EPIDEMIOLOGIC

STUDY DESIGNS

By Tarekegn Solomon(BSc in PH, MPH in Epidemiology )

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 Learning objective:
At the end of this session the student will be able to:

• Explain types of epidemiological study designs

• Distinguish between Descriptive and analytical,

observational and experimental study designs

• Differentiate Advantages and limitations of major

epidemiological study types

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 EPIDEMIOLOGIC STUDY DESIGN
 A study Design:
 is a specific plan or protocol for conducting the study, to
translate the conceptual hypothesis into an operational one.

 The goal of epidemiological studies is understanding the

frequency, pattern, and causes of disease in populations

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 Epidemiologic study designs cont…

Descriptive studies
provide information on patterns of disease
occurrence in a population.

Often provide clues about disease causation

that can be pursued by analytical research
designs

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 Epidemiologic study designs cont…
Analytic studies
 uses comparison groups to determine whether the
characteristics of those with a given health condition are
alike/different from the comparison group.

 concerned with the search for causes and effects.

 quantify the association between exposures and outcomes

and to test hypotheses about causal relationships that have
usually been generated from descriptive studies.

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 Epidemiologic study designs cont…
 Observational- Studies of disease burden or etiology,
investigator passively observes nature

 Experimental- Studies in which the investigator

actively manipulates which groups receive a preventive
or treatment intervention of interest, and then follows
over time.

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The ‘tree’ of epidemiological studies
Epidemiological Studies

Observational Experimental

Descriptive Analytic Trial

Cross-Sectional Cross-sectional

Ecological

Cohort

Case-control
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“Tree” of Epidemiologic study designs

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 Epidemiologic study designs cont…

Clinical Observation- Unanswered phenomena

observed at the bedside (cases report & series).
I. Case reports:
• clinician reports on an unusual disease or association

• Sporadic discoveries of a unique clinical observation

stimulating to investigate disease etiology.

• E.g. a clinician reported benign hepatocellular adenomas in

woman who had taken oral contraceptives

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 II. Case series:
• A series of clinical observations sharing most of
similarities

• Aggregates individual cases in one report.

• For example: a cluster of homosexual men in Los

Angeles(USA) with a similar clinical syndrome final
confirmed as AIDS

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 Epidemiologic study designs cont…
 Case report and series are employed in clinical, rather
than epidemiologic studies

 But often are precursors to epidemiologic studies.

 Use for case definition & clinical picture ascertainment

at initial time.

 Eg. Recognition of AIDS began with case reports and

case series's describing cases of young men in California
and New York City with Pneumocystis carinii
pneumonia (PCP) and Kaposi's
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 III. Ecological study
• Examines the relationship between exposure and disease with
population-level rather than individual-level data.

• E.g. colon cancer rates are higher in U.S. that use mostly
surface water and high per capita meat consumption.

• These relationships suggest that something about surface

water, e.g., chlorination, and something about meat
consumption, e.g., saturated fat intake, might be factors in the
development of colon cancer.

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 Ecological study…

• However, since exposure is not known at the individual

level, it is possible that the cases of colon cancer are not
themselves people who drink chlorinated water or eat
meat.

• The attempt to infer individual characteristics or

relationship from group-level measures is called the
“ecologic fallacy”

• Once the hypothesis has been advanced, analytic studies

are the next epidemiologic recourse.
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 IV. Cross-sectional study
 The frequency of occurrence of a given disease and
exposure to one or several factors hypothesized to
influence the probability of occurrence of the disease are
determined at the same time.

 Exposure status and disease status are measured at one

point in time or over a period.

 It is prevalence study. Comparison of prevalence among

exposed and non-exposed.

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Cross sectional study cont…

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 Cross-sectional cont…
 Advantages
• Determining prevalence data
• Reasonably inexpensive (less time & other resources)
• Ideal to evaluate associations between disease & fixed factors
(age, sex…)
• If in defined population, highly representative & generalizable.

 Limitations
• Difficult to differentiate causal from non-causal associations.
• Difficult to differentiate Sequence of events- egg & chicken
dilemma.

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 V. Case control study
 A case-control study is one in which persons with a condition
("cases") and suitable comparison subjects ("controls") are
identified, and then the two groups are compared with respect to
prior exposure.

 Exposure status is traced backward in time.

 The word case is related to the outcome of interest in the study,

which commonly comprises individuals with the health problem of
interest.

 The comparison group (control) supplies information about the

expected risk factor pattern in the population from which the case
group is drawn.
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Case-Control Studies

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Example of case control study

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 Steps in conducting case-control study
I. Define who is a case
- Establish strict diagnostic criteria
- All who fulfill the criteria will be cases
- Those who don’t fulfill will be controls
II. Select a sample of cases from cases
- This sample must be representative of the cases
Sources of cases
1. Hospital (health institution)
2. Population
III. Select controls from control population
- Should be representative of control population.
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Steps in conducting case-control study cont…
- Should be similar to cases except outcome of interest.
- Should be selected by the same method as cases.
Sources of controls
1. Hospital
2. Population control
3. Special controls
IV. Measure the level of exposure in cases & controls by
using similar criteria.
V. Compare the exposure status in cases & controls

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Advantages
 Cheap, easy and quick studies
 Multiple exposures can be examined
 Rare diseases and diseases with long latency can be
studied

Disadvantage
 Inefficient for the evaluation of rare exposure
 Subject to bias (selection, recall, misclassification)
 Direct incidence estimation is not possible
 Temporal relationship is not clear
 Multiple outcomes cannot be studied
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 VI. Cohort studies
Definition
• Cohort: A group of individuals with a common
characteristic or experience
• The term cohort has military, not medical, roots. A
cohort was a 300–600-man unit in the Roman army

Figure : An early cohort in search of favorable outcomes

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 Cohort studies
• Definition
• Cohort : a group of individual with a common characteristic
or experience.
• Cohort studies: Epidemiologic study in which subsets of a
defined population are sampled on the basis of exposure to one
factor hypothesized to influence the probability of occurrence of
a given disease.

• In a follow-up study, people without the disease are followed

up to see who develops it, and disease incidence in persons with
a characteristic is compared with incidence in persons without
the characteristic
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 Types of Cohort study
Depending on the temporal relationship between the
initiation of the study and the occurrence of the disease,
there are two types of cohort studies.
1. Prospective - At the beginning of the study the outcome
has not yet occurred. Regarded as more reliable than the
retrospective, if the sample size is large and follow-up
complete.
2. Retrospective - Both exposure and outcome status have
occurred at the beginning of the study. This is possible
where medical records permit accurate assessment of
both risk factors and disease outcomes. Efficient in cost
and time. Often uses of data collected for other purposes.
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 Prospective cohort study
Disease
Study starts Exposure occurrenc
e

time

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Retrospective study

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 Steps in conducting cohort study
1. Define exposure
2. Select exposed group
3. Select non-exposed group
4. Follow and collect data on outcome
5. Compare outcome b/n exposed & non-exposed

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 Advantages of Cohort Studies
- Accurate relative risk (risk ratio) estimation
- Can examine rare exposures (asbestos > lung cancer)
- Temporal relationship can be inferred
- Time-to-event analysis is possible
- Magnitude of a risk factor’s effect can be quantified
- Selection and information biases are decreased
- Multiple outcomes can be studied

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 Disadvantages of Cohort Studies
-Lengthy and expensive
- Not suitable for rare diseases
- Not suitable for diseases with long-latency
- Unexpected environmental changes may influence the
association
- Non-response, migration and loss-to-follow-up biases
- Sampling, ascertainment and observer biases are still
possible

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 VII. Experimental/Interventional Study
 Individuals are allocated in the intervention and control
groups by investigator.

 If properly done, it can produce high quality data. This

is the gold standard study design.

 The key is that the investigator assign into either group,

whether it is done randomly or not.
 Always prospective.
E.g. Assign children randomly to get chloroquine or not,
and see how many develop symptomatic malaria.
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SCHEMATIC DISPLAY OF EXPERIMENTAL
STUDY

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 Classification of Intervention Studies
Based on population

 Clinical trial - usually performed in clinical setting

and the subjects are patients.

 Field trial- used in testing medicine for preventive

purpose and the subjects are healthy people.

 Community trial - a field trial in which the unit of

the study is group of people/community.

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 Classification cont…
Based on design

 Uncontrolled trial - no control group. control will be

past experience (history).

 Non-randomized controlled - there is control group

but allocation into either group is not randomized.

 Randomized controlled - there is control group and

allocation into either group is randomized.

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 Classification cont…
Based on Trial Objective
 Phase I - trial on small subjects to test a new drug with
small dosage to determine the toxic effect.
 Phase II - trial on small group to determine the
therapeutic effect.
 Phase III - study on large population to test
effectiveness.
– Randomized Controlled Trial (RCT)
– Often it is multi-centered …… licensing
 Phase IV– Post marketing surveillance
– Long term prospective assessment of effects & side
effects
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 Experimental cont…
 The main distinction from other types of analytic studies
is that individuals are allocated into experiment or control
group by the investigators.

 The comparison groups in intervention study are known

as the intervention group and the control group.

 The intervention group receives the test drug (the

preventive activity such as health education, diet and
exercise). The control group shall be offered the best
known alternative or a placebo activity with no known
effect on the outcome.
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 Experimental cont…
 It is very important that the two groups gain equal
amount of attention in the study.

Problems Related to Intervention Studies

 Ethical considerations
 Feasibility/practicability
 Cost

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 Experimental cont…
 The quality of "gold standard" in intervention studies can
be achieved through :
• Randomization
• Use of placebo
• Double Blinding
 Randomization: random allocation of both intervention
and control groups by lottery or random number table.
Advantages
study group will be comparable- confounders

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 Experimental cont…
 Blinding - is when the observers and/or subjects are kept
ignorant as to the group to which the subjects are
assigned
- Open/Unblind = all know which intervention a patient is
receiving.
- Single blind = subjects are ignorant
- Double blind = observer and subject are ignorant
- Triple blind = observer, subject and analyst are ignorant
 Placebo - an inert agent indistinguishable from the active
treatment. Use of placebo minimizes bias in the
ascertainment of both subjective disease outcomes and
side effects (blinding).
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Summary

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