Studies that are unimpeachably thorough, non-political, unbiased, and properly designed… These are the standards to which everyone in clinical research aspires. Yet, the difficulties in designing trials and interpreting data are subtle and ever present. The new edition of Clinical Trials in Oncology provides a concise, nontechnical, and now thoroughly up-to-date review of methods and issues related to clinical trials. The authors emphasize the importance of proper study design, analysis, and data management and identify the major pitfalls that are seemingly inherent in these processes. This edition includes a new section that describes recent innovations in Phase I designs. Another new section on microarray data examines the challenges presented by massive data sets and describes approaches used to meet those challenges. As always, the authors use clear, lucid prose and a multitude of real-world trials as examples to convey the principles of successful trials without the need for a strong statistics or mathematics background. Although the book focuses on cancer trials, the issues and concepts are important in any clinical setting. Clinical Trials in Oncology, Second Edition works to improve the mutual understanding by clinicians and statisticians of the principles of clinical trials and helps them avoid the many hazards that can jeopardize the success of a trial.
Author(s): Stephanie Green, John Crowley, Jacqueline Benedetti, Angela Smith
Edition: 2ed.
Publisher: CRC
Year: 2002
Language: English
Pages: 258
Interdisciplinary Statistics CLINICAL TRIALS in ONCOLOGY, Second Edition......Page 1
Acknowlegdments......Page 11
Contents......Page 6
Bibliography......Page 242
1.1 A brief history of clinical trials......Page 12
1.2 The Southwest Oncology Group......Page 17
1.3 Example trials......Page 18
1.4 The reason for this book......Page 20
2.1 Introduction......Page 21
2.2 The Phase II trial — estimation......Page 29
2.3 The Phase III trial – hypothesis testing......Page 33
2.3.1 Response as the outcome......Page 34
2.3.2 Survival as the outcome......Page 40
2.4 The proportional hazards model......Page 47
2.5 Sample size calculations......Page 49
2.6 Concluding remarks......Page 50
3.1 Introduction......Page 51
RYRY3 Uligibility treatments endpoints......Page 52
RYRYM Assumptions for sample size calculation......Page 53
RYRYR Traditional designs......Page 58
RYRY3 Newer Phase I designs......Page 59
RYRY: Eonsiderations for biologic agents......Page 62
3.4 Phase II trials......Page 63
RY:YR The Standard Southwest Oncology roup Phase II design......Page 64
activityY......Page 66
not......Page 68
pilot studiesj......Page 71
StratiYcation factors......Page 72
Randomization schemes......Page 73
adaptive allocation......Page 74
intent to treat principle......Page 75
randomized consent designs......Page 76
Placebo’controlled......Page 77
RYMY3 Two’arm trials......Page 79
prove......Page 80
proportional hazards assumptionY......Page 81
Multiple endpoints......Page 82
proving......Page 83
3.6 Conclusion......Page 86
4.1 Introduction......Page 88
4.2 Types of multi-arm trials......Page 89
4.3 Significance level......Page 92
4.4 Power......Page 93
5.1 Planned interim analyses......Page 105
5.2 Data monitoring committees: Rationale and responsibilities......Page 111
5.3 Monitoring committees: Composition......Page 115
5.4 Examples......Page 120
5.5 Concluding remarks......Page 130
6.1 Introduction: Why worry?......Page 131
6.2 Protocol development......Page 136
6.3 Data collection......Page 144
6.4 Protocol management and evaluation......Page 151
6.5 Quality assurance audits......Page 157
6.6 Training......Page 158
6.7 Data base management......Page 159
6.8 Conclusion......Page 162
6.9 Appendix: Examples......Page 163
CHAPTER 7: Reporting of Results......Page 172
7.1 Timing of report......Page 173
7.2 Required information......Page 175
7.3 Analyses......Page 177
7.4 Conclusion......Page 186
8.2 Historical controls......Page 187
8.3 Competing risks......Page 194
8.4 Outcome by outcome analyses......Page 201
8.5 Subset analyses......Page 209
8.6 Surrogate endpoints......Page 212
9.1 Introduction......Page 215
9.2 Some background and notation......Page 216
9.3 Identification of prognostic factors......Page 218
9.4 Forming prognostic groups......Page 225
9.5 Analysis of microarray data......Page 230
9.6 Meta-Analysis......Page 232
9.7 Concluding remarks......Page 238
CHAPTER 10: Summary and Conclusions......Page 239
