Statistics applied to clinical trials / Ton J. Cleophas, Aeilko H. Zwinderman, and Toine F. Cleophas.

Format:

Book

Author/Creator:

Cleophas, Ton J. M.

Contributor:

Zwinderman, Aeilko H.

Cleophas, Toine F.

Frances Houston Medical Book Fund.

Language:

English

Subjects (All):

Drugs--Testing--Statistical methods.

Drugs.

Clinical trials--Statistical methods.

Clinical trials.

Pharmaceutical Preparations.

Clinical Trials as Topic.

Data Interpretation, Statistical.

Drugs--Testing.

Medical Subjects:

Pharmaceutical Preparations.

Clinical Trials as Topic.

Data Interpretation, Statistical.

Physical Description:

ix, 210 pages : illustrations ; 25 cm

Edition:

Second edition.

Place of Publication:

Boston : Kluwer Academic Publishers, [2002]

Summary:

This book not only explains classical statistical analyses of clinical trials, but addresses relatively novel issues, including equivalence testing, interim analyses, sequential analyses, and meta-analyses, and provides a framework of the best statistical methods currently available for such purposes. The book is not only useful for investigators involved in the field of clinical trials, but also for all physicians who wish to better understand the data of trials as currently published.

Contents:

Chapter 1 Hypotheses, Data, Stratification

2. Two main hypotheses in drug trials: efficacy and safety 2

3. Different types of data: continuous data 3

4. Different types of data: proportions, percentages and contingency tables 8

5. Different types of data: correlation coefficient 11

6. Stratification issues 13

7. Randomized versus historical controls 14

8. Factorial designs 15

Chapter 2 The Analysis of Efficacy Data of Drug Trials

2. The principle of testing statistical significance 18

3. Unpaired T-Test 21

4. Null hypothesis testing of 3 or more unpaired samples 23

5. Three methods to test statistically a paired sample 24

6. Null-hypothesis testing of 3 or more paired samples 28

7. Paired data with a negative correlation 29

8. Rank testing 35

Chapter 3 The Analysis of Safety Data of Drug Trials

1. Introduction, summary display 39

2. Four methods to analyze two unpaired proportions 40

3. Chi-square to analyze more than two unpaired proportions 42

4. McNemar's test for paired proportions 43

5. Survival analysis 44

Chapter 4 Equivalence Testing

2. Overview of possibilities with equivalence testing 49

3. Equivalence testing, a new gold standard? 51

4. Validity of equivalence trials 51

Chapter 5 Statistical Power and Sample Size

1. What is statistical power 53

2. Emphasis on statistical power rather than null-hypothesis testing 54

3. Power computations 56

4. Example of power computation using the T-Table 57

5. Calculation of required sample size, rationale 59

6. Calculations of required sample size, methods 59

7. Testing not only superiority but also inferiority of a new treatment (type III error) 62

Chapter 6 Interim Analyses

2. Monitoring 65

3. Interim analysis 66

4. Group-sequential design of interim analysis 69

5. Continuous sequential statistical techniques 69

Chapter 7 Multiple Statistical Inferences

2. Multiple comparisons 73

3. Primary and secondary variables 78

Chapter 8 Principles of Linear Regression

2 More on paired observations 84

3 Using statistical software for simple linear regression 87

4 Multiple linear regression 89

5 Another real data example of multiple linear regression 93

Chapter 9 Subgroup Analysis Using Multiple Linear Regression: Confounding, Interaction, Synergism

3 Model 96

4 (I.) Increased precision of efficacy 98

5 (II.) Confounding 99

6 (III.) Interaction and synergism 100

7 Estimation, and hypothesis testing 101

8 Goodness-of-fit 102

9 Selection procedures 103

Chapter 10 Curvilinear Regression

2. An example: curvilinear regression analysis of ambulatory blood pressure measurements 106

3. Methods, statistical model 106

4. Results 108

Chapter 11 Meta-Analysis

3. Clearly defined hypotheses 121

4. Thorough search of trials 121

5. Strict inclusion criteria 122

6. Uniform data analysis 122

7. Discussion, where are we now? 131

Chapter 12 Crossover Studies with Continuous Variables: Power Analysis

3. Mathematical model 134

4. Hypothesis testing 135

5. Statistical power of testing 137

Chapter 13 Crossover Studies with Binary Responses

3. Assessment of carryover and treatment effect 144

4. Statistical model for testing treatment and carryover effects 145

5. Results 146

Chapter 14 Post-Hoc Analysis in Clinical Trials, a Case for Logistic Regression Analysis

1. Multivariate methods 151

3. Logistic regression equation 154

Chapter 15 Quality-of-Life Assessments in Clinical Trials

3. Some terminology 158

4. Defining QOL in a subjective or objective way 160

5. The patients' opinion is an important independent-contributor to QOL 160

6. Lack of sensitivity of QOL-assessments 162

7. Odds ratio analysis of effects of patient characteristics on QOL data provides increased precision 162

Chapter 16 Statistics for the Analysis of Genetic Data

2. Some terminology 168

3. Genetics, genomics, proteonomics, data mining 170

4. Genomics 171

Chapter 17 Relationship Among Statistical Distributions

3. Variances 178

4. The normal distribution 179

5. Null-hypothesis testing with the normal or the t-distribution 181

6. Relationship between the normal distribution and chi-square distribution, null-hypothesis testing with the chi-square distribution 183

7. Examples of data where variance is more important than mean 185

8. Chi-square can be used for multiple samples of data 186

Chapter 18 Statistics is Not "Bloodless" Algebra

2. Statistics is fun because it proves your hypothesis was right 191

3. Statistical principles can help to improve the quality of the trial 192

4. Statistics can provide worthwhile extras to your research 192

5. Statistics is not like algebra bloodless 193

6. Statistics can turn art into science 194

7. Statistics for support rather than illumination? 194

8. Statistics can help the clinician to better understand limitations and benefits of current research 195

9. Limitations of statistics 195.

Notes:

Includes bibliographical references and index.

Local Notes:

Acquired for the Penn Libraries with assistance from the Frances Houston Medical Book Fund.

ISBN:

1402005695

1402005709

OCLC:

49395985