Methods and Applications of Sample Size Calculation and Recalculation in Clinical Trials

Front Matter ....Pages i-xiv
Front Matter ....Pages 1-1
 Introduction  (Meinhard Kieser)....Pages 3-10
 Statistical Test and Sample Size Calculation  (Meinhard Kieser)....Pages 11-16
Front Matter ....Pages 17-17
 Comparison of Two Groups for Normally Distributed Outcomes and Test for Difference or Superiority  (Meinhard Kieser)....Pages 19-36
 Comparison of Two Groups for Continuous and Ordered Categorical Outcomes and Test for Difference or Superiority  (Meinhard Kieser)....Pages 37-45
 Comparison of Two Groups for Binary Outcomes and Test for Difference or Superiority  (Meinhard Kieser)....Pages 47-63
 Comparison of Two Groups for Time-to-Event Outcomes and Test for Difference or Superiority  (Meinhard Kieser)....Pages 65-77
 Comparison of More Than Two Groups and Test for Difference  (Meinhard Kieser)....Pages 79-89
 Comparison of Two Groups and Test for Non-Inferiority  (Meinhard Kieser)....Pages 91-112
 Comparison of Three Groups in the Gold Standard Non-Inferiority Design  (Meinhard Kieser)....Pages 113-124
 Comparison of Two Groups for Normally Distributed Outcomes and Test for Equivalence  (Meinhard Kieser)....Pages 125-132
 Multiple Comparisons  (Meinhard Kieser)....Pages 133-156
 Assessment of Safety  (Meinhard Kieser)....Pages 157-166
 Cluster-Randomized Trials  (Meinhard Kieser)....Pages 167-173
 Multi-Regional Trials  (Meinhard Kieser)....Pages 175-184
 Integrated Planning of Phase II/III Drug Development Programs  (Meinhard Kieser)....Pages 185-192
 Simulation-Based Sample Size Calculation  (Meinhard Kieser)....Pages 193-195
Front Matter ....Pages 197-197
 Basic Concepts of Sample Size Recalculation  (Meinhard Kieser)....Pages 199-200
Front Matter ....Pages 201-201
 Internal Pilot Study Designs  (Meinhard Kieser)....Pages 203-205
 A General Approach for Controlling the Type I Error Rate for Blinded Sample Size Recalculation  (Meinhard Kieser)....Pages 207-207
 Comparison of Two Groups for Normally Distributed Outcomes and Test for Difference or Superiority  (Meinhard Kieser)....Pages 209-224
 Comparison of Two Groups for Binary Outcomes and Test for Difference or Superiority  (Meinhard Kieser)....Pages 225-237
 Comparison of Two Groups for Normally Distributed Outcomes and Test for Non-Inferiority  (Meinhard Kieser)....Pages 239-246
 Comparison of Two Groups for Binary Outcomes and Test for Non-Inferiority  (Meinhard Kieser)....Pages 247-254
 Comparison of Two Groups for Normally Distributed Outcomes and Test for Equivalence  (Meinhard Kieser)....Pages 255-256
 Regulatory and Operational Aspects  (Meinhard Kieser)....Pages 257-258
 Concluding Remarks  (Meinhard Kieser)....Pages 259-260
Front Matter ....Pages 261-261
 Group Sequential and Adaptive Designs  (Meinhard Kieser)....Pages 263-269
 Sample Size Recalculation Based on Conditional Power  (Meinhard Kieser)....Pages 271-277
 Sample Size Recalculation Based on Optimization  (Meinhard Kieser)....Pages 279-287
 Regulatory and Operational Aspects  (Meinhard Kieser)....Pages 289-291
 Concluding Remarks  (Meinhard Kieser)....Pages 293-293
Back Matter ....Pages 295-396