Introduction to statistical methods for clinical trials:
Gespeichert in:
| Format: | Buch |
|---|---|
| Sprache: | English |
| Veröffentlicht: |
Boca Raton
Chapman & Hall/CRC
2008
|
| Schriftenreihe: | Texts in statistical science series
[72] |
| Schlagworte: | |
| Online-Zugang: | Publisher description Inhaltsverzeichnis |
| Beschreibung: | Includes bibliographical references (p. 405-426) and index |
| Beschreibung: | XXIII, 439 S. Ill. |
| ISBN: | 9781584880271 1584880279 |
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Datensatz im Suchindex
| _version_ | 1804137933489307648 |
|---|---|
| adam_text | Contents
List of figures
xi
List of tables
xv
Preface
xix
Author Attribution
xxiii
1
Introduction to Clinical Trials
1
1.1
History and Background
3
1.2
Ethics of Clinical Research
5
1.3
Types of Research Design and Types of Trials
9
1.4
The Need for Clinical Trials
15
1.5
The Randomization Principle
18
1.6
Timing of a Clinical Trial
18
1.7
Trial Organization
20
1.8
Protocol and Manual of Operations
22
1.9
Regulatory Issues
22
1.10
Overview of the Book
26
2
Defining the Question
29
2.1
Statistical Framework
31
2.2
Elements of Study Question
40
2.3
Outcome or Response Measures
44
2.4
The Surrogate Outcome
56
2.5
Composite Outcomes
64
2.6
Summary
73
2.7
Problems
73
3
Study Design
75
3.1
Early Phase Trials
76
3.2
Phase III/IV Trials
85
3.3
Non-inferiority Designs
101
3.4
Screening. Prevention, and Therapeutic Designs
106
3.5
Adaptive Designs
109
3.6
Conclusions
112
viii CONTENTS
3.7 Problems 112
4 Sample
Size
115
4.1 Sample
Size
versus Information 116
4.2
A
General Setup
for
Frequentisi
Designs 118
4.3
Loss to Follow-up and Non-adherence
122
4.4
Survival Data
124
4.5
Clustered Data
134
4.6
Tests for Interaction
136
4.7
Equivalence/Non-inferiority Trials
137
4.8
Other Considerations
138
4.9
Problems
139
5
Randomization
141
5.1
The Role of Randomization
141
5.2
Fixed Randomization Procedures
148
5.3
Treatment- and Response-Adaptive Randomization Procedures
155
5.4
Covariate-Adaptive Randomization Procedures
161
5.5
Summary and Recommendations
165
5.6
Problems
168
6
Data Collection and Quality Control
171
6.1
Planning for Collection of Clinical Trial Data
172
6.2
Categories of Clinical Data
185
6.3
Data Quality Control
194
6.4
Conclusions
199
7
Survival Analysis
201
7.1
Background
201
7.2
Estimation of Survival Distributions
203
7.3
Comparison of Survival Distributions
213
7.4
Regression Models
219
7.5
Composite Outcomes
227
7.6
Summary
228
7.7
Problems
229
8
Longitudinal Data
231
8.1
A Clinical Longitudinal Data Example
232
8.2
The Subject-specific Model
234
8.3
Two-stage Estimation
237
8.4
The Random-effects. Subject-specific Model
242
8.5
The Population-average (Marginal) Model
246
8.6
Restricted Maximum Likelihood Estimation (REML)
252
8.7
Standard Errors
253
8.8
Testing
255
CONTENTS ix
8.9
Additional Levels of Clustering
258
8.10
Generalized Estimating Equations for Non-normal Data
260
8.11
Missing Data
263
8.12
Summary
264
9
Quality of Life
267
9.1
Defining QoL
268
9.2
Types of QoL Assessments
268
9.3
Selecting a QoL Instrument
271
9.4
Developing a QoL Instrument
273
9.5
Quality of Life Data
273
9.6
Analysis of QoL Data
275
9.7
Summary
286
10
Data Monitoring and Interim Analysis
289
10.1
Data and Safety Monitoring
290
10.2
Examples
292
10.3
The Repeated Testing Problem
293
10.4
Group Sequential Tests
, 299
10.5
Triangular Test
311
10.6
Curtailment Procedures
315
10.7
Inference Following Sequential Tests
322
10.8
Discussion
329
10.9
Problems
336
11
Selected Issues in the Analysis
339
11.1
Bias in the Analysis of Clinical Trial Data
339
11.2
Choice of Analysis Population
340
11.3
Missing Data
354
11.4
Subgroup Analyses
364
11.5
Multiple Testing Procedures
370
11.6
Summary
375
11.7
Problems
376
12
Closeout and Reporting
377
12.1
Closing Out a Trial
377
12.2
Reporting Trial Results
378
12.3
Problems
392
A Delta Method, Maximum Likelihood Theory, and Informa¬
tion
393
A.I Delta Method
393
A.2 Asymptotic Theory for Likelihood Based Inference
393
A.3 Hypothesis Testing
395
A.4 Computing the MLE
399
x
CONTENTS
Α.
5 Information 400
Α.
6 Brownian Motion 403
References
405
Index 427
|
| adam_txt |
Contents
List of figures
xi
List of tables
xv
Preface
xix
Author Attribution
xxiii
1
Introduction to Clinical Trials
1
1.1
History and Background
3
1.2
Ethics of Clinical Research
5
1.3
Types of Research Design and Types of Trials
9
1.4
The Need for Clinical Trials
15
1.5
The Randomization Principle
18
1.6
Timing of a Clinical Trial
18
1.7
Trial Organization
20
1.8
Protocol and Manual of Operations
22
1.9
Regulatory Issues
22
1.10
Overview of the Book
26
2
Defining the Question
29
2.1
Statistical Framework
31
2.2
Elements of Study Question
40
2.3
Outcome or Response Measures
44
2.4
The Surrogate Outcome
56
2.5
Composite Outcomes
64
2.6
Summary
73
2.7
Problems
73
3
Study Design
75
3.1
Early Phase Trials
76
3.2
Phase III/IV Trials
85
3.3
Non-inferiority Designs
101
3.4
Screening. Prevention, and Therapeutic Designs
106
3.5
Adaptive Designs
109
3.6
Conclusions
112
viii CONTENTS
3.7 Problems 112
4 Sample
Size
115
4.1 Sample
Size
versus Information 116
4.2
A
General Setup
for
Frequentisi
Designs 118
4.3
Loss to Follow-up and Non-adherence
122
4.4
Survival Data
124
4.5
Clustered Data
134
4.6
Tests for Interaction
136
4.7
Equivalence/Non-inferiority Trials
137
4.8
Other Considerations
138
4.9
Problems
139
5
Randomization
141
5.1
The Role of Randomization
141
5.2
Fixed Randomization Procedures
148
5.3
Treatment- and Response-Adaptive Randomization Procedures
155
5.4
Covariate-Adaptive Randomization Procedures
161
5.5
Summary and Recommendations
165
5.6
Problems
168
6
Data Collection and Quality Control
171
6.1
Planning for Collection of Clinical Trial Data
172
6.2
Categories of Clinical Data
185
6.3
Data Quality Control
194
6.4
Conclusions
199
7
Survival Analysis
201
7.1
Background
201
7.2
Estimation of Survival Distributions
203
7.3
Comparison of Survival Distributions
213
7.4
Regression Models
219
7.5
Composite Outcomes
227
7.6
Summary
228
7.7
Problems
229
8
Longitudinal Data
231
8.1
A Clinical Longitudinal Data Example
232
8.2
The Subject-specific Model
234
8.3
Two-stage Estimation
237
8.4
The Random-effects. Subject-specific Model
242
8.5
The Population-average (Marginal) Model
246
8.6
Restricted Maximum Likelihood Estimation (REML)
252
8.7
Standard Errors
253
8.8
Testing
255
CONTENTS ix
8.9
Additional Levels of Clustering
258
8.10
Generalized Estimating Equations for Non-normal Data
260
8.11
Missing Data
263
8.12
Summary
264
9
Quality of Life
267
9.1
Defining QoL
268
9.2
Types of QoL Assessments
268
9.3
Selecting a QoL Instrument
271
9.4
Developing a QoL Instrument
273
9.5
Quality of Life Data
273
9.6
Analysis of QoL Data
275
9.7
Summary
286
10
Data Monitoring and Interim Analysis
289
10.1
Data and Safety Monitoring
290
10.2
Examples
292
10.3
The Repeated Testing Problem
293
10.4
Group Sequential Tests
, 299
10.5
Triangular Test
311
10.6
Curtailment Procedures
315
10.7
Inference Following Sequential Tests
322
10.8
Discussion
329
10.9
Problems
336
11
Selected Issues in the Analysis
339
11.1
Bias in the Analysis of Clinical Trial Data
339
11.2
Choice of Analysis Population
340
11.3
Missing Data
354
11.4
Subgroup Analyses
364
11.5
Multiple Testing Procedures
370
11.6
Summary
375
11.7
Problems
376
12
Closeout and Reporting
377
12.1
Closing Out a Trial
377
12.2
Reporting Trial Results
378
12.3
Problems
392
A Delta Method, Maximum Likelihood Theory, and Informa¬
tion
393
A.I Delta Method
393
A.2 Asymptotic Theory for Likelihood Based Inference
393
A.3 Hypothesis Testing
395
A.4 Computing the MLE
399
x
CONTENTS
Α.
5 Information 400
Α.
6 Brownian Motion 403
References
405
Index 427 |
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| illustrated | Illustrated |
| index_date | 2024-07-02T21:44:10Z |
| indexdate | 2024-07-09T21:20:10Z |
| institution | BVB |
| isbn | 9781584880271 1584880279 |
| language | English |
| lccn | 2007036202 |
| oai_aleph_id | oai:aleph.bib-bvb.de:BVB01-016681347 |
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| physical | XXIII, 439 S. Ill. |
| publishDate | 2008 |
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| publisher | Chapman & Hall/CRC |
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| series | Texts in statistical science series |
| series2 | Texts in statistical science series |
| spelling | Introduction to statistical methods for clinical trials ed. by Thomas D. Cook... Boca Raton Chapman & Hall/CRC 2008 XXIII, 439 S. Ill. txt rdacontent n rdamedia nc rdacarrier Texts in statistical science series [72] Includes bibliographical references (p. 405-426) and index Clinical trials Statistical methods Clinical Trials as Topic methods Statistics as Topic methods Data Collection methods Research Design Klinisches Experiment (DE-588)4164223-5 gnd rswk-swf Methode (DE-588)4038971-6 gnd rswk-swf Statistik (DE-588)4056995-0 gnd rswk-swf Klinisches Experiment (DE-588)4164223-5 s Statistik (DE-588)4056995-0 s Methode (DE-588)4038971-6 s b DE-604 Cook, Thomas D. Sonstige oth Texts in statistical science series [72] (DE-604)BV022819715 72 http://www.loc.gov/catdir/enhancements/fy0826/2007036202-d.html Publisher description Digitalisierung UB Regensburg application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=016681347&sequence=000006&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Introduction to statistical methods for clinical trials Texts in statistical science series Clinical trials Statistical methods Clinical Trials as Topic methods Statistics as Topic methods Data Collection methods Research Design Klinisches Experiment (DE-588)4164223-5 gnd Methode (DE-588)4038971-6 gnd Statistik (DE-588)4056995-0 gnd |
| subject_GND | (DE-588)4164223-5 (DE-588)4038971-6 (DE-588)4056995-0 |
| title | Introduction to statistical methods for clinical trials |
| title_auth | Introduction to statistical methods for clinical trials |
| title_exact_search | Introduction to statistical methods for clinical trials |
| title_exact_search_txtP | Introduction to statistical methods for clinical trials |
| title_full | Introduction to statistical methods for clinical trials ed. by Thomas D. Cook... |
| title_fullStr | Introduction to statistical methods for clinical trials ed. by Thomas D. Cook... |
| title_full_unstemmed | Introduction to statistical methods for clinical trials ed. by Thomas D. Cook... |
| title_short | Introduction to statistical methods for clinical trials |
| title_sort | introduction to statistical methods for clinical trials |
| topic | Clinical trials Statistical methods Clinical Trials as Topic methods Statistics as Topic methods Data Collection methods Research Design Klinisches Experiment (DE-588)4164223-5 gnd Methode (DE-588)4038971-6 gnd Statistik (DE-588)4056995-0 gnd |
| topic_facet | Clinical trials Statistical methods Clinical Trials as Topic methods Statistics as Topic methods Data Collection methods Research Design Klinisches Experiment Methode Statistik |
| url | http://www.loc.gov/catdir/enhancements/fy0826/2007036202-d.html http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=016681347&sequence=000006&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA |
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