Sample Size and Power

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Phase II: Two-Stage Optimal Design • Seek to rule out undesirably low response probability – E.g. only 20% respond (p0=0.20) • Seek to rule out p0 in favor of p1; shows “useful” activity – E.g. 40% are stable (p1=0.40)
Phase II Example: Two-Stage Optimal Design • Single arm, two stage, using an optimal design & predefined response • Rule out response probability of 20% (H 0 : p=0.20) • Level that demonstrates useful activity i is 40% (H 1 :p=0.40) • α = 0.10, β = 0.10
Page 1 and 2: Sample Size and Power Laura Lee Joh
Page 3 and 4: Misconceptions • A small p-value
Page 5 and 6: Take Away Message • Get some inpu
Page 7 and 8: Superiority vs. Equivalence Superio
Page 9 and 10: Vocabulary (2) • Follow-up period
Page 11 and 12: Power Depends on Sample Size • Po
Page 13 and 14: Power Changes • 2n = 32, 2 sample
Page 15 and 16: Power should be….? • Phase III:
Page 17 and 18: Outline Power ‣Basic Sample Size
Page 19 and 20: Basic Sample Size Information • W
Page 21 and 22: Take Away • Study’s primary out
Page 23 and 24: Sample Size Formula Information •
Page 25 and 26: Sample Size & Study Design • Rand
Page 27 and 28: Phase I: Dose Escalation • Dose l
Page 29 and 30: Phase I (cont.) • Maximum Tolerat
Page 31 and 32: Phase I Enroll 3 people 0/3 DLT 1/3
Page 37 and 38: Number of pts with DLT Decision 0/3
Page 39 and 40: Phase I • MANY new methods • Se
Page 41: Phase II Design Problems • Might
Page 45 and 46: Table from Simon (1989)
Page 47 and 48: Phase II Example • Initially enro
Page 49 and 50: Sample Size Differences • If the
Page 51 and 52: Phase II: Historical Controls • W
Page 53 and 54: Study Design Phase II Summary Advan
Page 55: Number of Patients to Enroll? • 1
Page 58: Phase III Survival • Look at nomo
Page 61 and 62: 1-Sample N Example • Study effect
Page 63 and 64: Short Helpful Hints • In humans n
Page 65 and 66: Sample Size: Iteration and the Use
Page 67 and 68: Sample Size: Change Standard d Devi
Page 69 and 70: Sleep Aid Example: 2 Arms Investiga
Page 71 and 72: Sample Size: Change Effect or Diffe
Page 73 and 74: Sample Size: Change Standard d Devi
Page 75 and 76: Live Statistical Consult! • Sampl
Page 77 and 78: Sample Size Rulers
Page 79 and 80: Put in 1-Sample Example #s • 1 ar
Page 81 and 82: Put in 2-Sample Example #s • 2 ar
Page 83 and 84: Other Designs?
Page 85 and 86: Examples in the Text • Several wi
Page 87 and 88: Take Home: What you need for N •
Page 89 and 90: Outline Power Basic sample size inf
Page 91 and 92: K:1 Sample Size Shortcut • Use eq
Page 93 and 94:
How many controls? k = 2n n 0 − n
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Formulas and Example Risk of event
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Balanced Designs: Easier to Find Po
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Multiple Comparisons • If you hav
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Outline Power Basic Sample Size Inf
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No Prior Information • "Sample si
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Number of Available Patients • "T
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Conclusions: What Impacts Sample Si
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Top 10 Statistics Questions 10.Exac
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Top 10 Statistics Questions 4. Inte
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Analysis Follows Design Questions
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Resources: General/Text Books • A
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Resources: Articles • Simon R. Op
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Resources: FDA Guidance • http://
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Questions?
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Sample Size and Power

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