Ebook: Cancer Clinical Trials: A Critical Appraisal

The controlled clinical trial has become an essential part of the clinician's decision-making process. Clinical trials, however, still raise methodological problems that are important and at the same time controversial: subgroup analysis and interactions, meta-analy­ sis of similar trials, consideration of subjective clinical opinions and those of the public at large, assessment of quality of life, pre­ vention trials, and so on. In February 1987 we took our third step along the road to evaluating these issues in dialogues between cli­ nicians, psychologists, legal experts, and statisticians. The talks presented at the meeting were revised by the authors afterwards and have been rearranged by the editors to form a strictly organ­ 1 2 ized book. The two preceding meetings in 1978 and 1981 focused strongly on adjuvant therapy in primary breast cancer, but this top­ ic served merely as a nucleus in the third meeting. This meeting, although called the Third Heidelberg Symposium was forced to leave Heidelberg and in fact was held in Freiburg. Without the interest and enthusiasm of Professor Martin Schu­ macher and his colleagues in Freiburg the meeting would never have taken place. The meeting was generously supported again by the Federal Ministry of Research and Technology (Bundesministe­ rium flir Forschung und Technologie, BMFT) within the framework of the West German BMFT Breast Cancer Study Group. We are grateful, in particular, to Mr. Hans W. Herzog for his personal in­ volvement. Juni 1988 H. Scheurlen, R. Kay, M.

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The book is devoted to improved communication between clinicians, psychologists, and legal experts on the one hand and methodologists on the other. Mathematical formalism is therefore kept to a minimum and on a level acceptable to all sides. The contributions provide strictly organized coverage of the following topics: scientific fundamentals; heterogeneous treatment effects; combined information in historical controls, overviews, and comprehensive cohort studies; subjective opinions and informed consent; and assessment of quality of life. It is these aspects that are crucial in the planning of clinical trials and in the analysis and interpretation of results. Since there is still much debate between clinicians and statisticians on the subject, this book may prevent the reader from embarking on poorly planned clinical trials.

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The book is devoted to improved communication between clinicians, psychologists, and legal experts on the one hand and methodologists on the other. Mathematical formalism is therefore kept to a minimum and on a level acceptable to all sides. The contributions provide strictly organized coverage of the following topics: scientific fundamentals; heterogeneous treatment effects; combined information in historical controls, overviews, and comprehensive cohort studies; subjective opinions and informed consent; and assessment of quality of life. It is these aspects that are crucial in the planning of clinical trials and in the analysis and interpretation of results. Since there is still much debate between clinicians and statisticians on the subject, this book may prevent the reader from embarking on poorly planned clinical trials.
Content:
Front Matter....Pages I-XI
The Crooked Path Toward the Objectivation of Therapeutic Experience....Pages 1-5
Randomised Trials: The Case for Science in Medicine....Pages 6-17
A Critical Assessment of Approaches to Improving the Efficiency of Cancer Clinical Trials....Pages 18-26
Statistical Standards for Protocols and Protocol Deviations....Pages 27-33
The Design of Cancer Prevention Trials....Pages 34-48
Strategies for Subgroup Analysis in Clinical Trials....Pages 49-54
Statistical Tools for Subset Analysis in Clinical Trials....Pages 55-66
Competing Risks, Interactions and Crossing Hazards....Pages 67-74
Significance Testing in the Comparison of Survival Curves from Clinical Trials of Cancer Treatment....Pages 75-81
Estimating the Magnitude of Benefits from Adjuvant Therapy....Pages 82-86
Treatment by Protocol: Assessment of Gains and Losses....Pages 87-94
The Use of Data Bases and Historical Controls in Treatment Comparisons....Pages 95-98
Limitations and Advantages of Meta-analysis in Clinical Trials....Pages 99-104
Overview of Adjuvant Radiotherapy for Breast Cancer....Pages 105-107
Overview of Randomized Trials of Postoperative Adjuvant Radiotherapy in Breast Cancer....Pages 108-129
Combining Randomized and Nonrandomized Patients in the Statistical Analysis of Clinical Trials....Pages 130-137
The Comprehensive Cohort Study: The Use of Registry Data to Confirm and Extend a Randomized Trial....Pages 138-148
Treatment Decisions in Breast Cancer....Pages 149-170
Randomization and Follow-up Care of Patients in a Breast Cancer Clinical Trial: Personal Experience....Pages 171-173
Alternative Treatments for Small Cancers of the Breast: Motives and Preferences of Patients Having to Choose....Pages 174-178
Subjective Theories on Etiology and Treatment of Cancer....Pages 179-184
Individual Cases and the Scientific Method — A Conflict? Legal Aspects of Cancer Clinical Trials in the Federal Republic of Germany....Pages 185-190
The Value of Small Clinical Trials....Pages 191-194
A Predictive Approach to Selecting the Size of a Clinical Trial, Based on Subjective Clinical Opinion....Pages 195-206
Statistical Properties and Use of Sequential Methods in Randomized Clinical Trials when the Response Criterion Is Censored....Pages 207-215
Measurement of Quality of Life in Clinical Trials of Therapy....Pages 216-230
A Modular Approach to Quality-of-Life Assessment in Cancer Clinical Trials....Pages 231-249
Effects of Breast Conservation on Psychological Morbidity Associated with Diagnosis and Treatment of Early Breast Cancer....Pages 250-257
Evaluation of Psychosocial Aspects in a Breast Preservation Trial....Pages 258-269
Back Matter....Pages 270-272

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The book is devoted to improved communication between clinicians, psychologists, and legal experts on the one hand and methodologists on the other. Mathematical formalism is therefore kept to a minimum and on a level acceptable to all sides. The contributions provide strictly organized coverage of the following topics: scientific fundamentals; heterogeneous treatment effects; combined information in historical controls, overviews, and comprehensive cohort studies; subjective opinions and informed consent; and assessment of quality of life. It is these aspects that are crucial in the planning of clinical trials and in the analysis and interpretation of results. Since there is still much debate between clinicians and statisticians on the subject, this book may prevent the reader from embarking on poorly planned clinical trials.
Content:
Front Matter....Pages I-XI
The Crooked Path Toward the Objectivation of Therapeutic Experience....Pages 1-5
Randomised Trials: The Case for Science in Medicine....Pages 6-17
A Critical Assessment of Approaches to Improving the Efficiency of Cancer Clinical Trials....Pages 18-26
Statistical Standards for Protocols and Protocol Deviations....Pages 27-33
The Design of Cancer Prevention Trials....Pages 34-48
Strategies for Subgroup Analysis in Clinical Trials....Pages 49-54
Statistical Tools for Subset Analysis in Clinical Trials....Pages 55-66
Competing Risks, Interactions and Crossing Hazards....Pages 67-74
Significance Testing in the Comparison of Survival Curves from Clinical Trials of Cancer Treatment....Pages 75-81
Estimating the Magnitude of Benefits from Adjuvant Therapy....Pages 82-86
Treatment by Protocol: Assessment of Gains and Losses....Pages 87-94
The Use of Data Bases and Historical Controls in Treatment Comparisons....Pages 95-98
Limitations and Advantages of Meta-analysis in Clinical Trials....Pages 99-104
Overview of Adjuvant Radiotherapy for Breast Cancer....Pages 105-107
Overview of Randomized Trials of Postoperative Adjuvant Radiotherapy in Breast Cancer....Pages 108-129
Combining Randomized and Nonrandomized Patients in the Statistical Analysis of Clinical Trials....Pages 130-137
The Comprehensive Cohort Study: The Use of Registry Data to Confirm and Extend a Randomized Trial....Pages 138-148
Treatment Decisions in Breast Cancer....Pages 149-170
Randomization and Follow-up Care of Patients in a Breast Cancer Clinical Trial: Personal Experience....Pages 171-173
Alternative Treatments for Small Cancers of the Breast: Motives and Preferences of Patients Having to Choose....Pages 174-178
Subjective Theories on Etiology and Treatment of Cancer....Pages 179-184
Individual Cases and the Scientific Method — A Conflict? Legal Aspects of Cancer Clinical Trials in the Federal Republic of Germany....Pages 185-190
The Value of Small Clinical Trials....Pages 191-194
A Predictive Approach to Selecting the Size of a Clinical Trial, Based on Subjective Clinical Opinion....Pages 195-206
Statistical Properties and Use of Sequential Methods in Randomized Clinical Trials when the Response Criterion Is Censored....Pages 207-215
Measurement of Quality of Life in Clinical Trials of Therapy....Pages 216-230
A Modular Approach to Quality-of-Life Assessment in Cancer Clinical Trials....Pages 231-249
Effects of Breast Conservation on Psychological Morbidity Associated with Diagnosis and Treatment of Early Breast Cancer....Pages 250-257
Evaluation of Psychosocial Aspects in a Breast Preservation Trial....Pages 258-269
Back Matter....Pages 270-272
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