.0157

95.3857

91.6455

78.7749

9.26 %

27.54 %

 

0.15

0.00

0.0154

−0.0119

−0.0155

96.8064

94.2754

79.4074

6.15 %

27.04 %

 

0.20

0.00

0.0112

−0.0100

−0.0147

97.8693

96.1853

80.1669

4.03 %

26.14 %

  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)