Introduction to statistical methods for clinical trials /
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| Imprint: | Boca Raton : Chapman & Hall/CRC, c2008. |
|---|---|
| Description: | xxiii, 439 p. : ill. ; 25 cm. |
| Language: | English |
| Series: | Chapman & Hall/CRC texts in statistical science series Texts in statistical science. |
| Subject: | |
| Format: | Print Book |
| URL for this record: | http://pi.lib.uchicago.edu/1001/cat/bib/6806407 |
Table of Contents:
- List of figures
- List of tables
- Preface
- Author Attribution
- 1. Introduction to Clinical Trials
- 1.1. History and Background
- 1.2. Ethics of Clinical Research
- 1.3. Types of Research Design and Types of Trials
- 1.4. The Need for Clinical Trials
- 1.5. The Randomization Principle
- 1.6. Timing of a Clinical Trial
- 1.7. Trial Organization
- 1.8. Protocol and Manual of Operations
- 1.9. Regulatory Issues
- 1.10. Overview of the Book
- 2. Defining the Question
- 2.1. Statistical Framework
- 2.2. Elements of Study Question
- 2.3. Outcome or Response Measures
- 2.4. The Surrogate Outcome
- 2.5. Composite Outcomes
- 2.6. Summary
- 2.7. Problems
- 3. Study Design
- 3.1. Early Phase Trials
- 3.2. Phase III/IV Trials
- 3.3. Non-inferiority Designs
- 3.4. Screening, Prevention, and Therapeutic Designs
- 3.5. Adaptive Designs
- 3.6. Conclusions
- 3.7. Problems
- 4. Sample Size
- 4.1. Sample Size versus Information
- 4.2. A General Setup for Frequentist Designs
- 4.3. Loss to Follow-up and Non-adherence
- 4.4. Survival Data
- 4.5. Clustered Data
- 4.6. Tests for Interaction
- 4.7. Equivalence/Non-inferiority Trials
- 4.8. Other Considerations
- 4.9. Problems
- 5. Randomization
- 5.1. The Role of Randomization
- 5.2. Fixed Randomization Procedures
- 5.3. Treatment- and Response-Adaptive Randomization Procedures
- 5.4. Covariate-Adaptive Randomization Procedures
- 5.5. Summary and Recommendations
- 5.6. Problems
- 6. Data Collection and Quality Control
- 6.1. Planning for Collection of Clinical Trial Data
- 6.2. Categories of Clinical Data
- 6.3. Data Quality Control
- 6.4. Conclusions
- 7. Survival Analysis
- 7.1. Background
- 7.2. Estimation of Survival Distributions
- 7.3. Comparison of Survival Distributions
- 7.4. Regression Models
- 7.5. Composite Outcomes
- 7.6. Summary
- 7.7. Problems
- 8. Longitudinal Data
- 8.1. A Clinical Longitudinal Data Example
- 8.2. The Subject-specific Model
- 8.3. Two-stage Estimation
- 8.4. The Random-effects, Subject-specific Model
- 8.5. The Population-average (Marginal) Model
- 8.6. Restricted Maximum Likelihood Estimation (REML)
- 8.7. Standard Errors
- 8.8. Testing
- 8.9. Additional Levels of Clustering
- 8.10. Generalized Estimating Equations for Non-normal Data
- 8.11. Missing Data
- 8.12. Summary
- 9. Quality of Life
- 9.1. Defining QoL
- 9.2. Types of QoL Assessments
- 9.3. Selecting a QoL Instrument
- 9.4. Developing a QoL Instrument
- 9.5. Quality of Life Data
- 9.6. Analysis of QoL Data
- 9.7. Summary
- 10. Data Monitoring and Interim Analysis
- 10.1. Data and Safety Monitoring
- 10.2. Examples
- 10.3. The Repeated Testing Problem
- 10.4. Group Sequential Tests
- 10.5. Triangular Test
- 10.6. Curtailment Procedures
- 10.7. Inference Following Sequential Tests
- 10.8. Discussion
- 10.9. Problems
- 11. Selected Issues in the Analysis
- 11.1. Bias in the Analysis of Clinical Trial Data
- 11.2. Choice of Analysis Population
- 11.3. Missing Data
- 11.4. Subgroup Analyses
- 11.5. Multiple Testing Procedures
- 11.6. Summary
- 11.7. Problems
- 12. Closeout and Reporting
- 12.1. Closing Out a Trial
- 12.2. Reporting Trial Results
- 12.3. Problems
- A. Delta Method, Maximum Likelihood Theory, and Information
- A.1. Delta Method
- A.2. Asymptotic Theory for Likelihood Based Inference
- A.3. Hypothesis Testing
- A.4. Computing the MLE
- A.5. Information
- A.6. Brownian Motion
- References
- Index
