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Spring , 2008. STAT 6395 Special Topic in Statistics: Epidemiology. Filardo and Ng, 2008. I. Epidemiology.
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Spring , 2008 • STAT 6395 • Special Topic in Statistics: • Epidemiology Filardo and Ng, 2008
I. Epidemiology The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control health problems
Persons affected Place Time Distribution Epidemiology: The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control of health problems
Determinants All the physical, biological, social, cultural, and behavioral factors that influence health Epidemiology: The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control of health problems
Health-related states or events • Diseases • Mortality (death) • Specific causes of death • Injuries • Disability • Health-related behaviors • Physiological measurements • Results of preventive regimens • Clinical outcomes • Provision and use of health services Epidemiology: The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control of health problems
Specified populations • Residents of a defined geographic area • Students who attend a specified school • Persons who belong to a specified organization • Workers at a specified workplace Epidemiology: The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control of health problems
Translation Study Results Scientific articles and presentations at meetings Clinical guidelines Prevention programs Quality of care improvement programs Patient safety programs Epidemiology: The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control of health problems
Control Operations or programs aimed at reducing the adverse impact of the disease on the community • Prevention • Cure • Management Epidemiology: The study of the distribution and determinants of health-related states or events in specified populations and the translation of study results to control of health problems
Primary prevention An action taken to prevent the development of a disease in a person who is well and does not have the disease in question Operations or programs aimed at reducing the adverse impact of the disease on the community: 1) Prevention; 2) Cure; 3) Management
Secondary prevention (Cure and management) The identification and treatment of people who have already developed a disease or precursors of the disease, through screening, at an early enough stage in the disease’s natural history (early detection) such that intervention will be more effective than if the disease had been discovered later Operations or programs aimed at reducing the adverse impact of the disease on the community: 1) Prevention; 2) Cure; 3) Management
II. Objectives of Epidemiology • To describe the extent of disease in the community • To identify risk factors (factors that influence a person’s risk of acquiring a disease) for disease and the etiology or cause of disease • To study the natural history (course from onset to resolution) and prognosis of disease • To evaluate both existing and new preventive and therapeutic measures (including health care delivery) • To provide the foundation for developing public policy and regulatory decisions relating to environmental problems
…bottom line, Epidemiology research requires a multidisciplinary effort and statisticians play a key role in: • Hypothesis development • Study execution
Hypothesis development Epidemiologists study the specific distribution and determinants of specific diseases Development of hypotheses to test in an epidemiologic study requires in-depth knowledge of the disease and determinants under study • Medical and biological sciences • Social and behavioral sciences Epidemiology Research hypothesis development and study execution
Study execution • Statistics ------------>Biostatistics • Medical and biological sciences • Social and behavioral sciences Epidemiology Research hypothesis development and study execution
Biostatistics • Separate chance observations from meaningful observations • Sampling • Sophisticated statistical analyses Epidemiology Research Biostatistics, medical and biological sciences, social and behavioral sciences
Medical and biological sciences • Microbiology, e.g., to identify infectious agents • Clinical medicine and pathology, (e.g., to identify cases of disease) • Molecular biology, (e.g., to identify genotype of individuals) • Biochemistry, (e.g., to measure serum hormone levels) Epidemiology Research Biostatistics, medical and biological sciences, social and behavioral sciences
Social and behavioral science • Design questionnaires for obtaining valid information • Design effective interventions for lifestyle changes Epidemiology Research Biostatistics, medical and biological sciences, socialand behavioral sciences
Epidemiology Sub-disciplines • Disease-specific subject matter • Exposure-specific subject matter • Health services research
Disease-specific subject matter • Infectious disease epidemiology • HIV/AIDS epidemiology • Malaria epidemiology • Chronic disease epidemiology • Cancer epidemiology • Cardiovascular epidemiology • Perinatal epidemiology • Neuroepidemiology • Psychiatric epidemiology Epidemiology disciplines Disease-specific subject matter, Exposure-specific subject matter, Health services research
Determinant (exposure)-specific subject matter • Environmental epidemiology • Occupational epidemiology • Pyschosocial epidemiology • Genetic epidemiology • Nutritional epidemiology • Pharmacoepidemiology Epidemiology disciplines Disease-specific subject matter, Exposure-specificsubject matter, Health services research
Health services research • Operations research: the study of the placement of health services in the community and the optimum utilization of such services • Program evaluation • Clinical epidemiology or outcomes research: the study of illness outcomes in persons seen by providers of health care; evaluation of medical treatments Epidemiology disciplines Disease-specific subject matter, Exposure-specific subject matter, Health services research
Types of epidemiologic studies • Experimental • Observational (this course will focus on this second type of studies)
Experimental Studies • Studies in which conditions are under the control of the investigator(s). • The investigators assigns subjects to different study groups. The effect of the treatment is determined by comparing the outcome of interest in these groups. Type of studies Experimental, Observational
Experimental studies (examples) • Randomized clinical trial (unit of study is the individual) • Community trial (unit of study is the community) Type of studies Experimental, Observational
Observational studies • Studies in which the investigators does not control conditions, but rather observe nature taking its course by gathering information, recording, classifying, counting, and analyzing the collected data. • Exposure and disease outcomes would have occurred whether or not the studies have been performed because there were no ‘a priori’ intervention(s) on the part of the investigators. Type of studies Experimental, Observational
Observational studies (examples) • Descriptive • Analytic Type of studies Experimental, Observational
Descriptive studies Studies aimed at describing the distribution of disease or other health-related variables with respect to person (age, gender, race, socioeconomic status), place (census tract, county, state, country, urban/rural), and time (season, year) Type of studies Experimental, Observational (descriptive)
Descriptive studies • Often use routinely-collected data • Can define high-risk groups • Can be used for hypothesis generation, but generally not hypothesis testing Type of studies Experimental, Observational (descriptive)
Analytic studies • Test specific etiologic hypotheses • To generate new etiologic hypotheses • To suggest mechanisms of causation • To generate preventive hypotheses • To suggest or identify potential methods for disease prevention In these studies, the epidemiologist observes the relationship between an exposure and a disease or other health outcome. Type of studies Experimental, Observational (analytic)
Definition: Exposure A potential causal agent or characteristic, such as infectious agent, behavior, dietary factor, medication, medical treatment, genetic makeup, environmental agent, or physiologic state (e.g., serum level of a hormone or nutrient; blood pressure). An exposure may be harmful or beneficial Type of studies Observational Analytic
Types of analytic studies • Cohort studies • Case-control studies Type of studies Observational
Cohort studies A study in which a group of persons exposed to a factor of interest and a group of persons not exposed are followed and compared with respect to the incidence rate of the disease or other condition of interest Time Type of studies Observational Cohort studies
Cohort studies (study schema) Type of studies Observational Cohort studies
Case-Control studies Studies in which a group of persons with a disease (cases) and a comparison group of persons without the disease (controls) are compared with respect to the history of past exposures to factors of interest Past Present Type of studies Observational Case-Control
Case-Control studies (study schema) Type of studies Observational Case-Control
Either descriptive or analytic studies • Cross-sectional studies • Ecologic studies Type of studies Observational
Cross-sectional studies Studies of the distribution of exposures and/or disease in a defined population at one given point in time Type of studies Observational Cross-sectional
Ecologic studies Studies of the association between exposures and disease in which the units of analysis are populations or groups of people, rather than individuals. This involves the assessment of the correlation of exposure rates and disease rates among different groups or populations. Causality, though??? Type of studies Observational Ecologic
Example involving several types of study designs Ecologic study results Case-Control study results Cohort study results Randomized controlled trial results
Which type of study is the ‘Gold Standard’ and/or more common/feasible? Experimental Efficacy Observational Effectiveness controlled setting (difficult to reproduce in real life) real life setting
Focus of this course is on observational Epidemiologic research (research regarding the direct study of disease in human populations)
Some triumphs of observational Epidemiology • Smoking causes lung cancer • Identification of cardiovascular disease risk factors • Characterization of how HIV spreads through a population • Identification of occupational hazards (e.g., asbestos)
Three ‘Eras’ of Epidemiology • Sanitary (1800-1875) • Infectious disease (1875-1950) • Chronic disease (1950-present)
Sanitary ‘Era’ (1800-1875) • Miasma theory of disease – poisoning by foul emanations (miasma) from the soil, water, and air. • Created national vital statistics systems: much valuable descriptive epidemiology • Demonstrated clustering of disease in slums and among the poor • Solutions – sewage systems, drainage, clean water supplies, garbage collection, decent housing • Incorrect miasma theory, but solutions were a major contribution to public health Lesson: prevention doesn’t necessarily require understanding of cause Sanitary, Infectious disease, Chronic disease
Infectious disease ‘Era’ (1875-1950) • Germ theory: single microscopic agents relate one-to-one to specific diseases • Epidemiology took a back seat to laboratory science, although in the1920s-30s, the germ theory was broadened to accommodate the interactive roles of host (immune and nutritional status), environment, and agent in infectious disease • Other epidemiologic contributions: • Occupational exposures as causes of cancer • Specific vitamin deficiencies as causes of disease Sanitary, Infectious disease, Chronic disease