Clinical Trials: A Methodologic Perspective (3rd Edition) | KitaabNow

Clinical Trials: A Methodologic Perspective (3rd Edition)

 32,356
  • Author: Steven Piantadosi
  • ISBN: 9781118959206
  • Publisher: Wiley Publishing
  • Edition: 3rd
  • Publication Date: October 27, 2017
  • Format: Hardback – 928 pages
  • Language: English


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Description

Presents elements of clinical trial methods that are essential in planning, designing, conducting, analyzing, and interpreting clinical trials with the goal of improving the evidence derived from these important studies

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 authors 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, and there are numerous new or revised exercises throughout the book

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.

This book is accompanied by downloadable files available below under the DOWNLOADS tab.

These files include:

  • MATHEMATICA program – A set of downloadable files that tracks the chapters, containing code pertaining to each.
  • SAS PROGRAMS and DATA FILES used in the book.

The following software programs, included in the downloadables, were developed by the author, Steven Piantadosi, M.D., Ph.D:

  • RANDOMIZATION – This program generates treatment assignments for a clinical trial using blocked stratified randomization.
  • CRM – Implements the continual reassessment methods for dose finding clinical trials.
  • OPTIMAL – Calculates two-stage optimal phase II designs using the Simon method.
  • POWER – This is a power and sample size program for clinical trials.
Table of Contents
  1. Preface to the Third Edition xxv
  2. About the Companion Website xxviii
  3. Preliminaries 1
    1. Introduction 1
    2. Audiences 2
    3. Scope 3
    4. Other Sources of Knowledge 5
    5. Notation and Terminology 6
    6. Examples, Data, and Programs 9
    7. Summary 9
  4. Clinical Trials as Research 10
    1. Introduction 10
    2. Research 13
    3. Defining Clinical Trials 19
    4. Practicalities of Usage 29
    5. Nonexperimental Designs 35
    6. Summary 41
    7. Questions for Discussion 41
  5. Why Clinical Trials are Ethical 43
    1. Introduction 43
    2. Duality 47
    3. Historically Derived Principles of Ethics 57
    4. Contemporary Foundational Principles 65
    5. Methodologic Reflections 72
    6. Professional Conduct 79
    7. Summary 85
    8. Questions for Discussion 86
  6. Contexts for Clinical Trials 87
    1. Introduction 87
    2. Drugs 91
    3. Devices 95
    4. Prevention 99
    5. Complementary and Alternative Medicine 106
    6. Surgery and Skill-Dependent Therapies 116
    7. A Brief View of Some Other Contexts 130
    8. Summary 135
    9. Questions for Discussion 136
  7. Measurement 137
    1. Introduction 137
    2. Objectives 140
    3. Measurement Design 143
    4. Surrogate Outcomes 162
    5. Summary 170
    6. Questions for Discussion 171
  8. Random Error and Bias 172
    1. Introduction 172
    2. Clinical Bias 181
    3. Statistical Bias 188
    4. Summary 194
    5. Questions for Discussion 194
  9. Statistical Perspectives 196
    1. Introduction 196
    2. Differences in Statistical Perspectives 197
    3. Frequentist 202
    4. Bayesian 204
    5. Likelihood 210
    6. Statistics Issues 212
    7. Summary 215
    8. Questions for Discussion 216
  10. Experiment Design in Clinical Trials 217
    1. Introduction 217
    2. Trials As Simple Experiment Designs 218
    3. Goals of Experiment Design 223
    4. Design Concepts 225
    5. Design Features 230
    6. Special Design Issues 237
    7. Importance of the Protocol Document 244
    8. Summary 252
    9. Questions for Discussion 253
  11. The Trial Cohort 254
    1. Introduction 254
    2. Cohort Definition and Selection 255
    3. Modeling Accrual 264
    4. Inclusiveness, Representation, and Interactions 267
    5. Summary 275
    6. Questions for Discussion 275
  12. Development Paradigms 277
    1. Introduction 277
    2. Pipeline Principles and Problems 281
    3. A Simple Quantitative Pipeline 286
    4. Late Failures 292
    5. Summary 300
    6. Questions for Discussion 301
  13. Translational Clinical Trials 302
    1. Introduction 302
    2. 11.2 Inferential Paradigms 308
    3. Evidence and Theory 312
    4. Translational Trials Defined 313
    5. Information From Translational Trials 317
    6. Sample Size for Translational Trials 324
    7. Validity 327
    8. Summary 328
    9. Questions for Discussion 328
  14. Early Development and Dose-Finding 329
    1. Introduction 329
    2. Basic Concepts 330
    3. Essential Concepts for Dose versus Risk 333
    4. Dose-Ranging 338
    5. Dose-Finding is Model Based 344
    6. General Dose-Finding Issues 354
    7. Summary 366
    8. Questions for Discussion 368
  15. Middle Development 370
    1. Introduction 370
    2. Characteristics of Middle Development 372
    3. Design Issues 375
    4. Middle Development Distills True Positives 379
    5. Futility and Nonsuperiority Designs 381
    6. Dose–Efficacy Questions 385
    7. Randomized Comparisons 386
    8. Cohort Mixtures 392
    9. Summary 395
    10. Questions for Discussion 396
  16. Comparative Trials 397
    1. Introduction 397
    2. Elements of Reliability 398
    3. Biomarker-Based Comparative Designs 402
    4. Some Special Comparative Designs 408
    5. Summary 411
    6. Questions for Discussion 412
  17. Adaptive Design Features 413
    1. Introduction 413
    2. Some Familiar Adaptations 418
    3. Biomarker Adaptive Trials 423
    4. Re-Designs 425
    5. Seamless Designs 427
    6. Barriers to the Use of AD 428
    7. Adaptive Design Case Study 428
    8. Summary 429
    9. Questions for Discussion 429
  18. Sample Size and Power 430
    1. Introduction 430
    2. Principles 431
    3. Early Developmental Trials 436
    4. Simple Estimation Designs 438
    5. Event Rates 451
    6. Staged Studies 455
    7. Comparative Trials 457
    8. Expanded Safety Trials 478
    9. Other Considerations 481
    10. Summary 489
    11. Questions for Discussion 490
  19. Treatment Allocation 492
    1. Introduction 492
    2. Randomization 494
    3. Constrained Randomization 500
    4. Adaptive Allocation 504
    5. Other Issues Regarding Randomization 507
    6. Unequal Treatment Allocation 514
    7. Randomization Before Consent 519
    8. Summary 520
    9. Questions for Discussion 520
  20. Treatment Effects Monitoring 522
    1. Introduction 522
    2. Administrative Issues in Trial Monitoring 527
    3. Organizational Issues Related to Monitoring 537
    4. Statistical Methods for Monitoring 545
    5. Summary 570
    6. Questions for Discussion 572
  21. Counting Subjects and Events 573
    1. Introduction 573
    2. Imperfection and Validity 574
    3. Treatment Nonadherence 575
    4. Protocol Nonadherence 580
    5. Data Imperfections 583
    6. Summary 588
    7. Questions for Discussion 589
  22. Estimating Clinical Effects 590
    1. Introduction 590
    2. Dose-Finding and Pharmacokinetic Trials 594
    3. Middle Development Studies 599
    4. Randomized Comparative Trials 606
    5. Problems With P-Values 616
    6. Strength of Evidence Through Support Intervals 620
    7. Special Methods of Analysis 622
    8. Exploratory Analyses 628
    9. Summary 639
    10. Questions for Discussion 640
  23. Prognostic Factor Analyses 644
    1. Introduction 644
    2. Model-Based Methods 647
    3. Adjusted Analyses of Comparative Trials 661
    4. PFAS Without Models 666
    5. Summary 669
    6. Questions for Discussion 669
  24. Factorial Designs 671
    1. Introduction 671
    2. Characteristics of Factorial Designs 672
    3. Treatment Interactions 675
    4. Examples of Factorial Designs 680
    5. Partial Fractional and Incomplete Factorials 682
    6. Summary 683
    7. Questions for Discussion 683
  25. Crossover Designs 684
    1. Introduction 684
    2. Advantages and Disadvantages 686
    3. Analysis 691
    4. Classic Case Study 696
    5. Summary 696
    6. Questions for Discussion 697
  26. Meta-Analyses 698
    1. Introduction 698
    2. A Sketch of Meta-Analysis Methods 700
    3. Other Issues 705
    4. Summary 707
    5. Questions for Discussion 708
  27. Reporting and Authorship 709
    1. Introduction 709
    2. General Issues in Reporting 710
    3. Clinical Trial Reports 715
    4. Authorship 726
    5. Other Issues in Disseminating Results 731
    6. Summary 732
    7. Questions for Discussion 733
  28. Misconduct and Fraud in Clinical Research 734
    1. Introduction 734
    2. Research Practices 741
    3. Approach to Allegations of Misconduct 743
    4. Characteristics of Some Misconduct Cases 747
    5. Lessons 754
    6. Clinical Investigators’ Responsibilities 757
    7. Summary 759
    8. Questions for Discussion 760
  29. Appendix A Data and Programs 761
    1. A.1 Introduction 761
    2. A.2 Design Programs 761
    3. A.3 Mathematica Code 763
  30. Appendix B Abbreviations 764
  31. Appendix C Notation and Terminology 769
    1. C.1 Introduction 769
    2. C.2 Notation 769
    3. C.3 Terminology and Concepts 772
  32. Appendix D Nuremberg Code 788
    1. D.1 Permissible Medical Experiments 788
  33. References 790
  34. Index 871
Author Biography

Steven Piantadosi, MD, PhD, is the Phase One Foundation Distinguished Chair and Director of the Samuel Oschin Cancer Institute, and Professor of Medicine at Cedars-Sinai Medical Center in Los Angeles, California. Dr. Piantadosi is one of the world’s leading experts in the design and analysis of clinical trials for cancer research. He has taught clinical trial methods extensively in formal courses and short venues. He has advised numerous academic programs and collaborations nationally regarding clinical trial design and conduct, and has served on external advisory boards for the National Institutes of Health and other prominent cancer programs and centers. The author of more than 260 peer-reviewed scientific articles, Dr. Piantadosi has published extensively on research results, clinical applications, and trial methodology. While his papers have contributed to many areas of oncology, he has also collaborated on diverse studies outside oncology including lung disease and degenerative neurological disease

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