Catalogue


Fundamentals of clinical research : bridging medicine, statistics, and operations /
Antonella Bacchieri, Giovanni Della Cioppa.
imprint
Milano : Springer, c2007.
description
xxv, 343 p. : ill. ; 25 cm.
ISBN
8847004918, 9788847004917
format(s)
Book
Holdings
More Details
imprint
Milano : Springer, c2007.
isbn
8847004918
9788847004917
catalogue key
6200603
 
Includes bibliographical references (p. [331]-335) and index.
A Look Inside
About the Author
Author Affiliation
Antonella Bacchieri as assistant professor at the Department of Statistical Sciences of the University of Padua
Reviews
Review Quotes
From the reviews:"The first three chapters of the book provide important foundational information. ...Chapters 4, 6, and 7 are the stars of this book. The authors lay out in great detail the complex process of arriving at a measure of treatment effect. We statisticians are so used to having this handed to us that we never think about how important this process is. The authors also provide pragmatic advice about whether to select your sample to be mildly ill or seriously ill, or whether you should choose healthy volunteers instead." Steve Simon, Journal of Biopharmaceutical Statistics, Issue 2, 2008"Main aim of this book is to explain the three pillars of clinical research: medicine, statistics and logistics in an easily digestible manner. … A very lucid and well-structured account of the basic statistical and epidemiological concepts is definitely a great plus point of this book … . I have truly enjoyed reading this book and it gives a refreshing and focused outline of the clinical research. I would recommend all clinicians who aspire to be/are involved in clinical research to give it an audience." (Faisel Yunus, Journal of Applied Statistics, Vol. 35 (2), 2008)"Readership: … the clinical researcher who wants an intelligent discussion on the (mainly) statistical aspects of clinical research. It would make a useful background reading for a course in clinical trials. … The book is well written. … is essentially an extended essay on the application of statistical inference in clinical research." (M. J. Campbell, International Statistical Review, Vol. 75 (3), 2007)"A comprehensive introductory text that addresses statistical methods and operational issues in the conduct of clinical research. ... The authors avoid overuse of mathematical formulas and carefully balance practical and theoretical aspects of clinical research. Format and writing clarity are good, with numerous examples used to illustrate concepts throughout the text. ... Recommended Readership: Doctoral and postdoctoral trainees and junior or research inexperienced clinical researchers and scientists." (Paul V. Targonslci, Mayo Clinic Proceedings, Vol. 82 (11), November, 2007)"The text is directed toward physicians conducting clinical trials research and statisticians consulting on clinical trials. … I recommend it for physicians, statisticians, and technical personnel with a novice knowledge level of clinical trials. At the same time it could be used as a text for an introductory graduate level class in clinical trials. To those entering the field of clinical trials I wholeheartedly recommend Fundamentals of Clinical Research … as it provides a foundation pertinent to a variety of aspects of clinical trials research." (Gregory E. Gilbert, The American Statistician, Vol. 63 (1), February, 2009)
From the reviews: "The first three chapters of the book provide important foundational information. ...Chapters 4, 6, and 7 are the stars of this book. The authors lay out in great detail the complex process of arriving at a measure of treatment effect. We statisticians are so used to having this handed to us that we never think about how important this process is. The authors also provide pragmatic advice about whether to select your sample to be mildly ill or seriously ill, or whether you should choose healthy volunteers instead." Steve Simon, Journal of Biopharmaceutical Statistics, Issue 2, 2008 "Main aim of this book is to explain the three pillars of clinical research: medicine, statistics and logistics in an easily digestible manner. ... A very lucid and well-structured account of the basic statistical and epidemiological concepts is definitely a great plus point of this book ... . I have truly enjoyed reading this book and it gives a refreshing and focused outline of the clinical research. I would recommend all clinicians who aspire to be/are involved in clinical research to give it an audience." (Faisel Yunus, Journal of Applied Statistics, Vol. 35 (2), 2008) "Readership: ... the clinical researcher who wants an intelligent discussion on the (mainly) statistical aspects of clinical research. It would make a useful background reading for a course in clinical trials. ... The book is well written. ... is essentially an extended essay on the application of statistical inference in clinical research." (M. J. Campbell, International Statistical Review, Vol. 75 (3), 2007) "A comprehensive introductory text that addresses statistical methods and operational issues in the conduct of clinical research. ... The authors avoid overuse of mathematical formulas and carefully balance practical and theoretical aspects of clinical research. Format and writing clarity are good, with numerous examples used to illustrate concepts throughout the text. ... Recommended Readership: Doctoral and postdoctoral trainees and junior or research inexperienced clinical researchers and scientists." (Paul V. Targonslci, Mayo Clinic Proceedings, Vol. 82 (11), November, 2007) "The text is directed toward physicians conducting clinical trials research and statisticians consulting on clinical trials. ... I recommend it for physicians, statisticians, and technical personnel with a novice knowledge level of clinical trials. At the same time it could be used as a text for an introductory graduate level class in clinical trials. To those entering the field of clinical trials I wholeheartedly recommend Fundamentals of Clinical Research ... as it provides a foundation pertinent to a variety of aspects of clinical trials research." (Gregory E. Gilbert, The American Statistician, Vol. 63 (1), February, 2009) "The authors' book on methods and issues in clinical research is directed at biomedical researchers as well as statisticians. ... it addresses the integration of medical and statistical components of clinical research in order to create a logical flow of reasoning understandable for both the medical/biological and the statistical expert. ... The whole book is written in a convenient, comprehensible style ... and is therefore useful also for readers having more advanced knowledge and experience in the field of clinical trials." (Lutz Edler, Zentralblatt MATH, Vol. 1210, 2011)
"The first three chapters of the book provide important foundational information. ...Chapters 4, 6, and 7 are the stars of this book. The authors lay out in great detail the complex process of arriving at a measure of treatment effect. We statisticians are so used to having this handed to us that we never think about how important this process is. The authors also provide pragmatic advice about whether to select your sample to be mildly ill or seriously ill, or whether you should choose healthy volunteers instead." Steve Simon, Journal of Biopharmaceutical Statistics, Issue 2, 2008
To find out how to look for other reviews, please see our guides to finding book reviews in the Sciences or Social Sciences and Humanities.
Summaries
Back Cover Copy
The scope of clinical research is to evaluate the effect of a treatment on the evolution of a disease in the human species.The treatment can be pharmacological, surgical, psychological/behavioral or organizational/logistic. The disease, intended as an impairment of a state of well-being or a condition capable of provoking such impairment over time, can be universally accepted as such (e.g. a cancer or a bone fracture) or perceived as such only by limited groups of individuals in a given cultural context (e.g. hair loss or weight gain). The course of the disease that ones wishes to change can be the one with no intervention or, more frequently, the one observed with the available treatment. The evaluation of the effect of a treatment on the course of a disease is a lengthy process, which progresses in increasingly complex stages. A detailed coverage of the logistic, administrative and legal aspects of clinical research is outside the scope of this book. However, throughout the book we keep reminding the reader of these aspects because, as already mentioned, we firmly believe they have a crucial role in determining the success of a study. The history of clinical research is paved with relics of studies started with great pomp, riding great ideas and great hopes, which drowned miserably because of inadequate logistical preparation. In our experience, the excessive complexity of a clinical trial is the single most frequent cause of failure: the study is perfect on paper, but impossible to implement by patients and staff alike. The distance between the principal investigators and the reality of clinical research in its day-to-day practice is often the main cause of such disasters. We warmly encourage everyone involved in clinical research to get involved in the logistics of a study, learning from colleagues responsible for its practical conduct (clinical research associates, data managers, etc.) and to take part, in person, in the practical implementation of a trial before attempting to design a study protocol. The book ends with a brief description of the drug development process and to the phases of clinical development. The authors
Back Cover Copy
The scope of clinical research is to evaluate the effect of a treatment on the evolution of a disease in the human species.The treatment can be pharmacological, surgical, psychological/behavioral or organizational/logistic. The disease, intended as an impairment of a state of well-being or a condition capable of provoking such impairment over time, can be universally accepted as such (e.g. a cancer or a bone fracture) or perceived as such only by limited groups of individuals in a given cultural context (e.g. hair loss or weight gain). The course of the disease that ones wishes to change can be the one with no intervention or, more frequently, the one observed with the available treatment. The evaluation of the effect of a treatment on the course of a disease is a lengthy process, which progresses in increasingly complex stages.A detailed coverage of the logistic, administrative and legal aspects of clinical research is outside the scope of this book. However, throughout the book we keep reminding the reader of these aspects because, as already mentioned, we firmly believe they have a crucial role in determining the success of a study. The history of clinical research is paved with relics of studies started with great pomp, riding great ideas and great hopes, which drowned miserably because of inadequate logistical preparation. In our experience, the excessive complexity of a clinical trial is the single most frequent cause of failure: the study is perfect on paper, but impossible to implement by patients and staff alike. The distance between the principal investigators and the reality of clinical research in its day-to-day practice is often the main cause of such disasters. We warmly encourage everyone involved in clinical research to get involved in the logistics of a study, learning from colleagues responsible for its practical conduct (clinical research associates, data managers, etc.) and to take part, in person, in the practical implementation of a trial before attempting to design a study protocol.The book ends with a brief description of the drug development process and to the phases of clinical development. The authors
Bowker Data Service Summary
The scope of clinical research is to evaluate the effect of a treatment on the evolution of a disease in the human species. This book embraces the three pillars of clinical research - medicine, statistics and operations - systematically covering the building blocks of a clinical study.
Long Description
In recent years many introductory textbooks on clinical trial methodology have been published, some of which are excellent, in addition to a very extensive specialist literature. Nevertheless, here is a new book on methods and issues in clinical research. The objectives can be summarized in three points. 1. Integrate medical and statistical components of clinical research. 2. Do justice to the operational and practical requirements of clinical research. 3. Give space to the ethical implications of methodological issues in clinical research. The scope of clinical research is to evaluate the effect of a treatment on the evolution of a disease in the human species. The treatment can be pharmacological, surgical, psychological/behavioral or organizational/logistic. The disease, intended as an impairment of a state of well-being or a condition capable of provoking such impairment over time, can be universally accepted as such (e.g. a cancer or a bone fracture) or perceived as such only by limited groups of individuals in a given cultural context (e.g. hair loss or weight gain). The course of the disease that ones wishes to change can be the one with no intervention or, more frequently, the one observed with the available treatment. The evaluation of the effect of a treatment on the course of a disease is a lengthy process, which progresses in increasingly complex stages. A detailed coverage of the logistic, administrative and legal aspects of clinical research is outside the scope of this book. However, throughout the book we keep reminding the reader of these aspects because, as already mentioned, we firmly believe they have a crucial role in determining the success of a study. Thehistory of clinical research is paved with relics of studies started with great pomp, riding great ideas and great hopes, which drowned miserably because of inadequate logistical preparation. In our experience, the excessive complexity of a clinical trial is the single most frequent cause of failure: the study is perfect on paper, but impossible to implement by patients and staff alike. The distance between the principal investigators and the reality of clinical research in its day-to-day practice is often the main cause of such disasters. We warmly encourage everyone involved in clinical research to get involved in the logistics of a study, learning from colleagues responsible for its practical conduct (clinical research associates, data managers, etc.) and to take part, in person, in the practical implementation of a trial before attempting to design a study protocol. The book ends with a brief description of the drug development process and to the phases of clinical development.
Long Description
In recent years many introductory textbooks on clinical trial methodology have been published, some of which are excellent, in addition to a very extensive specialist literature. Nevertheless, here is a new book on methods and issues in clinical research. The objectives can be summarized in three points. 1. Integrate medical and statistical components of clinical research. 2. Do justice to the operational and practical requirements of clinical research. 3. Give space to the ethical implications of methodological issues in clinical research. The scope of clinical research is to evaluate the effect of a treatment on the evolution of a disease in the human species. The treatment can be pharmacological, surgical, psychological/behavioral or organizational/logistic. The disease, intended as an impairment of a state of well-being or a condition capable of provoking such impairment over time, can be universally accepted as such (e.g. a cancer or a bone fracture) or perceived as such only by limited groups of individuals in a given cultural context (e.g. hair loss or weight gain). The course of the disease that ones wishes to change can be the one with no intervention or, more frequently, the one observed with the available treatment. The evaluation of the effect of a treatment on the course of a disease is a lengthy process, which progresses in increasingly complex stages. A detailed coverage of the logistic, administrative and legal aspects of clinical research is outside the scope of this book. However, throughout the book we keep reminding the reader of these aspects because, as already mentioned, we firmly believe they have a crucial role in determining the success of a study. The history of clinical research is paved with relics of studies started with great pomp, riding great ideas and great hopes, which drowned miserably because of inadequate logistical preparation. In our experience, the excessive complexity of a clinical trial is the single most frequent cause of failure: the study is perfect on paper, but impossible to implement by patients and staff alike. The distance between the principal investigators and the reality of clinical research in its day-to-day practice is often the main cause of such disasters. We warmly encourage everyone involved in clinical research to get involved in the logistics of a study, learning from colleagues responsible for its practical conduct (clinical research associates, data managers, etc.) and to take part, in person, in the practical implementation of a trial before attempting to design a study protocol. The book ends with a brief description of the drug development process and to the phases of clinical development.
Long Description
In recent years many introductory textbooks on clinical trial methodology have been published, some of which are excellent, in addition to a very extensive specialist literature. Nevertheless, here is a new book on methods and issues in clinical research. The objectives can be summarized in three points. 1. Integrate medical and statistical components of clinical research. 2. Do justice to the operational and practical requirements of clinical research. 3. Give space to the ethical implications of methodological issues in clinical research.The scope of clinical research is to evaluate the effect of a treatment on the evolution of a disease in the human species. The treatment can be pharmacological, surgical, psychological/behavioral or organizational/logistic. The disease, intended as an impairment of a state of well-being or a condition capable of provoking such impairment over time, can be universally accepted as such (e.g. a cancer or a bone fracture) or perceived as such only by limited groups of individuals in a given cultural context (e.g. hair loss or weight gain). The course of the disease that ones wishes to change can be the one with no intervention or, more frequently, the one observed with the available treatment. The evaluation of the effect of a treatment on the course of a disease is a lengthy process, which progresses in increasingly complex stages.A detailed coverage of the logistic, administrative and legal aspects of clinical research is outside the scope of this book. However, throughout the book we keep reminding the reader of these aspects because, as already mentioned, we firmly believe they have a crucial role in determining the success of a study. The history of clinical research is paved with relics of studies started with great pomp, riding great ideas and great hopes, which drowned miserably because of inadequate logistical preparation. In our experience, the excessive complexity of a clinical trial is the single most frequent cause of failure: the study is perfect on paper, but impossible to implement by patients and staff alike. The distance between the principal investigators and the reality of clinical research in its day-to-day practice is often the main cause of such disasters. We warmly encourage everyone involved in clinical research to get involved in the logistics of a study, learning from colleagues responsible for its practical conduct (clinical research associates, data managers, etc.) and to take part, in person, in the practical implementation of a trial before attempting to design a study protocol.The book ends with a brief description of the drug development process and to the phases of clinical development.
Main Description
Here is a new book on methods and issues in clinical research. Its objectives can be summarized in three points. 1. Integrate medical and statistical components of clinical research. 2. Do justice to the operational and practical requirements of clinical research. 3. Give space to the ethical implications of methodological issues in clinical research. The book ends with a brief description of the drug development process and the phases of clinical development.
Main Description
This book embraces the three pillars of clinical research: medicine, statistics and operations ('operations' is the logistics of research), and systematically covers the building blocks of a clinical study. The authors, a statistician and a physician, have many years of hands-on experience in the planning, conduct, analysis, interpretation and reporting of clinical trials. In writing this book, they had two main objectives. First, create a language and a logical flow of reasoning that, while precise and rigorous, are understandable to both the physician/biologist and the statistician. This common platform will help a constructive interaction between the two disciplines, which is absolutely crucial for good study plans to be generated. Second, do justice to operations, which is the discipline - and art - of making it all happen. Scientists often neglect operations and generate study plans which may be scientifically and methodologically brilliant, but fail miserably, because they are unfeasible in the real life settings where studies are conducted. Throughout the book the reader is confronted with the practical reality of clinical research.
Table of Contents
Forewordp. VII
Acknowledgementsp. XI
Contentsp. XIII
Introductionp. XIX
Variability of Biological Phenomena and Measurement Errorsp. 1
Phenotypic Variabilityp. 1
Temporal Variabilityp. 2
Measurement-Related Variabilityp. 3
The Measurementp. 3
Measurement Errorsp. 7
Variability of Diagnostic Testsp. 10
Summaryp. 13
Distinctive Aspects of a Biomedical Study. Observational and Experimental Studiesp. 14
Distinctive Features of Biomedical Studiesp. 14
The Study Protocolp. 18
Observational Studiesp. 21
Experimental Studiesp. 23
Summaryp. 27
Observational Studiesp. 28
Basic Designs of Observational Studiesp. 29
Prospective or Cohort Studiesp. 30
Retrospective Studies or Control Casesp. 36
Sample Sizep. 40
Bias and Confoundingp. 40
Control of Bias in Epidemiologyp. 44
Control of the Phenomenon of Confoundingp. 49
Advantages and Disadvantages of the Different Types of Observational Studiesp. 53
Summaryp. 56
Defining the Treatment Effectp. 58
From the Single Measurement to the Signalp. 58
Identification and Quantification of the End-Points (Individual Subject Level)p. 64
Methodological Characteristics of the End-Pointp. 64
Discriminating between Primary and Secondary End-Points and between Efficacy and Safety/Tolerability End-Pointsp. 66
Identification and Quantification of the Signals (Group Level)p. 69
Statistical Considerationsp. 70
Practical, Regulatory, Marketing and Pharmaco-Economic Considerationsp. 73
Selection and Characterization of the Primary End-Point and Signal: an Examplep. 75
Stage One: Define the Main Therapeutic Levelp. 75
Stage Two: Define the Primary End-Point (Individual Patient Level)p. 77
Stages Three and Four: Define the Group Indicator, the Signal, and the Threshold of Clinical Relevance (Treatment Group and Study Levels)p. 79
More Than One Question in the Same Study: the Problem of Multiple Statistical Testsp. 80
Validation of Measurement Scalesp. 84
Special Types of End-Pointsp. 85
Summaryp. 88
Probability, Inference and Decision Makingp. 90
Probabilityp. 91
Definitionsp. 91
Probability Distribution and Probability Density Functionp. 92
Normal or Gaussian Distributionp. 99
Basic Concepts of Inferencep. 100
Hypothesis Testing and Statistical Formulation of the Medical Questionp. 103
Statistical Estimation as the Tool for Evaluation of Clinical Relevancep. 105
Statistical Inference in the Frequentist and the Bayesian Approachesp. 106
Two Digressions: Measures of Variability and Likelihood Functionp. 110
Measures of Variabilityp. 110
Likelihood Functionp. 114
Frequentist (Classical) Analysis of a Clinical Trialp. 119
Hypothesis Testing: the Frequentist Solutionp. 120
Estimation of the Effect: the Frequentist Solutionp. 135
Bayesian Analysis of a Clinical Trialp. 138
Hypothesis Testing: the Bayesian Solutionp. 138
Estimation of the Effect: the Bayesian Solutionp. 144
Some Additional Considerations on the Frequentist and Bayesian Approachesp. 146
Parametric and Non-Parametric Inferencep. 149
Statistical Decision Making in the Medical Fieldp. 150
Evidence-Based Medicinep. 152
Summaryp. 154
The Choice of the Samplep. 157
Which Subjects Should Form the Sample?p. 157
Characteristics of the Patients to be Enrolled in the Studyp. 157
Mechanism of Subject Selectionp. 163
How Many Subjects Should Form the Sample?p. 164
Statistical Considerationsp. 164
Medical and Practical Aspectsp. 169
Summaryp. 171
The Choice of Treatmentsp. 172
Study Treatmentsp. 172
How Many Treatmentsp. 175
What Treatmentsp. 176
Blinding of the Study Treatmentsp. 178
Packaging and Logisticsp. 178
Concomitant Treatmentsp. 180
Summaryp. 182
Experimental Design: Fallacy of "Before-After" Comparisons in Uncontrolled Studiesp. 183
Experimental Design: Introductory Conceptsp. 183
Before-After Comparison in a Single Group of Subjectsp. 185
Temporal Variations of the Diseasep. 186
Temporal Variations of Staff, Equipment and Environmentp. 188
Statistical Regression Toward the Meanp. 189
The Basic Principlep. 189
Areas of Biomedical Experiments Affected by Regression Toward the Meanp. 191
How to Minimize the Effect of Regression Toward the Meanp. 193
Learning Effectp. 195
Psychological Effectp. 196
The Before-After Design Without Control Group in Oncologyp. 197
Summaryp. 198
Experimental Design: the Randomized Blinded Study as an Instrument to Reduce Biasp. 200
Introductionp. 200
Randomization as Antidote Against Selection Biasp. 203
Definition and Conceptual Frameworkp. 203
Types of Randomizationp. 206
Other Methods for Assigning Patients to Treatmentsp. 215
Blinding of Treatments as Antidote Against Assessment Biasp. 216
A Priori Definition of the Statistical Methods and Populations as Antidote Against the Analysis Biasp. 221
Methods of Statistical Analysisp. 221
Analysis Populationsp. 222
Comparison Between an Observational and an Experimental Studyp. 224
Summaryp. 227
Experimenta Designsp. 228
Introductionp. 228
Parallel Group Designp. 233
Characteristicsp. 233
Advantages and Disadvantagesp. 233
Conditions of Applicabilityp. 234
Variants of the Parallel Group Designp. 234
Completely Randomized Parallel Group Designp. 234
Stratified Parallel Group Designp. 235
Parallel Group Randomized Block Designp. 239
Balanced Incomplete Block Designp. 242
Other Designs with Comparison Between Subjects: Dose-Escalation and Dose-Titrationp. 244
Dose-Escalation Designp. 244
Dose-Titration Designp. 246
Complete Cross-Over Designp. 247
Characteristicsp. 247
Advantages and Disadvantagesp. 252
Conditions of Applicabilityp. 255
Variants of the Cross-Over Designp. 256
Variants Based on the Type of Randomizationp. 256
Incomplete Cross-Over Designsp. 257
Other Designs with Within-Subject Comparisons: Simultaneous Treatments and Single Patient Designsp. 261
Simultaneous Treatments Designp. 261
Cross-Over Design on a Single Patient (or "N of 1" Design)p. 261
Factorial Designsp. 263
Characteristicsp. 263
Advantages and Disadvantagesp. 268
Conditions of Applicabilityp. 270
Split-Plot Designp. 271
Characteristicsp. 271
Conditions of Applicabilityp. 273
Non-Controlled Designs in Phase II Oncology Studiesp. 273
Summaryp. 275
Study Variants Applicable to More than One Type of Design: Equivalence Studies, Interim Analyses, Adaptive Plans and Repeated Measurementsp. 277
Equivalence and Non-Inferiority Studiesp. 277
Characteristicsp. 277
The Statistical Analysis of an Equivalence Studyp. 280
Planning and Implementation Problemsp. 281
Analysis and Interpretation Problemsp. 285
Studies with Interim Analyses and Sequential Designsp. 287
Definitions and Classificationp. 288
Conditions of Applicabilityp. 290
Choice of the End-Pointsp. 292
Data Management Issuesp. 293
Statistical Issues and Decision Making Criteriap. 294
Conflict of Interest and Confidentiality Issuesp. 298
Adaptive (Flexible) Designsp. 299
Studies with Repeated Measurementsp. 301
Summaryp. 302
The Drug Development Process and the Phases of Clinical sx Researchp. 304
Overview of the Preclinical Development Processp. 304
The Phases of Clinical Developmentp. 308
Introductionp. 308
Phase Ip. 309
Phase IIp. 311
Phase IIIp. 313
Registration Dossierp. 315
Phase IVp. 319
Project Managementp. 321
The Phases of Clinical Development for Oncology Compoundsp. 322
Phase Ip. 322
Phase IIp. 323
Phase IIIp. 323
Accelerating Clinical Developmentp. 325
Summaryp. 327
Areas under the Curve of the Standard Normal Distributionp. 329
Referencesp. 331
Analytical Indexp. 337
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