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Foreword I; Foreword II; Preface; Acknowledgements; Contents; Contributors; Basic Principles; 1 The Research Question and the Hypothesis; The Research Question; Getting Started; Does Your Idea Show Promise?; Refining Your Thoughts; Secondary Research Questions; The Hypothesis; "Practical Exercise"; Summary; References; 2 Primary and Secondary Endpoints; General Concepts; Measurement; The Primary Endpoint; Secondary Endpoints; Pros and Cons of Common Endpoints; Mortality; Morbidity; Pain; Procedural Outcomes; Pharmaceutical; Molecular Information; Quality of Life; Economics/Costs
"Practical Exercise"Summary; References; 3 Intervention and Control Groups; General Concepts; Interventions; Pharmacologic Intervention; Procedural Intervention; Patient Care Intervention; Control Groups; Blinding; "Practical Exercise"; Summary; References; 4 Subject Selection; General Concepts; Inclusion Criteria; Exclusion Criteria; Vulnerable Populations; Informed Consent; "Practical Exercise"; Summary; References; Study Designs; 5 Clinical Phases of Device and Drug Evaluation with Emphasis on Early Phase Trials; Overview of Regulatory Phases for Investigational Agents
Regulatory Phases for Device ApprovalFeasibility Study; Pivotal Study; Regulatory Phases for Trials of Drugs, Vaccines and Biologics; Phase 0 Trials; Phase I Clinical Studies; Estimation of Initial Safety and Tolerability; Pharmacokinetics/Pharmacodynamics (PK/PD); Early Measurement of Drug Activity; Phase I Trial Designs; Phase II Clinical Studies; Phase IIa Studies; Proof of Concept Studies; Example; Phase IIb Studies; Phase II Trial Designs; Phase III Clinical Studies; Procedural Trials; References; 6 Overview of the Randomized Clinical Trial and the Parallel Group Design
Clinical Research Milestones Before the Advent of the Modern Randomized Clinical TrialStreptomycin for the Treatment of Tuberculosis; The Benefits of Randomization; Trial Timing; Ethics; Feasibility; Randomization Is Not Enough, What Happens After Randomization Is Just as Important; The Two-Parallel-Group Trial; The Multiple-Parallel-Group Trial; Example; Conclusion; References; 7 Non-inferiority and Equivalence Trials; Overview; Hypothesis Testing; Confidence Interval Approach for Equivalence and Non-inferiority Trials
Example: Oral Versus Intratympanic Steroids for Treatment of Idiopathic Sudden Sensorineural Hearing LossExample: ACOSOG Z6051-Laparoscopic-Assisted Resection Versus Open Resection of Stage II or III Rectal Cancer; Choosing a Non-inferiority Design Versus a Traditional Parallel Group Design; Choice of the Non-inferiority (or Equivalence) Margin; Non-inferiority Is Not Transitive; Poor Study Conduct Makes Establishing Non-inferiority Easier; Guidance on Non-inferiority Trials; Summary; References; 8 Factorial Designs; Definitions and Examples of Factorial Designs
"Practical Exercise"Summary; References; 3 Intervention and Control Groups; General Concepts; Interventions; Pharmacologic Intervention; Procedural Intervention; Patient Care Intervention; Control Groups; Blinding; "Practical Exercise"; Summary; References; 4 Subject Selection; General Concepts; Inclusion Criteria; Exclusion Criteria; Vulnerable Populations; Informed Consent; "Practical Exercise"; Summary; References; Study Designs; 5 Clinical Phases of Device and Drug Evaluation with Emphasis on Early Phase Trials; Overview of Regulatory Phases for Investigational Agents
Regulatory Phases for Device ApprovalFeasibility Study; Pivotal Study; Regulatory Phases for Trials of Drugs, Vaccines and Biologics; Phase 0 Trials; Phase I Clinical Studies; Estimation of Initial Safety and Tolerability; Pharmacokinetics/Pharmacodynamics (PK/PD); Early Measurement of Drug Activity; Phase I Trial Designs; Phase II Clinical Studies; Phase IIa Studies; Proof of Concept Studies; Example; Phase IIb Studies; Phase II Trial Designs; Phase III Clinical Studies; Procedural Trials; References; 6 Overview of the Randomized Clinical Trial and the Parallel Group Design
Clinical Research Milestones Before the Advent of the Modern Randomized Clinical TrialStreptomycin for the Treatment of Tuberculosis; The Benefits of Randomization; Trial Timing; Ethics; Feasibility; Randomization Is Not Enough, What Happens After Randomization Is Just as Important; The Two-Parallel-Group Trial; The Multiple-Parallel-Group Trial; Example; Conclusion; References; 7 Non-inferiority and Equivalence Trials; Overview; Hypothesis Testing; Confidence Interval Approach for Equivalence and Non-inferiority Trials
Example: Oral Versus Intratympanic Steroids for Treatment of Idiopathic Sudden Sensorineural Hearing LossExample: ACOSOG Z6051-Laparoscopic-Assisted Resection Versus Open Resection of Stage II or III Rectal Cancer; Choosing a Non-inferiority Design Versus a Traditional Parallel Group Design; Choice of the Non-inferiority (or Equivalence) Margin; Non-inferiority Is Not Transitive; Poor Study Conduct Makes Establishing Non-inferiority Easier; Guidance on Non-inferiority Trials; Summary; References; 8 Factorial Designs; Definitions and Examples of Factorial Designs