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Epidemiology and Beyond - 2nd Edition

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 Foundations of
Evidence-Based
Medicine
Clinical Epidemiology and Beyond
Second Edition
Contents

A word from the author xv

Author xix

Part 1 HOW DO WE SEE THINGS IN MEDICINE: Our trains of thought 1

1 How do we see medicine, health and disease? A basic set of rules and fundamental paradigms
(including evidence!) 3
Introductory comments 3
1.1 The art, craft and science of medicine 4
1.1.1 Craft of medicine 5
1.1.2 Science of medicine 6
1.1.3 Scientific method 7
1.2 The goals of medicine and its ensuing strategies: Health protection, disease prevention
and health promotion 7
1.3 How do we define and understand health and disease? 8
1.3.1 Health 9
1.3.2 Disease 9
1.3.3 Syndrome 10
1.3.4 Impairment, disability, handicap 10
1.4 What is evidence-based medicine (EBM) and what is its place in medicine today? 11
1.4.1 Evidence-based medicine defined and its steps 11
1.4.2 Evidence-based clinical medicine (EBCM) 13
1.4.3 Evidence-based community medicine and public health (EBCMPH) 13
1.4.4 Evidence-based health care (EBHC) 14
1.4.5 Grading evidence and evaluating the entire EBM process 16
1.4.6 Criticism of evidence-based medicine 16
1.5 Other ‘new’ medicines: Contradictory or complementary to EBM? 16
1.5.1 Patient-centered medicine 16
1.5.2 Personalized medicine 17
1.5.3 Evidence-based practice 17
1.5.4 Lathology 17
1.5.5 Interpretive medicine 17
1.5.6 Functional medicine 17
1.5.7 Stratified medicine 17
1.5.8 Precision medicine 18
1.6 Conclusions: Understanding the remainder of this book 18
References 18
2 The work of physicians with individuals and communities: Epidemiology and other partners in
evidence-based medicine 23
2.1 Common logic in dealing with individual patients and communities 23
2.2 Patterns of reasoning in practice and research and key ways to decisions 24
2.2.1 Key ways to make decisions 24

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2.3 Related fields in research and practice 29

2.3.1 Epidemiology 30
2.3.2 Clinical epidemiology 31
2.3.3 Biostatistics 32
2.3.4 Values beyond healing and cure: Health economics 32
2.3.5 Qualitative research 33
2.3.5.1 Qualitative research in general 34
2.3.5.2 Qualitative research in medicine, nursing and public health 35
2.3.5.3 Conclusions about qualitative research 35
2.3.6 Evidence-based medicine 36
2.3.6.1 Historical context of evidence-based medicine 36
2.3.6.2 What is ‘evidence’? 37
2.3.6.3 What is evidence-based medicine? 38
2.3.6.4 Grading and evaluating evidence and its uses 40
2.3.6.5 What does the future hold for EBM? 41
2.4 Conclusions: Fulfilling the Hippocratic Oath 41
References 43
3 The logic in modern medicine: Reasoning and underlying concepts 49
Introductory comments 49
3.1 Logic in medicine 50
3.2 Logic around us 50
3.2.1 Philosophy in medicine is more than ethics: Some definitions of common terms
in philosophy 50
3.2.2 Definitions of logic 51
3.2.3 Some basic elements and principles of logic 51
3.2.3.1 Arguments and syllogisms 51
3.2.3.2 Deduction and induction in logic and medicine 52
3.2.3.3 Abduction 53
3.2.3.4 Implications of logic for medicine 53
3.3 Uncertainty and probability in medicine 55
3.3.1 Determinism versus uncertainty 55
3.3.1.1 ‘Classical’ theory of probability 55
3.3.1.2 Chaos theory vs. probability theory: Beyond classical logic and probability 57
3.3.1.3 Fuzzy logic and fuzzy sets theory 59
3.3.1.4 Dual process of reasoning and decision thinking: Structured reasoning
and decision-making versus autonomous rapid problem-solving 62
3.4 Conclusions 67
References 67

Part 2 HOW DO WE DO THINGS IN MEDICINE: Gathering and evaluating evidence 73

4 Producing evidence: Classification, objectives and worthiness of medical research 75

4.1 Classification of studies of disease 75
4.1.1 Descriptive, observational analytical and experimental studies 75
4.1.2 Longitudinal, cross-sectional and semi-longitudinal studies 76
4.2 General scope and quality of evidence 77
4.2.1 Worthiness of studies and the crucial role of the research question within a research protocol 77
4.2.2 Classification of evidence and ensuing recommendations 81
4.2.3 Does good evidence apply to my patient(s)? 81
4.3 Conclusion 83
References 83
5 Assessing the health of individuals and communities: Health indicators, indexes and scales 85
5.1 Rates and ratios 85
5.1.1 Rates and ratios applied to disease occurrence and deaths 86
5.1.2 Morbidity, mortality and case fatality 87
5.1.3 Crude rates and specific rates 87
 Contents ix

5.2 Prevalence and incidence as measures of morbidity 87

5.2.1 Prevalence and incidence 88
5.2.2 Incidence density or force of morbidity 89
5.3 Using observed rates as probabilities of health concerns 89
5.3.1 Probabilities of preexisting health concerns: diagnostic guess (estimate) 89
5.3.2 Probability and risk of future health concerns in healthy individuals 89
5.3.3 Probability and prognosis of additional health concerns (or of deterioration in health)
in already sick individuals 89
5.4 Relationship between various measures of morbidity and mortality 89
5.4.1 Prevalence, incidence and disease duration 90
5.4.2 Mortality, incidence and case fatality 90
5.5 Using health indexes in describing disease spread 90
5.5.1 Attack rates and secondary attack rates 92
5.5.2 Point estimates and interval estimates of rates 93
5.5.3 Crude rates and standardized rates 94
5.6 The most important health indicators and indexes 94
5.6.1 The most important health indicators 94
5.6.2 Indicators from the field of demographics and routine vital health 95
5.6.3 Indicators based on births 96
5.6.4 Indicators based on frequencies of disease cases and deaths 97
5.6.5 Indicators for the child and adolescent periods 99
5.7 Mortality in the general population and its causes 99
5.8 Morbidity in the general population 99
5.9 Occurrence of common signs and symptoms in medical outpatients 100
5.10 Other health indicators 100
5.10.1 Assessing well-being in the general population: composite indicators 100
5.10.2 Assessing wellness in patients 101
5.10.3 Health assessment of individuals according to beneficial and harmful habits
and exposure to various factors 101
5.11 Conclusions 101
References 101
6 Identifying cases of disease: Clinimetrics and diagnosis 105
6.1 From clinical observation to diagnosis: Clinimetrics 106
6.1.1 Bedside clinimetrics 107
6.1.2 Hard and soft data in clinimetrics 108
6.2 Assembling a diagnostic entity (category) 110
6.2.1 Diagnosis based on one variable only 110
6.2.2 Diagnostic entity based on several manifestations (variables) 111
6.2.3 Syndrome versus disease as diagnostic entities 111
6.3 Intellectual process involved in making a diagnosis 111
6.3.1 Diagnosis by pattern recognition 112
6.3.2 Diagnosis by arborization (multiple branching) 112
6.3.3 Diagnosis by exhaustive exploration of data: Inductive diagnosis 113
6.3.4 Hypothetico-deductive diagnosis 113
6.3.5 Computer-assisted diagnosis 113
6.4 Qualitative diagnosis and its validity: ‘Does the patient have the disease?’ 113
6.4.1 Internal validity of a diagnostic test 114
6.4.1.1 Sensitivity of a test 115
6.4.1.2 Specificity of a test 115
6.4.1.3 Relation between sensitivity and specificity 116
6.4.1.4 Serial and parallel testing 116
6.4.1.5 Predictive value of a positive test result 116
6.4.1.6 Predictive value of a negative test result 117
6.4.1.7 Variability of predictive values of diagnostic and screening tests 117
6.4.1.8 Validity as reflected by true and false positive results 118
6.4.1.9 Differential diagnosis 121
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6.4.2 External validity of a diagnostic test 121

6.5 Quantitative diagnosis and its clinimetric indexes: ‘How severe is this case?’ 122
6.5.1 Indexes for one disease entity: Examples 123
6.5.2 Indexes covering several diagnostic entities: Examples 123
6.6 Comments on screening tests and screening programs 124
6.6.1 General rules for implantation of screening programs 125
6.6.2 Research implications of screening 125
6.7 Studying the use of diagnostic tests in clinical practice 126
6.8 Major criteria of a valid diagnostic study 126
6.8.1 General criteria of an acceptable study in medical research 126
6.8.2 Specific criteria and steps in a valid study on diagnosis 126
6.9 The question of differential diagnosis 129
6.10 Diagnosis as seen in fuzzy theory 130
6.11 Conclusions 132
References 133
7 Describing what happens: Clinical case reports, case series, occurrence studies 139
7.1 Three types of descriptive studies and their objectives 140
7.2 Drawing clinical pictures or describing disease in an individual: Case reports 140
7.2.1 Disease history and disease course 141
7.2.1.1 Preclinical stages 142
7.2.1.2 Clinically manifest period of disease: Clinical stages 144
7.2.1.3 Convalescence, recovery and other outcomes of disease 146
7.2.1.4 Special comments on infectious diseases 146
7.2.2 How to complete single case studies or clinical case reports 147
7.2.2.1 Choosing the topic 148
7.2.2.2 Giving direction to a CCR 149
7.2.2.3 Evidence-based clinical case report 149
7.2.2.4 Desirable qualities of a CCR submitted for publication 150
7.2.3 Evidence from multiple case observations. Case series and systematic reviews of cases 151
7.2.3.1 Cross-sectional or longitudinal portraits of cases 151
7.2.3.2 Systematic reviews and meta-analyses of cases 152
7.2.3.3 Conclusions on case series reports 152
7.3 Picturing disease as an entity: Describing disease occurrence in the community—Descriptive
or occurrence studies 152
7.3.1 Objectives of occurrence studies 153
7.3.2 Desirable attributes of occurrence studies 153
7.3.3 How to understand an occurrence (descriptive) study, its structure and content 153
7.3.3.1 Structure 153
7.3.3.2 Persons, time and place in occurrence studies: Establishing the portrait
of disease spread 155
7.3.3.3 Drawing conclusions 157
7.3.4 Special kinds of occurrence (descriptive) studies 159
7.3.4.1 Epidemiological surveillance 159
7.3.4.2 Disease clustering 160
7.3.4.3 Medical record linkage 161
7.3.4.4 Cohort analysis or analysis by cohort 161
7.4 Conclusions 162
References 163
8 Search for causes of disease occurrence: Why does disease occur? 167
8.1 Concept of cause(s) in medicine 168
8.2 Basic concept and design of causal studies 169
8.3 Fundamental philosophy and criteria of the cause–effect relationship 170
8.3.1 Basic considerations 170
8.3.2 Prerequisites of a causal proof 171
8.3.3 Criteria of a causal relationship 173
 Contents xi

8.3.3.1 Major criteria of causality 173

8.3.3.2 Conditional criteria 174
8.3.3.3 Experimental proof as a reference criterion 174
8.3.4 Measures (indices) of strength and specificity of associations 174
8.4 Cohort study 175
8.4.1 Information provided 177
8.4.2 Comments on findings and their interpretation and meaning 178
8.4.3 Advantages and disadvantages of cohort studies 180
8.5 Case-control study 180
8.5.1 Information provided 180
8.5.2 Matched pairs case-control studies 181
8.5.3 Estimation of specificity of a causal relationship in a case-control study 181
8.5.4 Occurrence of disease and validity of odds ratio 182
8.5.5 Numbers needed to harm derived from case-control studies 182
8.5.6 Advantages and disadvantages of case-control studies 182
8.6 ‘Hybrid’ designs of analytical studies 183
8.7 Conclusions on causality 183
8.8 Major prerequisites for valid etiological studies 184
8.8.1 Exclusion of the effect of chance 184
8.8.2 Predictable results 185
8.8.3 Bringing observational analytical studies closer to the standards of experimental research 185
8.8.4 Good (clinimetrically valid) data 185
8.8.5 Sufficient number of study subjects (sample size) 185
8.8.6 Absence of bias 186
8.8.7 Satisfactory presentation of resulting data 188
8.8.8 Realistic and critical interpretation and conclusions 188
8.9 Advanced quantitative methods in etiological research: Multivariate and multivariable analysis 188
8.9.1 Studying more than one cause at a time 189
8.9.2 Studying relationships in space and time between various possible causal factors 190
8.9.3 Study of genetic factors as causes of disease 191
8.10 Investigation of disease outbreaks and their causes 192
8.11 Epidemiologic proof of causality in court: Contributions of physicians to decision-making
in tort litigation 194
8.12 Conclusions 196
References 199
9 The impact of treatment and other clinical and community health interventions:
A ‘does it work?’ evaluation 205
9.1 Basic paradigm and general considerations 205
9.1.1 Treatment and cure as a cause–effect relationship 206
9.1.2 Evaluation of treatment 207
9.2 Evaluation of treatment in disease cure 207
9.2.1 The basic design of treatment study in disease cure and prevention 208
9.2.2 Assessment of the impact of treatment: Treatment efficacy and effectiveness 209
9.2.3 Phases of evaluation of treatment 212
9.2.4 Designs of clinical trials 214
9.2.4.1 The golden standard: Randomized trials with parallel groups 214
9.2.4.2 Other types of randomized clinical trials 217
9.2.5 Assessment of a clinical trial’s quality 219
9.2.5.1 Research protocols: A priori prerequisites 219
9.2.5.2 Treatment modalities or ‘maneuvers’ in clinical trials 222
9.2.6 Further considerations 226
9.2.6.1 Clinical trials in special circumstances: Surgery and general medicine 226
9.2.6.2 Pharmacoepidemiology 226
9.2.6.3 Multicenter clinical trials 227
9.2.6.4 Medical technology assessment 228
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9.3 Evaluation of health promotion programs and interventions 228

9.4 Conclusions 230
References 231
10 Prognosis: Studies of disease course and outcomes 239
10.1 Conceptual considerations 240
10.1.1 Definition of prognosis 240
10.1.2 Prognosis as opposed to risk 240
10.1.3 Classification of prognosis according to interactions under study 241
10.1.4 Objectives of prognosis and prognostic studies 241
10.1.5 Basic expressions (measures) of prognosis: Events and outcomes 242
10.2 General methodology of prognostic studies 244
10.2.1 Descriptive studies of prognosis 245
10.2.2 Observational analytical studies of prognosis 245
10.2.3 Experimental studies of prognosis or clinical trials 246
10.3 Special methodology of prognostic studies: Survival or time-to-event analysis 246
10.3.1 Describing disease course and outcomes by establishing survival or time-to-event curves 246
10.3.1.1 Basic vocabulary 246
10.3.1.2 Describing what happens: Building survival tables and establishing a survival
or time-to-event curve 247
10.3.2 Comparison (analysis) of two or more curves: Explanation of what happens 248
10.3.2.1 An ‘eye’ test: Simple visual comparison of survival rates using confidence intervals 248
10.3.2.2 Establishing relative survival or time-to-event rates 248
10.3.2.3 Statistical comparison of surviving groups (rates): Log-rank test 249
10.3.2.4 Evaluating several causes of survival simultaneously: Cox regression analysis 249
10.3.3 Predicting a good or bad prognosis 249
10.4 Lessons for clinical research from experience gained in prognostic studies 250
10.4.1 Prognostic stratification 250
10.4.2 Prognostic migration in time 250
10.5 Outcomes research 251
10.6 Clinical prediction rules 251
10.7 Prognostic studies as subject of systematic reviews 252
10.8 How to construct a study on prognosis 252
10.8.1 A priori precautions, components and criteria proper to prognostic studies 252
10.8.2 A posteriori evaluation. Was the study good? 253
10.8.2.1 General acceptability of the study of prognosis 253
10.8.2.2 Applicability of a prognostic study to the individual patient 253
10.9 Conclusions: Considerations for further work in the area of prognosis 254
References 256

Part 3 PUTTING EXPERIENCES TOGETHER AND MAKING DECISIONS IN MEDICINE:

Structured uses of evidence 261

11 Analyzing and integrating a body of knowledge: Systematic reviews and meta-analysis of evidence 263
11.1 Definitions and objectives of meta-analysis, reviews and summaries of evidence 264
11.2 Original field of meta-analysis 264
11.2.1 Definition of meta-analysis 266
11.2.2 Narrative reviews 267
11.2.3 ‘Classical’ meta-analysis 267
11.2.3.1 The effect size in an original study 267
11.2.3.2 Average effect size across studies 268
11.2.3.3 Other ways to assess the effect 268
11.2.3.4 Assessment of homogeneity or heterogeneity of individual studies 268
11.2.3.5 File drawer problem and fail-safe number of studies 269
11.3 Meta-analytic procedures, methods and techniques in medicine 269
11.3.1 Measurement of effect or quantitative meta-analysis 270
 Contents xiii

11.3.1.1 Typical odds ratio or odds ratio across studies 270

11.3.1.2 Relative risk, attributable risk and etiological fraction across studies 272
11.3.1.3 Number needed to treat across studies 273
11.3.1.4 Cumulative meta-analysis 273
11.3.1.5 More advanced quantitative and graphical methods 274
11.3.2 Qualitative meta-analysis: The core of systematic reviews 274
11.3.2.1 Scoring of studies for quality 275
11.3.2.2 Best evidence synthesis 276
11.3.3 Beyond clinical trials: Meta-analysis in other fields of medical research 276
11.3.3.1 Diagnosis and ‘diagnosimetrics’ 276
11.3.3.2 Descriptive studies of risk 276
11.3.3.3 Analytical observational studies of causality 276
11.3.3.4 Studies of prognosis 277
11.4 Components and attributes of a good meta-analytic study 277
11.5 Conclusions and recommendations for the future 280
11.5.1 Information that should be contained in meta-analyses and systematic reviews 280
11.5.2 Projections (fields of application) of meta-analysis and systematic reviews 280
11.6 Advantages and disadvan­tages of meta-analysis 281
11.7 What next? 281
References 282
12 Using evidence and logic in everyday clinical reasoning, communication and legal and
scientific argumentation 289
12.1 Introducing evidence into the process of logic 290
12.2 Clinical rounds, reports, papers, testimonies and health policies as arguments 290
12.3 Our thinking and reasoning: Essential definitions and meanings 291
12.4 Logical argumentation in medicine 294
12.5 Argument and argumentation the Aristotelian way 294
12.6 Architecture and basic validity rules of arguments (syllogisms) 295
12.6.1 Categorical syllogism 296
12.6.2 Modern argument, Toulmin’s model: A ‘multiple (six-) element’ way of reasoning
to reach valid conclusions 297
12.6.3 Definitions 302
12.6.4 Fallacies 303
12.6.4.1 Classification of fallacies 303
12.6.4.2 Examples of fallacies 303
12.6.5 Reminder regarding some additional and fundamental considerations 305
12.6.5.1 Types of evidence 306
12.7 Ways to improve arguments and argumentation 306
12.8 Possible uses of evidence and argumentation in the area of fuzzy logic applications 307
12.8.1 Paradigm of fuzziness in medicine 307
12.8.2 Essentials of fuzzy argumentation 308
12.9 Conclusions 309
References 310
13 Decision analysis and decision-making in medicine: Beyond intuition, guts and flair 313
13.1 How decisions in medicine are made: General concepts 314
13.2 Basic vocabulary and reference readings 315
13.3 Direction-searching tools in decision-making 316
13.3.1 Decision trees and their analysis: Outline of the decision analysis process 316
13.3.1.1 Stages of decision analysis, or ‘growing, blooming, pruning and harvesting’
decision trees 318
13.3.1.2 Sensitivity analysis, or ‘fertilizing and grafting’ decision trees 321
13.3.2 Chagrin analysis 322
13.3.3 Threshold approach to clinical decisions 324
13.3.4 Cost–benefit analysis in therapeutic decision-making 325
13.3.5 Desirable components and attributes of clinical decision analysis 327
13.3.6 Advantages and limitations of decision analysis 327
xiv Contents

13.4 Direction-giving tools in decision-making 327

13.4.1 Tactical tools: Clinical algorithms and decision tables 328
13.4.1.1 Algorithms as flowcharts 328
13.4.1.2 Algorithms as decision tables 331
13.4.1.3 Advantages and limitations of clinical algorithms 332
13.4.2 Strategic tools for right decisions: Clinical practice guidelines 333
13.5 Decisions and decision-making in community medicine and public health 336
13.6 Conclusions 336
13.6.1 Pros and cons of decision analysis 336
13.6.2 The future of research in clinical decision-making 338
References 339

Epilogue: Widening horizons, staying in touch—What next? 345

Glossary: Preferred terms and their definitions in the context of this book 349

Index 387
A word from the author

This book is not a book about medical

causal or therapeutic discoveries, but
it should help you learn and know how
to make them and how to use them.

This book focuses mainly on reasoning, critical thinking reasoning in an increasingly complex world of uncertainty
and pragmatic decision-making in medicine, based on the and incomplete information of unequal quality. As such, this
author’s experience in clinical epidemiology and its crucial book outlines how to make the best possible crucial decisions
role in the emerging field of evidence-based medicine. in clinical care, disease prevention and health promotion.
In this book, let us examine evidence-based medicine Learning medicine not only involves memorizing a con-
(EBM) as: siderable volume of information, mastering sensory skills
and communicating with patients and health professionals.
It also requires excellent reasoning techniques, processing
●● an initiative common to all health sciences and pro- of information through sensory skills, judging the state of
fessions (with adaptations to their specificities), things and decision-making. Classical textbooks of medi-
●● a still young and developing domain, which goes cine or surgery examine mainly the former. This book cov-
well beyond the best evidence of noxious or ben- ers the latter.
eficial cause–effect relationships, This book is intended primarily for young and less young
●● a still young and developing domain, which requires physicians, as well as other health professionals from the
proper foundations, such as clinical and fundamen- fields of dentistry, veterinary medicine, nursing, clini-
tal epidemiology and biostatistics, cal nutrition, psychology and health administration who
●● a still young and developing domain requiring a want to acquire the fundamental information needed to
structured modern argumentative reasoning in better understand and ultimately practice evidence-based
building the best possible evidence, medicine. It is also intended to help in the critical read-
●● an equally valid and structured evidential way of ing of medical literature and in the understanding of mes-
making decisions, and sages and reasoning of health professionals, planners and
●● an evaluation of the short-term and long-term decision-makers. Experienced clinicians from various
effectiveness, efficacy and efficiency of the above specialties who teach in-house staff with various levels of
activities for the patient, community and health experience will want to refer to it to show their students
care provider. how to reason and translate their experience into bedside
decision-oriented research. Residents in specialty-training
programs around the world are becoming increasingly
Let us also see if evidence-based practice, once imple- involved in medical research and want to understand its
mented, is better than any of its research and practice alter- workings. Many of them learnt just enough epidemiology
natives in health sciences. and other fundamentals years ago to pass their exams and
We will succeed only partly in this endeavor, but we their clinical guides did not always offer appropriate learn-
must nevertheless go well beyond the original definition of ing experiences. Hence, the fundamentals of epidemiology
EBM towards a starting point for its real understanding and and other disciplines on which evidence-based medicine
practice. is based today are covered in this reading to the obvious
Modern medicine does not revolve exclusively around benefit of medical undergraduates at exam time. However,
new technologies such as magnetic resonance imaging, the primary focus of this book is on medical and biological
robotized microsurgery, genetic mapping or cloning of liv- thinking and decisions endorsed only after the application
ing organisms. Modern medicine also requires structured of relevant quantitative methods and techniques.

xv
xvi A word from the author

Medical students will learn about evidence-based medi- evidence is assembled in view of obtaining the best pos-
cine at a later date and in greater detail. Before that, they sible answer to clinical questions. The emphasis here is
must master the essentials of fundamental and clinical epi- on logical uses of evidence.
demiology and of logical thinking in medicine. Their final
and licensure examinations will cover not only the prin- Readers will find two unusual chapters in this reading
ciples of evidence-based medicine, but also the basics of (Chapters 3 and 12) that examine logic and critical think-
epidemiology and preventive or community medicine. This ing. Acquiring good evidence is not enough. Good evidence
reading should help them succeed. must also be put to good use both logically and critically.
Does this book follow current courses on epidemiology Some essentials of reasoning, in other words thinking
and other related disciplines? It does, but not entirely. In enlightened by logic, are presented in these chapters. In the
fact, it serves as a kind of propaedeutics to reasoning and past, this was not taught at all. Student intelligence and rem-
decision-making in health sciences practice and research. nants of logic picked up in college courses were expected
Many graduates and fully qualified health professionals to suffice. Hence, a more detailed explanation is justified.
wanting to refresh their knowledge of the foundations of Moreover, the ‘discussion’ and ‘conclusion’ sections of med-
evidence-based medicine such as epidemiology and clinical ical articles are essentially logical discourses about evidence
epidemiology will hopefully find this book a useful transi- produced and presented in ‘results’ and all preceding sec-
tion from the basic sciences of reasoning and measurement tions. Valuable evidence may, in fact, be lost due to poor
in medicine to their practice in today’s evidence-based world. logical use or misuse.
Most readers of this book will become family physicians The first and the third sections of the book discuss the
or clinicians in various specialties. Some will even embrace necessary framework for the second section, which is pri-
epidemiology, community medicine or public health. marily for busy medical students with exams in mind as well
Whatever career path is chosen, we all need the solid foun- as for other readers who may be less familiar with clinical
dations of organized reasoning and the ability to make ben- epidemiology and related domains, techniques and methods.
eficial decisions for patients and communities, as discussed Most of the chapters are introduced by an example of rea-
in this basic reading. soning in practice, in the form of a logical syllogism, modern
The most challenging endeavor for the author of this argumentation and critical thinking. These argumentations
book was to write for the uninitiated, curious, intelligent are not necessarily all valid. They tend instead to reproduce
and doubtful, while making the message ‘short and sweet’. reality and what may occur in real life with all its imperfec-
The easiest thing to do is to reach the enthusiasts. An old tions. Also, they underlie the need for good evidence for the
academic adage says that Assistant Professors (Lecturers) premises and conclusions of logical arguments.
teach more than they know, Associate Professors (Senior The first-time reader of the message of this book may
Lecturers) teach all they know, and Professors explain only be surprised by a simultaneous presentation and interface
what their students really need to know. The author has between:
gone through all these stages only to realize that the last is
the most difficult of all. ●● Conceiving, producing and using evaluating evidence
The reader will find that this book is organized accord- itself,
ing to the basic steps of clinical work with a patient and not ●● Basic and clinical epidemiology notions and methodolo-
according to some other scientific methodology, like descrip- gies supporting evidence, and
tive, analytical or experimental techniques or designs. ●● Informal logic, critical thinking and modern argumen-
Specifically, this book is divided into three sections: tation to manage health problems.

●● The first section, ‘How do we see things in medicine’, Why is all this presented altogether?
focuses on understanding how physicians think, reason Critical thinking and argumentation are needed in many
and make decisions; medical activities, such as:
●● The second section, ‘How do we do things in medicine’,
or ‘Gathering and evaluating evidence’, explains how ●● Organization of the physician’s own thought,
to obtain and evaluate good evidence at every step from ●● Transfer of thought in patient records and charts,
risk assessment and diagnosis to final therapeutic or ●● Assessing research information,
prevention decision-making. It offers essential defini- ●● Listening to the patient,
tions, formulae, outlines, flowcharts and checklists use- ●● Advising the patient clearly and understandably (tactics),
ful in health measurement, case and occurrence studies, ●● Developing, issuing and evaluating clinical guidelines
search for causes, clinical trials and prognosis. The core and other ways of strategic thinking,
of clinical epidemiology is in this section. ●● Defending and explaining medical care (as proposed and
●● The third section, ‘Putting experiences together and used) to a physician, the physician’s coworkers and legal
making decisions in medicine: Structured uses of evi- and social organizations and bodies, health administra-
dence’, shows how all the ‘bricks’ are integrated into tion included, and
the decision-making methodology and how medical ●● Writing and reading medical and other research papers.
 A word from the author xvii

Many ‘classics’ are excluded from the bibliography. The bibliography, however rich, is nevertheless restric-
Instead, references cover a variety of information, which tive. Most often, it includes basic expanded readings,
the reader might find in the literature when moving from practical applications or some key historical references or
more general topics to specific ones in health sciences. For ‘classics’, mainly from major and easily accessible medical
example, the diversity of definitions for such ordinary and journals. If needed, it can be expanded through an elec-
ubiquitous terms as art, science, logic or reasoning shows tronic and manual literature search and retrieval.
how medical thinking is part of a wider domain of thought Bibliographies and references are constantly evolving in
and human experience. their form and content. The references at the end of each
The message should be as explicit as possible. Hence, chapter were reviewed and updated as accurately as possi-
some unavoidable computational examples are intention- ble. References, especially electronic ones, evolve over time
ally numerically oversimplified. For many, percentages are and other changes have also occurred in the 16 years since
more understandable than much smaller proportions such the publication of the first edition of this book. Even if some
as those seen in real-life cancer epidemiology. However, we sources have changed and evolved in a way that prevents
should be aware of the real magnitude of health phenomena us from fully completing, referring and updating them, we
around us. More specific statistical techniques like stan- have kept them in this edition in their original version for
dardization of rates, establishing life tables and survival historical reasons. For example, when a specific topic was
curves, obtaining overall rates and ratios from stratified covered in the literature, even if a reference (electronic,
data or establishing confidence intervals for various obser- website) is less complete and sometimes no longer accessible
vations belong to other programs and sources dealing with the first edition of this book dates back to the beginning of
these methods and techniques. Some readers will find that this millennium), it is included here. Also, references reflect
notions and illustrative examples beyond the basics are only the involvement of various individuals and institutions in a
mentioned in passing. Both can be found, however, in the particular domain of interest at that time. degree involve-
extensive bibliography that accompanies this overview of ment of various individuals and institutions in a particular
evidence-based medicine tools. domain of interest at their time. Their other contributions
Some topics are repeatedly presented, such as the dual may be searched for using their names. Websites will con-
system of reasoning (in Chapters 2 and 6), grading the evi- tinue to evolve beyond the control of their visitors (includ-
dence (Chapters 2 and 4), deduction, induction, or abduc- ing the author of this book). Thank you, readers, for your
tion in medical thinking (Chapters 2 and 6) and pyramiding kind understanding.
the evidence (Chapters 2 and 12). This may be necessary to Clinical examples are kept as simple as possible, since
see and realize the relevance and distinctions of these topics medical novices are exposed to epidemiology in the early
in their different settings and applications from one medical stages of their training.
domain and activity to another. Also, the repetition (with In a state-of-the-art clinical practice, everything evolves:
modifications) should help first-time readers understand New medical technologies; normal values of clinical and
these topics faster. paraclinical observations (reference data); diagnostic crite-
The index is restricted in such a way that it highlights ria; treatment indications; drug doses; treatment effective-
only the definitions of the most important terms needed ness; prognostic information given to the patient. Examples
for the proper and necessary understanding of the essential from these areas are quoted here to illustrate medical logic
message. It can also be used as a foundation for the glossary. only and for didactic purposes. They should not be fol-
Such orismology (from Greek orismos, meaning definition lowed blindly in daily office work and bedside decision-­
and logos, meaning study), that is, study, use and evaluation making. Such information requires continuous updating
of definitions pertaining to medical practice and research, and revision.
justifies a considerable number of definitions from one Friends, colleagues and coworkers, who helped so much
chapter to another. in the production of this book, are in no way responsible for
Given the heterogeneity of health-related domains and potential errors—only the author is.
of the meaning of terms and health phenomena and enti- Any monograph is not a true monograph. For the first
ties, we have selected definitions specific to our domain and edition, the author was privileged to have several remarkable
this book. critical readers: Professors Jean Lambert (late) and Michèle
This book is conservatively written, in a manner similar Rivard (both Université de Montréal—biostatistics),
to a literary essay. There are no exercises, but many, hope- Geoffrey Norman (McMaster University—biostatistics),
fully well-chosen, practical examples. This structure was David Hitchcock (McMaster University—philosophy and
selected for two main reasons: Experienced lecturers who logic), Marianne Xhignesse (Université de Sherbrooke—
honor the author by referring to this book in their teachings family medicine), Jim Bellamy (University of Prince
almost always use their own exercises and problems in class. Edward Island—applications of fuzzy theory in medicine),
Also, they will correctly choose their own national health Gillian Mulvale (McMaster University—health economics).
statistics, priorities and clinical and public health experi- Illustrations were prepared by Jacques Cadieux (Université
ence to ensure relevance to the practice of medicine in their de Montréal—infographics). The language and style were
own countries, which may not necessarily be the author’s. reviewed by Nicole Kinney (Linguamax Services Ltd.), who
xviii A word from the author

has reviewed them remarkably again for this second edition. Today, other medical propaedeutics are also needed—how
The artwork was reviewed and updated for this edition by to think, reason and make decisions in medicine in a logi-
Steve Janzen, Senior Graphic Designer at Media Production cal, rational and organized manner. This is the focus of
Services, McMaster University. Dr. Joanna Koster, Senior the book.
Publisher—Medicine & Life Science, CRC Press/Taylor & Even the title of this book is a word of convenience.
Francis Group was an invaluable guide for this second edi- Labels come and go, be they epidemiology, evidence-based
tion as Ms. Kristine Mednansky, Senior Commissioning medicine or theory of medicine, but the common ground
Editor at CRC Press/Taylor & Francis, was for the first one. for all these paradigms remains: How to make medicine
The author remains indebted to all these people for their most beneficial for patients and communities.
guidance and for sparing readers from the worst in this The Hippocratic Oath tells us primum non nocere or
reading, especially in places where we agree to disagree. ‘first, do no harm’. The illogical, erratic and inefficient prac-
Many textbooks are affected by globalization and can be tice of medicine causes harm! In this respect, ‘big heart,
used successfully in different cultures, health systems, tra- small brain’ medicine must make room for ‘big heart, big
ditions and values. In this respect, examples in this book, brain’ medicine. In fact, is this not what we all want? Is this
like major causes of death or patterns of morbidity, are not what our patients and communities expect from us?
drawn either from the author’s North American experience Having said this, does the reader feel that about 500
or from countries that best illustrate the underlying mes- pages on these topics is too much? Textbooks of medicine or
sage, such as the epidemiological transition of the Japanese surgery are usually five times more voluminous and while
society or potentially extreme findings around the globe. they provide essential and vitally important ingredients for
They do not necessarily represent standards or ideal values ‘good medicine’, this book should help readers understand
to be adopted by all. what to do with such ingredients! If this book also enables
The author would like to offer readers a foundation on readers to learn how to think in medicine, the author will be
which to build their own specific experience, be it in family delighted since when he was very young, less experienced,
medicine, internal medicine, surgery, pediatrics, psychiatry, yet eager to learn, his teachers failed to touch upon these
community medicine or other specialties. Also, needless to topics.
say, dentistry, nursing, nutrition, medical records, sanitary Last but not least, the author remains indebted to CRC
and environmental engineering professionals share many Press/Taylor & Francis Group experts: Dr. Joanna Koster,
common ways of thinking with what might now be called Senior Publisher—Medicine & Life Science, for her initia-
Evidence-Based Medicine, Logic and Critical Thinking in tive to prepare, produce and publish this book, Ms. Linda
Medicine, encompassing past and present experiences in Leggio, Production Editor, for producing this book, and
epidemiology, clinical epidemiology, evidence-based medi- Mr. Arunkumar, Project Manager from Nova Techset, for
cine and other current streams of thought. preparing proofs for printing.
What makes a health professional? Not only perfect This second edition Foundations of Evidence-Based
examination or surgical skills, but also sound reasoning Medicine is attractive and easy to read. Readers will appre-
and decision-making. Earlier generations were taught ‘med- ciate the kind of presentation of pages which are followed
ical propaedeutics’: The ability to learn and know before in this book.
a ‘real thing’ occurs. This often involved the basic skills
required of a clinical, laboratory or community health clerk: Milos Jenicek
Interacting with patients; physical and paraclinical exami- Rockwood, Canada
nations; understanding and interpretation of basic findings.
Author

Milos Jenicek, MD, PhD, LMCC, FRCPC, CSPQ, is cur- 3. Clinical Epidemiology, Clinimetrics (in French with
rently Professor (Part-Time) in the Department of Health R. Cléroux, Épidémiologie clinique. Clinimétrie,
Research Methods, Evidence, and Impact (HEI), formerly EDISEM and Maloine, 1985)
the Department of Clinical Epidemiology and Biostatistics 4. Meta-Analysis in Medicine. Evaluation and Synthesis of
(CE&B), Faculty of Health Sciences, McMaster University, Clinical and Epidemiological Information (in French,
Hamilton, Ontario, Canada. He is also Professor Emeritus Méta-analyse en médecine. Évaluation et synthèse de
at the Université de Montréal, Montréal, Québec, Canada. l’information clinique et épidémiologique, EDISEM and
In 2009, he was elected Fellow of The Royal Society of Maloine, 1987), recognized by the James Lind Library
Medicine, London, UK. and The Journal of The Royal Society of Medicine as the
Dr. Jenicek has contributed to the evolution of epidemiol- first textbook on meta-analysis and systematic reviews
ogy as a general method of reasoning and decision-making in medicine
in medicine. Supported by the Université de Montréal, he 5. Epidemiology. The Logic of Modern Medicine (EPIMED
undertook short sabbaticals and study visits in the 1970s to International, 1995), also published in Spanish (Masson
various universities including Harvard, Johns Hopkins, Yale, Espana, 1996) and Japanese (Roppo Shupan, 1998)
Tufts, North Carolina at Chapel Hill and Uniformed Services 6. Medical Casuistics. Correctly Reporting Clinical Cases
University of Health Sciences at Bethesda to further enhance (in French, Casuistique médicale. Bien présenter un cas
his teaching and research. Academic, governmental and clinique, EDISEM and Maloine, 1997)
professional institutions in Western Europe (France, Spain, 7. Clinical Case Reporting in Evidence-Based Medicine,
Italy, Switzerland, Portugal), North Africa (Algeria, Tunisia, published by Butterworth Heinemann in 1999
Morocco) and on the Pacific Rim (Japan, Korea) have also 8. Clinical Case Reporting in Evidence-Based Medicine
benefitted from his lecturing, professional expertise, visiting (Butterworth Heinemann, 1999) appears as an
professorships and other professional initiatives. expanded second edition in English (Arnold, 2001),
During his term as Acting Chairman of the Department Italian (Il Pensiero Scientifico Editore, 2001), Korean
of Social and Preventive Medicine, Université de Montréal (Gyechuk Munwha, 2002) and Japanese (Igaku Schoin,
(1988–1989), Dr. Jenicek established a Clinical Epidemiology 2002)
teaching program at the graduate level. The core course of 9. Foundations of Evidence-Based Medicine, published
this program, taught by Dr. Jenicek, was also part of a similar in 2003 by Parthenon Publishing/CRC Press/Taylor &
program at McGill University, where Dr. Jenicek was Adjunct Francis Group
Professor as well at the time. Until 1991, Dr. Jenicek was a 10. Evidence-Based Practice. Logic and Critical Thinking
member of the Board of Examiners of the Medical Council in Medicine (with D.L. Hitchcock), published by the
of Canada (Committee on Preventive Medicine). In 2000, American Medical Association in 2005
he was invited by Kuwait University to act as an External 11. A Physician’s Self-Paced Guide to Critical Thinking
Examiner of its graduating medical students. (AMA Press, 2006)
In addition to numerous scientific papers and other col- 12. Fallacy-Free Reasoning in Medicine. Improving
laborations with leading medical journals, Dr. Jenicek has Communication and Decision Making in Research and
published 16 textbooks to date that reflect his national and Practice (AMA Press, 2009)
international initiatives: 13. Medical Error and Harm. Understanding, Prevention,
and Control was published by CRC Press/Taylor &
1. Introduction to Epidemiology (in French, Introduction à Francis in 2011
l’épidémiologie), (EDISEM and Maloine, 1975) 14. A Primer on Clinical Experience in Medicine. Reasoning,
2. Epidemiology. Principles, techniques, applications (in Decision Making, and Communication in Health
French with R. Cléroux, Épidémiologie. Principes, tech- Sciences (CRC Press/Taylor & Francis), written in 2012
niques, applications, EDISEM and Maloine, 1982, and proposes critical thinking as a part of clinical propae-
in Spanish, Salvat Editores, 1987) deutics for health professions
xix