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GROUP SEQUENTIAL METHODS with APPLICATIONS to LINICAL TRIALS
1 Introduction
1.1 About This Book
1.2 Why Sequential Methods
1.3 A Short History of Sequential and Group Sequential Methods
1.4 Some Examples
1.5 Chapter Organization: A Roadmap
1.6 Bibliography and Notes
2 Two-Sided Tests: Introduction
2.1 Two-Sided Tests for Comparing Two Treatments with Normal
Response of Known Variance
2.2 A Fixed Sample Test
2.3 Group Sequential Tests
2.4 Pocock’s Test
2.5 O’Brien & Fleming’s Test
2.6 Properties of Pocock and O’Brien & Fleming Tests
2.7 Other Tests
2.8 Conclusions
3 Two-Sided Tests: General Applications
3.1 A Unified Formulation
3.2 Applying the Tests with Equal Group Sizes
3.3 Applying the Tests with Unequal Increments in Information
3.4 Normal Linear Models
3.5 Other Parametric Models
3.6 Binary Data: Group Sequential Tests for Proportions
3.7 The Group Sequential Log-Rank Test for Survival Data
3.8 Group Sequential t-Tests
4 One-Sided Tests
4.1 Introduction
4.2 The Power Family of One-Sided Group Sequential Tests
4.3 Adapting Power Family Tests to Unequal Increments in
Information
4.4 Group Sequential One-Sided t-Tests
4.5 Whitehead’s Triangular Test
5 Two-Sided Tests with Early Stopping Under the Null Hypothesis
5.1 Introduction
5.2 The Power Family of Two-Sided Inner Wedge Tests
5.3 Whitehead’s Double Triangular Test
6 Equivalence Tests
6.1 Introduction
6.2 One-Sided Tests of Equivalence
6.3 Two-Sided Tests of Equivalence: Application to Comparative
Bioavailability Studies
6.4 Individual Bioequivalence: A One-Sided Test for Proportions
6.5 Bibliography and Notes
7 Flexible Monitoring: The Error Spending Approach
7.1 Unpredictable Information Sequences
7.2 Two-Sided Tests
7.3 One-Sided Tests
7.4 Data Dependent Timing of Analyses
7.5 Computations for Error Spending Tests
8 Analysis Following a Sequential Test
8.1 Introduction
8.2 Distribution Theory
8.3 Point Estimation
8.4 P-values
8.5 Confidence Intervals
9 Repeated Confidence Intervals
9.1 Introduction
9.2 Example: Difference of Normal Means
9.3 Derived Tests: Use of RCIs to Aid Early Stopping Decisions
9.4 Repeated P-values
9.5 Discussion
10 Stochastic Curtailment
10.1 Introduction
10.2 The Conditional Power Approach
10.3 The Predictive Power Approach
10.4 A Parameter-Free Approach
10.5 A Case Study with Survival Data
10.6 Bibliography and Notes
11 General Group Sequential Distribution Theory
11.1 Introduction
11.2 A Standard Joint Distribution for Successive Estimates of a
Parameter Vector
11.3 Normal Linear Models
11.4 Normal Linear Models with Unknown Variance: Group Sequential
t-Tests
11.5 Example: An Exact One-Sample Group Sequential t-Test
11.6 General Parametric Models: Generalized Linear Models
11.7 Bibliography and Notes
12 Binary Data
12.1 A Single Bernoulli Probability
12.2 Two Bernoulli Probabilities
12.3 The Odds Ratio and Multiple 2 × 2 Tables
12.4 Case-Control and Matched Pair Analyses
12.5 Logistic Regression: Adjusting for Covariates
12.6 Connection with Survival Analysis
13 Survival Data
13.1 Introduction
13.2 The Log-Rank Test
13.3 The Stratified Log-Rank Test
13.4 Group Sequential Methods for Survival Data with Covariates
13.5 Repeated Confidence Intervals for a Hazard Ratio
13.6 Example: A Clinical Trial for Carcinoma of the Oropharynx
13.7 Survival Probabilities and Quantiles
13.8 Bibliography and Notes
14 Internal Pilot Studies: Sample Size Re-estimation
14.1 The Role of an Internal Pilot Phase
14.2 Sample Size Re-estimation for a Fixed Sample Test
14.3 Sample Size Re-estimation in Group Sequential Tests
15 Multiple Endpoints
15.1 Introduction
15.2 The Bonferroni Procedure
15.3 A Group Sequential Hotelling Test
15.4 A Group Sequential Version of O’Brien’s Test
15.5 Tests Based on Other Global Statistics
15.6 Tests Based on Marginal Criteria
15.7 Bibliography and Notes
16 Multi-Armed Trials
16.1 Introduction
16.2 Global Tests
16.3 Monitoring Pairwise Comparisons
16.4 Bibliography and Notes
17 Adaptive Treatment Assignment
17.1 A Multi-Stage Adaptive Design
17.2 A Multi-Stage Adaptive Design with Time Trends
17.3 Validity of Adaptive Multi-stage Procedures
17.4 Bibliography and Notes
18 Bayesian Approaches
18.1 The Bayesian Paradigm
18.2 Stopping Rules
18.3 Choice of Prior Distribution
18.4 Discussion
19 Numerical Computations for Group Sequential Tests
19.1 Introduction
19.2 The Basic Calculation
19.3 Error Probabilities and Sample Size Distributions
19.4 Tests Defined by Error Spending Functions
19.5 Analysis Following a Group Sequential Test
19.6 Further Applications of Numerical Computation
19.7 Computer Software