Book description
Methods and Applications of Statistics in Clinical Trials, Volume 2: Planning, Analysis, and Inferential Methods includes updates of established literature from the Wiley Encyclopedia of Clinical Trials as well as original material based on the latest developments in clinical trials. Prepared by a leading expert, the second volume includes numerous contributions from current prominent experts in the field of medical research. In addition, the volume features:
Multiple new articles exploring emerging topics, such as evaluation methods with threshold, empirical likelihood methods, nonparametric ROC analysis, over- and under-dispersed models, and multi-armed bandit problems
Up-to-date research on the Cox proportional hazard model, frailty models, trial reports, intrarater reliability, conditional power, and the kappa index
Key qualitative issues including cost-effectiveness analysis, publication bias, and regulatory issues, which are crucial to the planning and data management of clinical trials
Table of contents
- Cover
- Half Title page
- Title page
- Copyright page
- Contributors
- Preface
- Chapter 1: Analysis of Over- and Underdispersed Data
-
Chapter 2: Analysis of Variance (ANOVA)
- 2.1 Introduction
- 2.2 Factors, Levels, Effects, and Cells
- 2.3 Cell Means Model
- 2.4 One-Way Classification
- 2.5 Parameter Estimation
- 2.6 The R(.) Notation—Partitioning Sum of Squares
- 2.7 ANOVA—Hypothesis of Equal Means
- 2.8 Multiple Comparisons
- 2.9 Two-Way Crossed Classification
- 2.10 Balanced and Unbalanced Data
- 2.11 Interaction Between Rows and Columns
- 2.12 Analysis of Variance Table
- References
- Chapter 3: Assessment of Health-Related Quality of Life
- Chapter 4: Bandit Processes and Response-Adaptive Clinical Trials: The Art of Exploration Versus Exploitation
- Chapter 5: Bayesian Dose-Finding Designs in Healthy Volunteers
-
Chapter 6: Bootstrap
- 6.1 Introduction
- 6.2 Plug-In Principle
- 6.3 Monte Carlo Sampling— The “Second Bootstrap Principle”
- 6.4 Bias and Standard Error
- 6.5 Examples
- 6.6 Model Stability
- 6.7 Accuracy of Bootstrap Distributions
- 6.8 Bootstrap Confidence Intervals
- 6.9 Hypothesis Testing
- 6.10 Planning Clinical Trials
- 6.11 How Many Bootstrap Samples Are Needed
- 6.12 Additional References
- References
- Chapter 7: Conditional Power in Clinical Trial Monitoring
- Chapter 8: Cost-Effectiveness Analysis
- Chapter 9: Cox-Type Proportional Hazards Models
-
Chapter 10: Empirical Likelihood Methods in Clinical Experiments
- 10.1 Introduction
- 10.2 Classical EL: Several Ingredients for Theoretical Evaluations
- 10.3 The Relationship Between Empirical Likelihood and Bootstrap Methodologies
- 10.4 Bayes Methods Based on Empirical Likelihoods
- 10.5 Mixtures of Likelihoods
- 10.6 An Example: ROC Curve Analyses Based on Empirical Likelihoods
- 10.7 Applications of Empirical Likelihood Methodology in Clinical Trials or Other Data Analyses
- 10.8 Concluding Remarks
- Appendix
- References
- Chapter 11: Frailty Models
- Chapter 12: Futility Analysis
- Chapter 13: Imaging Science in Medicine I: Overview
-
Chapter 14: Imaging Science in Medicine, II: Basics of X-Ray Imaging
- 14.1 Introduction to Medical Imaging: Different Ways of Creating Visible Contrast Among Tissues
- 14.2 What the Body Does to the X-Ray Beam: Subject Contrast From Differential Attenuation of the X-Ray Beam by Various Tissues
- 14.3 What the X-Ray Beam Does to the Body: Known Medical Benefits Versus Possible Radiogenic Risks
- 14.4 Capturing the Visual Image: Analog (20th Century) X-Ray Image Receptors
-
Chapter 15: Imaging Science in Medicine, III: Digital (21st Century) X-Ray Imaging
- 15.1 The Computer in Medical Imaging
- 15.2 The Digital Planar X-Ray Modalities: Computed Radiography and Digital Radiography and Fluoroscopy
- 15.3 Digital Fluoroscopy and Digital Subtraction Angiography
- 15.4 Digital Tomosynthesis: Planar Imaging in Three Dimensions
- 15.5 Computed Tomography: Superior Contrast in Three-Dimensional X-Ray Attenuation Maps
- Chapter 16: Intention-to-Treat Analysis
- Chapter 17: Interim Analyses
-
Chapter 18: Interrater Reliability
- 18.1 Definition
- 18.2 The Importance of Reliability in Clinical Trials
- 18.3 How Large a Reliability Coefficient Is Large Enough?
- 18.4 Design and Analysis of Reliability Studies
- 18.5 Estimate of the Reliability Coefficient—Parametric
- 18.6 Estimation of the Reliability Coefficient— Nonparametric
- 18.7 Estimation of the Reliability Coefficient—Binary
- 18.8 Estimation of the Reliability Coefficient—Categorical
- 18.9 Strategies to Increase Reliability (Spearman–Brown Projection)
- 18.10 Other Types of Reliabilities
- References
- Chapter 19: Intrarater Reliability
- Chapter 20: Kaplan—Meier Plot
-
Chapter 21: Logistic Regression
- 21.1 Introduction
- 21.2 Fitting the Logistic Regression Model
- 21.3 The Multiple Logistic Regression Model
- 21.4 Fitting the Multiple Logistic Regression Model
- 21.5 Example
- 21.6 Testing for the Significance of the Model
- 21.7 Interpretation of the Coefficients of the Logistic Regression Model
- 21.8 Dichotomous Independent Variable
- 21.9 Polytomous Independent Variable
- 21.10 Continuous Independent Variable
- 21.11 Multivariate Case
- References
- Chapter 22: Metadata
-
Chapter 23: Microarray
- 23.1 Introduction
- 23.2 What is a Microarray?
- 23.3 Other Array Technologies
- 23.4 Define Objectives of the Study
- 23.5 Experimental Design for Microarray
- 23.6 Data Extraction
- 23.7 Microarray Informatics
- 23.8 Statistical Analysis
- 23.9 Annotation
- 23.10 Pathway, GO, and Class-Level Analysis Tools
- 23.11 Validation of Microarray Experiments
- 23.12 Conclusions
- References
- Chapter 24: Multi-Armed Bandits, Gittins Index, and Its Calculation
- Chapter 25: Multiple Comparisons
- Chapter 26: Multiple Evaluators
-
Chapter 27: Noncompartmental Analysis
- 27.1 Introduction
- 27.2 Terminology
- 27.3 Objectives and Features of Noncompartmental Analysis
- 27.4 Comparison of Noncompartmental and Compartmental Models
- 27.5 Assumptions of NCA and Its Reported Descriptive Statistics
- 27.6 Calculation Formulas for NCA
- 27.7 Guidelines for Performance of NCA Based on Numerical Integration
- 27.8 Conclusions and Perspectives
- References
- Further Reading
-
Chapter 28: Nonparametric ROC Analysis for Diagnostic Trials
- 28.1 Introduction
- 28.2 Different Aspects of Study Design
- 28.3 Nonparametric Models and Hypotheses
- 28.4 Point Estimator
- 28.5 Asymptotic Distribution and Variance Estimator
- 28.6 Derivation of the Confidence Interval
- 28.7 Statistical Tests
- 28.8 Adaptations for Cluster Data
- 28.9 Results of a Diagnostic Study
- 28.10 Summary and Final Remarks
- References
- Chapter 29: Optimal Biological Dose for Molecularly Targeted Therapies
- Chapter 30: Over- and Underdispersion Models
- Chapter 31: Permutation Tests in Clinical Trials
- Chapter 32: Pharmacoepidemiology, Overview
- Chapter 33: Population Pharmacokinetic and Pharmacodynamic Methods
- Chapter 34: Proportions: Inferences and Comparisons
- Chapter 35: Publication Bias
- Chapter 36: Quality of Life
- Chapter 37: Relative Risk Modeling
- Chapter 38: Sample Size Considerations for Morbidity/Mortality Trials
- Chapter 39: Sample Size for Comparing Means
- Chapter 40: Sample Size for Comparing Proportions
- Chapter 41: Sample Size for Comparing Time-to-Event Data
- Chapter 42: Sample Size for Comparing Variabilities
-
Chapter 43: Screening, Models of
- 43.1 Introduction
- 43.2 What is Screening?
- 43.3 Why Use Modeling?
- 43.4 Characteristics of Screening Models
- 43.5 A Simple Disease and Screening Model
- 43.6 Analytic Models for Cancer
- 43.7 Simulation Models for Cancer
- 43.8 Model Fitting and Validation
- 43.9 Models for Other Diseases
- 43.10 Current State and Future Directions
- References
- Chapter 44: Screening Trials
- Chapter 45: Secondary Efficacy End Points
-
Chapter 46: Sensitivity, Specificity, and Receiver Operator Characteristic (ROC) Methods
- 46.1 Evaluating a Single Binary Test Against a Binary Criterion
- 46.2 Evaluation of a Single Binary Test: ROC Methods
- 46.3 Evaluation of a Test Response Measured on an Ordinal Scale: ROC Methods
- 46.4 Evaluation of Multiple Different Tests
- 46.5 The Optimal Sequence of Tests
- 46.6 Sampling and Measurement Issues
- 46.7 Summary
- References
- Chapter 47: Software for Genetics/Genomics
- Chapter 48: Stability Study Designs
-
Chapter 49: Subgroup Analysis
- 49.1 Introduction
- 49.2 The Dilemma of Subgroup Analysis
- 49.3 Planned Versus Unplanned Subgroup Analysis
- 49.4 Frequentist Methods
- 49.5 Testing Treatment by Subgroup Interactions
- 49.6 Subgroup Analyses in Positive Clinical Trials
- 49.7 Confidence Intervals for Treatment Effects within Subgroups
- 49.8 Bayesian Methods
- References
-
Chapter 50: Survival Analysis, Overview
- 50.1 Introduction
- 50.2 History
- 50.3 Survival Analysis Concepts
- 50.4 Nonparametric Estimation and Testing
- 50.5 Parametric Inference
- 50.6 Comparison with Expected Survival
- 50.7 The Cox Regression Model
- 50.8 Other Regression Models for Survival Data
- 50.9 Multistate Models
- 50.10 Other Kinds of Incomplete Observation
- 50.11 Multivariate Survival Analysis
- 50.12 Concluding Remarks
- References
-
Chapter 51: The FDA and Regulatory Issues
- 51.1 Caveat
- 51.2 Introduction
- 51.3 Chronology of Drug Regulation in the United States
- 51.4 FDA Basic Structure
- 51.5 IND Application Process
- 51.6 Drug Development and Approval Time Frame
- 51.7 NDA Process
- 51.8 U.S. Pharmacopeia and FDA
- 51.9 CDER Freedom of Information Electronic Reading Room
- 51.10 Conclusion
- Chapter 52: The Kappa Index
- Chapter 53: Treatment Interruption
-
Chapter 54: Trial Reports: Improving Reporting, Minimizing Bias, and Producing Better Evidence-Based Practice
- 54.1 Introduction
- 54.2 Reporting Issues in Clinical Trials
- 54.3 Moral Obligation to Improve the Reporting of Trials
- 54.4 Consequences of Poor Reporting of Trials
- 54.5 Distinguishing Between Methodological and Reporting Issues
- 54.6 One Solution to Poor Reporting: CONSORT 2010 and CONSORT Extensions
- 54.7 Impact of CONSORT
- 54.8 Guidance for Reporting Randomized Trial Protocols: SPIRIT
- 54.9 Trial Registration
- 54.10 Final Thoughts
- References
-
Chapter 55: U.S. Department of Veterans Affairs Cooperative Studies Program
- 55.1 Introduction
- 55.2 History of the Cooperative Studies Program (CSP)
- 55.3 Organization and Functioning of the CSP
- 55.4 Roles of the Biostatistician and Pharmacist in the CSP
- 55.5 Ongoing and Completed Cooperative Studies (1972–2000)
- 55.6 Current Challenges and Opportunities
- 55.7 Concluding Remarks
- References
-
Chapter 56: Women’s Health Initiative: Statistical Aspects and Selected Early Results
- 56.1 Introduction
- 56.2 WHI Clinical Trial and Observational Study
- 56.3 Study Organization
- 56.4 Principal Clinical Trial Comparisons, Power Calculations, and Safety and Data Monitoring
- 56.5 Biomarkers and Intermediate Outcomes
- 56.6 Data Management and Computing Infrastructure
- 56.7 Quality Assurance Program Overview
- 56.8 Early Results from the WHI Clinical Trial
- 56.9 Summary and Discussion
- References
- Chapter 57: World Health Organization (WHO): Global Health Situation
- Index
Product information
- Title: Methods and Applications of Statistics in Clinical Trials, Volume 2: Planning, Analysis, and Inferential Methods
- Author(s):
- Release date: June 2014
- Publisher(s): Wiley
- ISBN: 9781118304761
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