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Clinical Trials: A Methodologic Perspective, 2nd Edition

Clinical Trials: A Methodologic Perspective, 2nd Edition

Steven Piantadosi

ISBN: 978-1-118-62585-9 May 2013 720 Pages




Learn rigorous statistical methods to ensure valid clinical trials

This Second Edition of the critically hailed Clinical Trials builds on the text's reputation as a straightforward and authoritative presentation of statistical methods for clinical trials. Readers are introduced to the fundamentals of design for various types of clinical trials and 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 commonsense solutions.

The author bases the revisions and updates on his own classroom experience, as well as feedback from students, instructors, and medical and statistical professionals involved in clinical trials. The Second Edition greatly expands its coverage, ranging from statistical principles to controversial topics, including alternative medicine and ethics. 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 Edition will discover completely new chapters, including:
* Contexts for clinical trials
* Statistical perspectives
* Translational clinical trials
* Dose-finding and dose-ranging designs

Each chapter is accompanied by a summary to reinforce the key points. Revised discussion questions stimulate critical thinking and help readers understand how they can apply their newfound knowledge, and updated references are provided to direct readers to the most recent literature.

This text distinguishes itself with its accessible and broad coverage of statistical design methods--the crucial building blocks of clinical trials and medical research. Readers learn to conduct clinical trials that produce valid qualitative results backed by rigorous statistical methods.

Preface to the First Edition.

1. Preliminaries.

2. Clinical Trails as Research.

3. Why Clinical Trials Are Ethical.

4. Contexts for Clinical Trials.

5. Statistical Perspectives.

6. Clinical Trials as Experimental Designs.

7. Random Error and Bias.

8. Objectives and Outcomes.

9. Translational Clinical Trials.

10. Dose Finding Designs.

11. Sample Size and Power.

12. The Study Cohort.

13. Treatment Allocation.

14. Treatment Effects Monitoring.

15. Counting Patients and Events.

16. Estimating Clinical Effects.

17. Prognostic Factor Analyses.

18. Reporting and Authorship.

19. Factorial Designs.

20 Cross-Over Designs.

21. Meta-Analyses.

22. Misconduct and Fraud in Clinical Research.

Appendix A: Data and Programs.

Appendix B: Notation and Terminology.

Appendix C: Abbreviations.

Appendix D: Nuremberg Code.

Appendix E: Declaration of Helsinki.

Appendix F: NCI Data and Safety Monitoring Policy.

Appendix G: NIH Data and Safety Monitoring Policy.

Appendix H: Royal Statistical Society Code of Conduct.


Author Index.

Subject Index.

"…clearly written, practical presentations of the standard topics, and also fascinating discussion you won't find elsewhere…" (Journal of Clinical Research Best Practices, August 2007)

"…a very good addition to the clinical trials area." (Statistical Methods in Medical Research, October 2006)

"…an excellent reference for both clinicians and statisticians." (Journal of Biopharmaceutical Statistics, May/June 2006)

  • Covers vital design considerations
  • Emphasizes experimental designs to search for treatment advances
  • Focuses on concepts that unify
  • Explores areas of controversy such as ethics (now greatly expanded) and offers pragmatic information regarding allegations of fraud or misconduct
  • Includes summaries, revised discussion questions, and updated references in each chapter
  • Accompanied by an ftp site, dozens of new, redrawn, and/or updated illustrations, a comprehensive bibliography, and multiple indexes
  • Incorporates new content, including new chapters on contexts, perspectives, transitional trials, and early developmental drug design
  • Now, extensively class-tested