Steven Piantadosi

Clinical Trials (eBook, PDF)

A Methodologic Perspective

• Format: PDF

Produktbeschreibung

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. 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 trials 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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Produktdetails

• Verlag: John Wiley & Sons
• Seitenzahl: 928
• Erscheinungstermin: 9. Oktober 2017
• Englisch
• ISBN-13: 9781118959213
• Artikelnr.: 52551379

Autorenporträt

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.

Inhaltsangabe

Preface to the Third Edition xxv

About the Companion Website xxviii

1 Preliminaries 1

1.1 Introduction, 1

1.2 Audiences, 2

1.3 Scope, 3

1.4 Other Sources of Knowledge, 5

1.5 Notation and Terminology, 6

1.5.1 Clinical Trial Terminology, 7

1.5.2 Drug Development Traditionally Recognizes Four Trial Design Types, 7

1.5.3 Descriptive Terminology Is Better, 8

1.6 Examples, Data, and Programs, 9

1.7 Summary, 9

2 Clinical Trials as Research 10

2.1 Introduction, 10

2.2 Research, 13

2.2.1 What Is Research?, 13

2.2.2 Clinical Reasoning Is Based on the Case History, 14

2.2.3 Statistical Reasoning Emphasizes Inference Based on Designed Data Production, 16

2.2.4 Clinical and Statistical Reasoning Converge in Research, 17

2.3 Defining Clinical Trials, 19

2.3.1 Mixing of Clinical and Statistical Reasoning Is Recent, 19

2.3.2 Clinical Trials Are Rigorously Defined, 21

2.3.3 Theory and Data, 22

2.3.4 Experiments Can Be Misunderstood, 23

2.3.5 Clinical Trials and the Frankenstein Myth, 25

2.3.6 Cavia porcellus, 26

2.3.7 Clinical Trials as Science, 26

2.3.8 Trials and Statistical Methods Fit within a Spectrum of Clinical Research, 28

2.4 Practicalities of Usage, 29

2.4.1 Predicates for a Trial, 29

2.4.2 Trials Can Provide Confirmatory Evidence, 29

2.4.3 Clinical Trials Are Reliable Albeit Unwieldy and Messy, 30

2.4.4 Trials Are Difficult to Apply in Some Circumstances, 31

2.4.5 Randomized Studies Can Be Initiated Early, 32

2.4.6 What Can I learn from = 20?, 33

2.5 Nonexperimental Designs, 35

2.5.1 Other Methods Are Valid forMaking Some Clinical Inferences, 35

2.5.2 Some Specific Nonexperimental Designs, 38

2.5.3 Causal Relationships, 40

2.5.4 Will Genetic Determinism Replace Design?, 41

2.6 Summary, 41

2.7 Questions for Discussion, 41

3 Why Clinical Trials Are Ethical 43

3.1 Introduction, 43

3.1.1 Science and Ethics Share Objectives, 44

3.1.2 Equipoise and Uncertainty, 46

3.2 Duality, 47

3.2.1 Clinical Trials Sharpen, But Do Not Create, Duality, 47

3.2.2 A Gene Therapy Tragedy Illustrates Duality, 48

3.2.3 Research and Practice Are Convergent, 48

3.2.4 Hippocratic Tradition Does Not Proscribe Clinical Trials, 52

3.2.5 Physicians Always Have Multiple Roles, 54

3.3 Historically Derived Principles of Ethics, 57

3.3.1 Nuremberg Contributed an Awareness of the Worst Problems, 57

3.3.2 High-Profile Mistakes Were Made in the United States, 58

3.3.3 The Helsinki Declaration Was Widely Adopted, 58

3.3.4 Other International Guidelines Have Been Proposed, 61

3.3.5 Institutional Review Boards Provide Ethics Oversight, 62

3.3.6 Ethics Principles Relevant to Clinical Trials, 63

3.4 Contemporary Foundational Principles, 65

3.4.1 Collaborative Partnership, 66

3.4.2 Scientific Value, 66

3.4.3 Scientific Validity, 66

3.4.4 Fair Subject Selection, 67

3.4.5 Favorable Risk-Benefit, 67

3.4.6 Independent Review, 68

3.4.7 Informed Consent, 68

3.4.8 Respect for Subjects, 71

3.5 Methodologic Reflections, 72

3.5.1 Practice Based on Unproven Treatments Is Not Ethical, 72

3.5.2 Ethics Considerations Are Important Determinants of Design, 74

3.5.3 Specific