This Third Edition builds on the text’s reputation as a straightforward, detailed, and authoritative presentation of quantitative methods for clinical trials. Readers will encounter the principles of design for various types of clinical trials, and are then skillfully guided through the complete process of planning the experiment, assembling a study cohort, assessing data, and reporting results. Throughout the process, the author alerts readers to problems that may arise during the course of the trial and provides common sense solutions. All stages of therapeutic development are discussed in detail, and the methods are not restricted to a single clinical application area.

The author bases current revisions and updates on his own experience, classroom instruction, and feedback from teachers and medical and statistical professionals involved in clinical trials. The Third Edition greatly expands its coverage, ranging from statistical principles to new and provocative topics, including alternative medicine and ethics, middle development, comparative studies, and adaptive designs. At the same time, it offers more pragmatic advice for issues such as selecting outcomes, sample size, analysis, reporting, and handling allegations of misconduct. Readers familiar with the First and Second Editions will discover revamped exercise sets, an updated and extensive reference section, new material on endpoints and the developmental pipeline, among others, and revisions of numerous sections.

In addition, this book:

• Features accessible and broad coverage of statistical design methods—the crucial building blocks of clinical trials and medical research—now complete with new chapters on overall development, middle development, comparative studies, and adaptive designs
• Teaches readers to design clinical trials that produce valid qualitative results backed by rigorous statistical methods
• Contains an introduction and summary in each chapter to reinforce key points
• Includes discussion questions to stimulate critical thinking and help readers understand how they can apply their newfound knowledge
• Provides extensive references to direct readers to the most recent literature
• Includes numerous new or revised exercises throughout

Clinical Trials: A Methodologic Perspective, Third Edition is a textbook accessible to advanced undergraduate students in the quantitative sciences, graduate students in public health and the life sciences, physicians training in clinical research methods, and biostatisticians and epidemiologists.

Contents

۱: Preliminaries;
۲: Clinical Trials as Research
۳: Why Clinical Trials Are Ethical
۴: Contexts for Clinical Trials
۵: Measurement
۶: Random Error and Bias
۷: Statistical Perspectives
۸: Experiment Design in Clinical Trials
۹: The Trial Cohort
۱۰: Development Paradigms
۱۱: Translational Clinical Trials
۱۲: Early Development and Dose-Finding
۱۳: Middle Development
۱۴: Comparative Trials
۱۵: Adaptive Design Features
۱۶: Sample Size and Power
۱۷: Treatment Allocation
۱۸: Treatment Effects Monitoring
۱۹: Counting Subjects and Events
۲۰: Estimating Clinical Effects
۲۱: Prognostic Factor Analyses
۲۲: Factorial Designs
۲۳: Crossover Designs
۲۴: Meta-Analyses
۲۵: Reporting and Authorship
۲۶: Misconduct and Fraud in Clinical Research
Appendix A: Data and Programs
Appendix B: Abbreviations
Appendix C: Notation and Terminology
Appendix D: Nuremberg Code