Sample Size Calculations in Clinical Research

by Chow, Shein-Chung; Shao, Jun; Wang, Hansheng

• ISBN13: 9780824709709
• ISBN10: 0824709705
• Edition: 2nd
• Format: Hardcover
• Copyright: 2003-03-04
• Publisher: CRC

Summary

This text enforces statistical design procedures that ensure the validity, accuracy, reliability, and reproducibility of clinical studies. It illustrates formulas for sample size calculation with selected examples from studies on women's health, oncology, in vivo and in vitro bioequivalence, and the cardiovascular and central nervous systems. Topics of discussion include regulatory requirement, considerations prior to sample size calculation, confounding and interaction, crossover design versus parallel design, subgroup/interim analyses, Fisher's exact test, tests for goodness-of-fit and contingency tables, Cox's proportional hazards model, and comparing variabilities.

Table of Contents

Series Introduction	iii
Preface	v
Chapter 1. Introduction	1	(24)
1.1 Regulatory Requirement	2	(5)
1.2 Basic Considerations	7	(7)
1.3 Procedures for Sample Size Calculation	14	(7)
1.4 Aims and Structure of the Book	21	(4)
Chapter 2. Considerations Prior to Sample Size Calculation	25	(22)
2.1 Confounding and Interaction	26	(2)
2.2 One-Sided Test Versus Two-Sided Test	28	(2)
2.3 Crossover Design Versus Parallel Design	30	(2)
2.4 Subgroup/Interim Analyses	32	(4)
2.5 Data Transformation	36	(2)
2.6 Practical Issues	38	(9)
Chapter 3. Comparing Means	47	(34)
3.1 One-Sample Design	48	(7)
3.2 Two-Sample Parallel Design	55	(8)
3.3 Two-Sample Crossover Design	63	(5)
3.4 Multiple-Sample One-Way ANOVA	68	(4)
3.5 Multiple-Sample Williams Design	72	(4)
3.6 Practical Issues	76	(5)
Chapter 4. Large Sample Tests for Proportions	81	(34)
4.1 One-Sample Design	82	(5)
4.2 Two-Sample Parallel Design	87	(6)
4.3 Two-Sample Crossover Design	93	(4)
4.4 One-Way Analysis of Variance	97	(2)
4.5 Williams Design	99	(3)
4.6 Relative Risk - Parallel Design	102	(5)
4.7 Relative Risk - Crossover Design	107	(2)
4.8 Practical Issues	109	(6)
Chapter 5. Exact Tests for Proportions	115	(28)
5.1 Binomial Test	115	(4)
5.2 Fisher's Exact Test	119	(3)
5.3 Optimal Multiple-Stage Designs for Single Arm Trials	122	(17)
5.4 Flexible Designs for Multiple-Arm Trials	139	(2)
5.5 Remarks	141	(2)
Chapter 6. Tests for Goodness-of-Fit and Contingency Tables	143	(18)
6.1 Tests for Goodness-of-Fit	144	(2)
6.2 Test for Independence - Single Stratum	146	(3)
6.3 Test for Independence - Multiple Strata	149	(2)
6.4 Test for Categorical Shift	151	(5)
6.5 Carry-Over Effect Test	156	(3)
6.6 Practical Issues	159	(2)
Chapter 7. Comparing Time-to-Event Data	161	(24)
7.1 Basic Concepts	162	(2)
7.2 Exponential Model	164	(8)
7.3 Cox's Proportional Hazards Model	172	(5)
7.4 Weighted Log-Rank Test	177	(6)
7.5 Practical Issues	183	(2)
Chapter 8. Group Sequential Methods	185	(28)
8.1 Pocock's Test	186	(4)
8.2 O'Brien and Fleming's Test	190	(1)
8.3 Wang and Tsiatis' Test	191	(4)
8.4 Inner Wedge Test	195	(4)
8.5 Binary Variables	199	(1)
8.6 Time-to-Event Data	200	(2)
8.7 Alpha Spending Function	202	(2)
8.8 Sample Size Re-Estimation	204	(3)
8.9 Conditional Power	207	(2)
8.10 Practical Issues	209	(4)
Chapter 9. Comparing Variabilities	213	(42)
9.1 Comparing Intra-Subject Variabilities	214	(8)
9.2 Comparing Intra-Subject CVs	222	(9)
9.3 Comparing Inter-Subject Variabilities	231	(8)
9.4 Comparing Total Variabilities	239	(13)
9.5 Practical Issues	252	(3)
Chapter 10. Bioequivalence Testing	255	(22)
10.1 Bioequivalence Criteria	256	(1)
10.2 Average Bioequivalence	257	(4)
10.3 Population Bioequivalence	261	(2)
10.4 Individual Bioequivalence	263	(6)
10.5 in Vitro Bioequivalence	269	(8)
Chapter 11. Nonparametrics	277	(16)
11.1 Violation of Assumptions	278	(1)
11.2 One-Sample Location Problem	279	(4)
11.3 Two-Sample Location Problem	283	(4)
11.4 Test for Independence	287	(3)
11.5 Practical Issues	290	(3)
Chapter 12. Sample Size Calculation in Other Areas	293	(36)
12.1 Dose Response Studies	294	(8)
12.2 ANOVA with Repeated Measures	302	(6)
12.3 Quality of Life	308	(3)
12.4 Bridging Studies	311	(10)
12.5 Vaccine Clinical Trials	321	(8)
Appendix: Tables of Quantiles	329	(10)
References	339	(16)
Index	355