Adaptive design methods in clinical trials:
"One of the first books on the topic, Adaptive Design Methods in Clinical Trials presents the principles and methodologies in adaptive design and analysis the pertain to adaptations made to trial or statistical procedures that are based on accrued data of ongoing clinical trials. The book also...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Book |
| Language: | English |
| Published: |
Boca Raton, Fla.
Chapman & Hall/CRC
2007
|
| Series: | Chapman & Hall/CRC biostatistics series
17 |
| Subjects: | |
| Online Access: | Publisher description Inhaltsverzeichnis |
| Summary: | "One of the first books on the topic, Adaptive Design Methods in Clinical Trials presents the principles and methodologies in adaptive design and analysis the pertain to adaptations made to trial or statistical procedures that are based on accrued data of ongoing clinical trials. The book also offers a well-balanced summary of current regulatory perspectives and recently developed statistical methods in this area."--Cover, p. 4. |
| Item Description: | Includes bibliographical references (p. [255]-267) and index |
| Physical Description: | 277 S. graph. Darst. 24 cm |
| ISBN: | 1584887761 |
Staff View
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| 500 | |a Includes bibliographical references (p. [255]-267) and index | ||
| 520 | 1 | |a "One of the first books on the topic, Adaptive Design Methods in Clinical Trials presents the principles and methodologies in adaptive design and analysis the pertain to adaptations made to trial or statistical procedures that are based on accrued data of ongoing clinical trials. The book also offers a well-balanced summary of current regulatory perspectives and recently developed statistical methods in this area."--Cover, p. 4. | |
| 650 | 4 | |a Adaptive sampling (Statistics) | |
| 650 | 4 | |a Biometry |x methods | |
| 650 | 4 | |a Clinical Trials as Topic |x methods | |
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Record in the Search Index
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| adam_text | Contents
1 Introduction 1
1.1 What Is Adaptive Design? 3
1.2 Regulatory Perspectives 6
1.3 Target Patient Population 8
1.4 Statistical Inference 10
1.5 Practical Issues 11
1.5.1 Moving target patient population 12
1.5.2 Adaptive randomization 13
1.5.3 Adaptive hypotheses 14
1.5.4 Adaptive dose-escalation trials 15
1.5.5 Adaptive group sequential design 15
1.5.6 Adaptive sample size adjustment 16
1.5.7 Adaptive seamless phase II/III design ... 17
1.5.8 Adaptive treatment switching 18
1.5.9 Bayesian and hybrid approaches 18
1.5.10 Clinical trial simulation 19
1.5.11 Case studies 19
1.6 Aims and Scope of the Book 20
2 Protocol Amendment 23
2.1 Actual Patient Population 23
2.2 Estimation of Shift and Scale Parameters 26
2.2.1 The case where n Actual is random
and ^Actual is fixed 28
2.3 Statistical Inference 31
2.3.1 Test for equality 33
2.3.2 Test for non-inferiority/superiority 34
2.3.3 Test for equivalence 34
2.4 Sample Size Adjustment 35
2.4.1 Test for equality 35
2.4.2 Test for non-inferiority/superiority 36
2.4.3 Test for equivalence 37
2.5 Statistical Inference with Covariate Adjustment . . 38
2.5.1 Population and assumption 38
2.5.2 Conditional inference 39
2.5.3 Unconditional inference 40
2.6 Concluding Remarks 43
3 Adaptive Randomization 47
3.1 Conventional Randomization 48
3.2 Treatment-Adaptive Randomization 52
3.3 Covariate-Adaptive Randomization 55
3.4 Response-Adaptive Randomization 58
3.5 Issues with Adaptive Randomization 70
3.6 Summary 73
4 Adaptive Hypotheses 75
4.1 Modifications of Hypotheses 76
4.2 Switch from Superiority to Non-Inferiority 78
4.3 Concluding Remarks 87
5 Adaptive Dose-Escalation Trials 89
5.1 Introduction 89
5.2 CRM in Phase I Oncology Study 91
5.3 Hybrid Frequentist-Bayesian Adaptive Design ... 93
5.4 Simulations 100
5.5 Concluding Remarks 104
6 Adaptive Group Sequential Design 107
6.1 Sequential Methods 108
6.2 General Approach for Group Sequential Design . . . 112
6.3 Early Stopping Boundaries 114
6.4 Alpha Spending Function 122
6.5 Group Sequential Design Based on Independent
p-Values 123
6.6 Calculation of Stopping Boundaries 125
6.7 Group Sequential Trial Monitoring 128
6.8 Conditional Power 133
6.9 Practical Issues 135
7 Adaptive Sample Size Adjustment 137
7.1 Sample Size Re-estimation without
Unblinding Data 138
7.2 Cui-Hung-Wang s Method 140
7.3 Proschan-Hunsberger s Method 142
7.4 Muller-Schafer Method 146
7.5 Bauer-Kohne Method 146
7.6 Generalization of Independent p-Value
Approaches 148
7.7 Inverse-Normal Method 157
7.8 Concluding Remarks 158
8 Adaptive Seamless Phase II/III Designs 161
8.1 Why a Seamless Design Is Efficient 161
8.2 Step-Wise Test and Adaptive Procedures 162
8.3 Contrast Test and Naive p-Value 163
8.4 Comparison of Seamless Designs 165
8.5 Drop-the-Loser Adaptive Design 167
8.6 Summary 171
9 Adaptive Treatment Switching 173
9.1 Latent Event Times 174
9.2 Proportional Hazard Model with Latent
Hazard Rate 177
9.2.1 Simulation results 179
9.3 Mixed Exponential Model 181
9.3.1 Biomarker-based survival model 182
9.3.2 Effect of patient enrollment rate 184
9.3.3 Hypothesis test and power analysis 187
9.3.4 Application to trials with treatment
switch 189
9.4 Concluding Remarks 193
10 Bayesian Approach 195
10.1 Basic Concepts of Bayesian Approach 195
10.1.1 Bayes rule 196
10.1.2 Bayesian power 200
10.2 Multiple-Stage Design for Single-Arm Trial 201
10.2.1 Classical approach for two-stage
design 202
10.2.2 Bayesian approach 203
10.3 Bayesian Optimal Adaptive Designs 205
10.4 Concluding Remarks 209
11 Clinical Trial Simulation 211
11.1 Simulation Framework 212
11.2 Early Phases Development 213
11.2.1 Dose limiting toxicity (DLT) and maximum
tolerated dose (MTD) 214
11.2.2 Dose-level selection 214
11.2.3 Sample size per dose level 215
11.2.4 Dose-escalation design 215
11.3 Late Phases Development 215
11.3.1 Randomization rules 216
11.3.2 Early stopping rules 216
11.3.3 Rules for dropping losers 216
11.3.4 Sample size adjustment 217
11.3.5 Response-adaptive randomization 217
11.3.6 Utility-offset model 218
11.3.7 Null-model versus model approach 219
11.3.8 Alpha adjustment 219
11.4 Software Application 220
11.4.1 Overview of ExpDesign studio 220
11.4.2 How to design a trial with
ExpDesign studio 222
11.4.3 How to design a conventional trial 222
11.4.4 How to design a group sequential trial . . . 223
11.4.5 How to design a multi-stage trial 224
11.4.6 How to design a dose-escalation trial .... 225
11.4.7 How to design an adaptive trial 227
11.5 Examples 227
11.5.1 Early phases development 228
11.5.2 Late phases development 230
11.6 Concluding Remarks 235
12 Case Studies 239
12.1 Basic Considerations 239
12.1.1 Dose and dose regimen 240
12.1.2 Study endpoints 240
12.1.3 Treatment duration 240
12.1.4 Logistical considerations 241
12.1.5 Independent data monitoring
committee 241
12.2 Adaptive Group Sequential Design 241
12.2.1 Group sequential design 241
12.2.2 Adaptation 242
12.2.3 Statistical methods 243
12.2.4 Case study — an example 243
12.3 Adaptive Dose-Escalation Design 244
12.3.1 Traditional dose-escalation design 244
12.3.2 Adaptation 245
12.3.3 Statistical methods 245
12.3.4 Case study — an example 245
12.4 Adaptive Seamless Phase II/III Design 247
12.4.1 Seamless phase II/III design 247
12.4.2 Adaptation 248
12.4.3 Methods 248
12.4.4 Case study — some examples 249
12.4.5 Issues and recommendations 252
Bibliography 255
Index 269
|
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| id | DE-604.BV035907245 |
| illustrated | Illustrated |
| indexdate | 2024-07-09T22:07:09Z |
| institution | BVB |
| isbn | 1584887761 |
| language | English |
| lccn | 2006048510 |
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| physical | 277 S. graph. Darst. 24 cm |
| publishDate | 2007 |
| publishDateSearch | 2007 |
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| publisher | Chapman & Hall/CRC |
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| series | Chapman & Hall/CRC biostatistics series |
| series2 | Chapman & Hall/CRC biostatistics series |
| spelling | Chow, Shein-Chung Verfasser aut Adaptive design methods in clinical trials Shein-Chung Chow ; Mark Chang Boca Raton, Fla. Chapman & Hall/CRC 2007 277 S. graph. Darst. 24 cm txt rdacontent n rdamedia nc rdacarrier Chapman & Hall/CRC biostatistics series 17 Includes bibliographical references (p. [255]-267) and index "One of the first books on the topic, Adaptive Design Methods in Clinical Trials presents the principles and methodologies in adaptive design and analysis the pertain to adaptations made to trial or statistical procedures that are based on accrued data of ongoing clinical trials. The book also offers a well-balanced summary of current regulatory perspectives and recently developed statistical methods in this area."--Cover, p. 4. Adaptive sampling (Statistics) Biometry methods Clinical Trials as Topic methods Clinical trials Clinical trials Statistical methods Experimental design Models, Statistical Research Design Chang, Mark 1955- Verfasser (DE-588)1044405805 aut Chapman & Hall/CRC biostatistics series 17 (DE-604)BV023097394 17 http://www.loc.gov/catdir/enhancements/fy0710/2006048510-d.html Publisher description HBZ Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=018764584&sequence=000002&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Chow, Shein-Chung Chang, Mark 1955- Adaptive design methods in clinical trials Chapman & Hall/CRC biostatistics series Adaptive sampling (Statistics) Biometry methods Clinical Trials as Topic methods Clinical trials Clinical trials Statistical methods Experimental design Models, Statistical Research Design |
| title | Adaptive design methods in clinical trials |
| title_auth | Adaptive design methods in clinical trials |
| title_exact_search | Adaptive design methods in clinical trials |
| title_full | Adaptive design methods in clinical trials Shein-Chung Chow ; Mark Chang |
| title_fullStr | Adaptive design methods in clinical trials Shein-Chung Chow ; Mark Chang |
| title_full_unstemmed | Adaptive design methods in clinical trials Shein-Chung Chow ; Mark Chang |
| title_short | Adaptive design methods in clinical trials |
| title_sort | adaptive design methods in clinical trials |
| topic | Adaptive sampling (Statistics) Biometry methods Clinical Trials as Topic methods Clinical trials Clinical trials Statistical methods Experimental design Models, Statistical Research Design |
| topic_facet | Adaptive sampling (Statistics) Biometry methods Clinical Trials as Topic methods Clinical trials Clinical trials Statistical methods Experimental design Models, Statistical Research Design |
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