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Table 3 The impact of mis-specifying p% on the design and analysis. At the design stage, the sample size required to achieve the target power is calculated using the conventional design ignoring response dichotomy and the p-embedded design recognizing response dichotomy but mis-specifying the true p% to be pm%. Given the sample size calculated, the empirical power (EP) is evaluated under the true response dichotomy p%. Target power is 80%. Lag duration is 1 month. Hazard ratio for responding patients is 0.3. Total study duration is 5 years

From: Treating non-responders: pitfalls and implications for cancer immunotherapy trial design

P-embedded design

Conventional design

Design

Analysis

Design

Analysis

Mis-specifying p*

Over-specifying p* = pm − 10

Under-specifying p* = pm + 10

Ignoring p*

pm (%)

N

p* (%)

EP

p* (%)

EP

N

p* (%)

EP

p* (%)

EP

20

269

10

44.90%

30

95.20%

27

10

6.61%

30

13.39%

30

137

20

59.30%

40

88.90%

20

8.81%

40

18.58%

40

89

30

63.70%

50

89.20%

30

13.39%

50

25.73%

50

68

40

70.30%

60

89.00%

40

18.58%

60

32.81%

60

52

50

71.40%

70

88.00%

50

25.73%

70

40.93%