Epidemiological Studies

Ephrem Mamo (MPHE, PhD Candidate)

Assistant Professor of Epidemiology

May, 2022
Addis Ababa, Ethiopia

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 Why Epidemiological Studies?
♦ To answer questions like:
– How big is the problem (magnitude)?
• Prevalence, incidence, mortality

– What, who and where of any health problem?

• Person characteristic of affected population
• Place characteristics (locality)

– What factors are associated with certain disease

• Specific factors related to causation

– To evaluate interventions
• Which drug is best for patients with X disease
• To evaluate any program 2
Categories of epidemiological studies
1. Descriptive epidemiological studies
Population as study subject
o Correlational /ecological studies
Individual as study subjects
o Case report / Case series
o Cross-sectional surveys
2. Analytic epidemiological studies
2.1 Observational studies
o Case-control study
o Cohort study
2.2 Experimental / intervention studies
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Epidemiological studies

Populations Ecologic

Case-series
Descriptive
Case-report
Individuals
Cross-sectional

Prospective
Cohort
Observational
Retrospective
Case-control
Analytical
Intervention Clinical trials
1. Descriptive Studies
• Some studies simply describe occurrence of disease or health
related problems
– Prevalence of a disease,
– Rate of certain behaviour

• When describing these factors, it does not link with anything

• However we can identify unusual distributions or
correlations

• These insights can be used to generate interesting hypothesis

(Case series, cross-sectional, ecological)
 Cont….
 Describes the general characteristics of the distribution of a
disease in relation to person, place and time.
Who? Where? When?

 It provides valuable information

To allocate resources efficiently and
To plan effective prevention or education programs.
It provides the first important clues about possible
determinants of a disease (formulation of hypothesis).
• less time consuming and less expensive.
• most common used

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A. Case report:
 It is the study of health profile of a single
individual using a careful and detailed report by
one or more clinicians.

 It is common form that is published in articles

 It is made using
 Simple history,
 Physical examination and
 Lab. / radiologic investigation.
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 Cont…
 Report is usually documented if there is unusual
medical occurrence, thus it may be first clue for
identification of a new disease.

 It is useful in constructing a natural history of

individual disease.
 E.g
It was a single case report that formulated the
hypothesis of oral contraceptive use increases
venous thrombo-embolism.
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B. Case series
 Individual case report can be expanded to a case
series, which describes characteristics of a number
of patients (usually 5-12) with a similar disease.

 Similar to case report, it is usually made on cases

having new and/ or unusual disease (giving interest
to clinicians)

 It is often used to detect the emergence of new

disease or an epidemics.
Eg. The first five AIDS cases in USA.
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 Cont…
Example:
Five young, previously health homosexual men were
diagnosed as having Pneumocystis carinii pneumonia at
Los Angeles hospital during a six month period from
1980 to 1981.

This form of pneumonia had been seen almost exclusively

among older men and women whose immune systems
were suppressed.

This unusual circumstance suggested that these

individuals were actually suffering with a previously
unknown disease, subsequently it was called AIDS.
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 Example of case series
• In 1974, Creech and Johnson reported a case series of
three men with angiosarcoma of the liver among
workers at a vinyl chloride plant.
•
• This number in such a small population during the time
period studied was clearly in excess of what was
expected

• this led to the formulation of the hypothesis that

occupational exposure to vinyl chloride caused hepatic
angiosacrcoma.
• Later the same year, this hypothesis was substantiated by
data from two analytic studies
Case reports or case series

 Useful for the recognition of new diseases,

 Useful for constructing of the natural history of a

disease,

 Use to formulate a hypothesis and to detect an

epidemic

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 Cont…
 Both case report and case series are able to formulate
a hypothesis but are not able to test for presence of
valid association.

 Fundamental limitation of case report is presence of a

risk factor that is simply coincidental (by chance)

 It is difficult to test for association because there is no

relevant comparison group

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2. Cross-sectional surveys
 Is generally called study of prevalence

 Survey is conducted in a population, to find prevalence

of a disease and exposure.

 Exposure and disease status are assessed simultaneously

among individuals at the same point in time .
 Data are obtained Only once.
 help in assessing the health status and health care needs
of a population
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 Cont….
 Cross-sectional surveys could provide information about the
frequency of a disease by furnishing a ‘snapshot’ at a
specified time.

 May be used first step in cohort or case control studies

 It can be considered as analytic study, if it assesses presence

of an association.

 Measures of association is made using odds ratio.

 For factors that remain unaltered overtime such as sex, race,
blood group,
 it can provide a good evidence.

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 Advantages of ….
• one-stop, one-time collection of data

• less expensive & easier to conduct

• provide much information useful for planning health

services and medical programs

• show relative distribution of conditions, disease, injury

and disability in groups and populations
• studies are based on a sample
Limitations

 Egg and hen phenomena: Since exposure and

disease status is assessed at a single point in
time, temporal relationship between
exposure and disease can not be clearly
determined.
 It may not show strong cause-effect
relationships if sample size is small.

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Analytical Studies

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 Case-Control Study

Exposure
Disease
? (Case)

? No disease
(Control)
Retrospective Nature

After identifying cases & controls investigators look backward in time to

assess their exposures
 Case-Control Study
• PAST PRESENT
• It is a type of an observational analytic study

• Compares one group among whom a problem is

present with another group where the problem is
absent in order to find out factors contributing to the
problem

• Problem examples- malnutrition, lung cancer,

neonatal death
Application of Case-Control studies
• It is good to do for rare diseases or outcomes

• Persons with the disease are compared with controls free of

disease, for presence of the factor under investigation

• Cases are compared to controls with respect to exposure

frequency

• It may be possible to explore a wide range of potential exposures

for a single outcome
 Cases
♦ It is the outcome of interest

♦ It can be
– A disease
eg. HIV status, Malaria caseness

– A behavior
eg Alcohol drinking habit, Cigarette smoking

– Occurrence of an event
eg migration

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 Selection of cases
• Define ‘disease’ and how it will be ascertained
Sources of cases
1) Hospital or medical care facility
• this approach is referred to as hospital-based case
control study
• is more common because it is relatively easy &
inexpensive to conduct
2) General population
• Referred as population-based case control study
 Source population

Exposed
Unexposed
 Source population

Exposed
Cases
Unexposed
 Control
• It is the comparison group

• It should be free of the disease of interest

• It should be as similar as the cases in all aspects except

for the disease of interest

• Controls must have the same opportunity of getting

exposure as cases and should be subjected to the same
inclusion and exclusion criteria.
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 Principles of Control Selection
• Controls should be selected from the same study
base (target population) as cases

• Should be selected independently of their exposure

status!!

• If they had developed illness, they should be

excluded or should be considered as cases

• Comparable information should be obtained from

controls as it is from cases
 Source population

Exposed
Cases
Unexposed

Controls
 How many control groups?
• when the number of available cases and controls is large and the
cost of obtaining information from both groups is comparable,
the optimal control-to-case ratio is 1:1.

• When the sample size of cases is limited, or when the cost of

obtaining information is greater for cases or controls, the
control-to-case ratio can be altered to achieve the desired sample
size.

• As the number of controls per case increases, the power of the

study also increases.
• not generally recommended that this ratio increase beyond 4:1
 Advantages of Case-control Studies

• Assesses well for rare diseases

• It can assess several exposures (determinants) of a
single outcome
• It can be completed within short time (Rapid)
• It can be performed with very cheap or Low cost
• A small sample size may be needed
• It can also be done from available data
• Not needing detailed ethical problem/ issues
 Disadvantages of Case-control Studies

• Problems with bias

– Recall
– Selection of controls

• Temporal Relationship of exposure-disease

Relationship difficulté to establish

• Not suitable for rare exposures

• No direct calculation of rates and risks

Cohort study
• It is also called follow up, longitudinal, prospective
study.

• The word cohort is used to designate a group of

people who share a common experience.
– a Birth cohort,
– a Cohort of smokers,
– a Cohort of MPH student graduates in 2013, etc.

• It is an observational study that measures incidence of

disease occurrence.
Cohort studies
• An investigator studies a group of exposed and
unexposed subjects and follows the study subjects
over a period of time and compares the incidence of
developing disease of interest in the 2 groups

PRESENT TIME FUTURE TIME

Diseased
Exposed
No disease

Diseased
Unexposed
No disease
1. Basic elements
• “Disease” free at entry

• Selected by exposure status rather than outcome

• Follow up is needed to determine the incidence

of the outcome in each exposure group
• For non communicable chronic diseases this may take years

• Compares incidence rates among exposed

against unexposed groups
a. Exposure status
• Study subjects should be disease free

• Define study subjects using inclusion and

exclusion criteria on the basis of exposure
status
– Environmental factors: smoking, air pollution,
pesticides

• Criteria can be specified by amount of

exposure
– Eg. Cigarette Smocking (# of cigarette per day)
b. Possible outcomes in cohort

• No disease

• Disease

• Lost to follow up
 Example
• Follow 100 children who received BCG
vaccination and another

• 100 who didn't get BCG vaccination and see

how many of them get tuberculosis.
 2. Features of cohort study

1. It shows temporal sequence

Exposure Disease

2. Good to assess effect of rare exposures

3. Could assess multiple effects of a single

exposure
3. Types of cohort studies

• Two forms of cohort study

– The major difference is on the basis of

• Initiation of study and the occurrence of
disease.

– The two forms are similar, because selection

of study subjects is made on the basis of their
exposure status.
 Cont…

1. Classical (Prospective) cohort

– The exposure may or may not have occurred at
the time when the study begin, but the outcome
has certainly not yet occurred.

2. Historical (Retrospective) Cohort

– Both the exposure and outcome have already
occurred when the study is initiated.
Steps in a prospective cohort study
1. Define the population at risk (=cohort)
2. Determine exposure status to a factor of interest of
all subjects in the cohort

3. Make sure that study subjects are free of the disease

of interest at time of enrolment

4. Follow exposed and non-exposed forward in time to

ascertain whether they develop the outcome of
interest
5. Compare the outcomes in the exposed and the
unexposed group with each other
Summary
Strength
Is of particular value when the exposure is rare.

Can examine multiple effects of a single exposure.

Can elucidate temporal relationship between

exposure and disease.

Allows direct measurement of incidence of disease in

the exposed and non exposed.
Summary
Limitations
Is inefficient for the evaluation of rare diseases,

If prospective, can be extremely expensive and time

consuming.
Validity of results can be seriously affected by losses
to follow up.
Loss to follow up is the major source of bias in cohort studies.
Members of cohort may be lost to follow up, if this proportion is
large, > 20- 25 %, it becomes difficult to validate.
 Retrospective cohort
both exposure and outcome of interest have already occurred when
the study is initiated
Advantages of retrospective cohort
• can be conducted much more quickly and cheaply
• it requires the availability of adequate records.

• Since these data were often recorded for purposes other than
investigation of the hypothesis of interest, this can result in
incomplete and possibly non-comparable information for all study
subjects.

• Often information on potential confounding factors is not available

from such records
Experimental/Intervention Study
Designs
 Research Designs
– determinant or intervention (treatment) is planned

– control (non-treatment) group is free from the

intervention

– Placebo - an inert agent indistinguishable from the

active treatment.

– selected into intervention (treatment) and control

(non-treatment) groups by randomization
(preferred).
 Types of experimental study designs
A. Classification based on the population studied
1. Laboratory experiments /Trials
effects of anti-cancer drugs in mice
2. Clinical trial
• usually performed in clinical settings
• the subjects are patients, effect of anti-cancer drugs in humans
3. Field trial
• used in testing medicine for preventive purpose
• subjects are healthy people e.g. vaccine trial
4. Community trial
• unit of the study is group of people/community e.g. fluoridation of
water to prevent dental caries
 Intervention
• Clinical Trials
– patients are study subjects
– intervention is not a primary preventive measure because subjects
are already diseased. usually intervention is a form of treatment.

– subjects are diagnosed with health outcome of interest and

selected to either treatment options.

– Example - Insulin Therapy and Diabetes

– Two treatment groups : Intensive insulin and standard insulin
therapy
 C. Classification based on objective

C.1 Phase I
trial on small subjects to test a new drug with small
dosage to determine the toxic effect
C.2 Phase II
trial on small group to determine the therapeutic
effect
C.3 Phase III
• study on large population
• usually randomized controlled trial
 The quality of "gold standard" in
intervention studies

Randomization

Use of placebo

Blinding
 Limitation
1. Ethical considerations
substances already known to be harmful should not be
used in this study.

2. Feasibility/ practical issues

-difficult to find a sufficiently large study subjects

3. Cost - experimental studies are often very expensive

because of the long follow-up period.
Thank you