Epidemiologic Studies

Our Lady of Fatima University

College of Medical Laboratory Science
Biostatistics and Epidemiology (BIOE 211)
Objectives
• explain the definition, characteristics, uses, advantages of descriptive
study designs:
• cross sectional
• cohort
• case-report & case series
• ecologic (correlational studies)
 Epidemiologic study designs
Epidemiologic
studies

Descriptive Analytical
studies studies

Population Individual Observational Experimental

Case report/ Cross Cross- Case Community

Ecologic RCT intervention
Case series sectional sectional control
trials

Cohort
Field trial
Descriptive vs Analytical
DESCRIPTIVE ANALYTICAL
• describes • explains
• more exploratory • more explanatory
• profiles characteristics of groups • analyzes why group has characteristics
• focuses on “what” • focuses on “why”
• assumes no hypothesis • assumes hypothesis
• no comparison between groups • comparison between groups over time
over time
Descriptive study
• study of:
• amount or occurrence of disease
• its distribution within the population
• for the purpose of identifying non-random variation in disease
occurrence
• 1st step in risk factor determination
• data lead to formulation of research hypothesis
• sources of data:
• routinely collected
• examples: census, vital registries, clinical records, employment
health examination
Uses of descriptive studies
• trend analysis
describe patterns of disease occurrence
• health care planning
efficient allocation of resources
health promotion and prevention programs for identified groups
• hypothesis generation
formulation of research questions & hypothesis
1st step in risk factor determination
Types of descriptive studies
• Ecologic (correlational studies)

• Case report & case series

• Cross-sectional studies (prevalence studies)

1. Ecologic Study
• correlational or aggregate studies
• measures the characteristics that represent entire population
• comparison of groups rather than individuals
• describes disease in relation to a factor of interest

• examples:
• Mortality from lung cancer & per capita cigarette sales
• Mortality from liver cancer & per capita alcohol consumption
1. Ecologic Study
• Characteristics:
• unit of observation & analysis: group
• represent average exposure and disease levels rather than actual
individual levels
• sources of data: population registries, census, vital registries,
large surveys
• may use prevalence, incidence or mortality data
1. Ecologic Study
• Primary analytical feature

Disease Disease Total

present absent
Exposure unknown unknown known
present
Exposure unknown unknown known
absent
Total known known known
1. Ecologic Study
• Types:
• Multi-group design
compare rate of diseases among DIFFERENT regions during SAME period
search for SPATIAL PATTERN
• Time-trend design (ecologic trend)
compare rate of disease OVER TIME in GEOGRAPHICALLY DEFINED
population
show temporal trends, forecast future rates and trends
• Mixed design
1. Ecologic Study
• examples:

• scatter plot of CHD mortality rates and cigarette sales

• identify the type of ecologic study

1. Ecologic Study
• examples:

• scatter plot of CHD mortality rates and cigarette sales

• identify the type of ecologic study

1. Ecologic Study
• Aims:
• to generate hypothesis

• to evaluate effectiveness of population interventions

1. Ecologic Study
• Advantages:
• low cost and convenience
• overcome measurement limitations of individual-level studies
• overcome design limitations of individual-level studies
• interest in ecological effects
• simplicity of analysis and presentation
1. Ecologic Study
• Limitations:
• cannot link exposure with disease at the individual level
• lack of ability to control for effects of potential confounding factors
• correlational data represent average exposure levels rather than
actual individual values
1. Ecologic Study
• Methods of analysis:
• graphical: scatter-plot
• computation of:
• correlational coefficient (r)
• coefficient of determination
• Linear regression analysis
• Y = a + bX
2. Case Reports & Case Series
• describes the experience of a single patient or group of patients with
similar diagnosis

• Case report:
• document unusual medical occurrences
• represents first clues in the identification of new disease or
adverse effects of exposures
• new syndromes or variants of known disease
2. Case Reports & Case Series
• Case series:
• collection of individual reports
• early means to identify the presence of an epidemic
• investigation of affected individuals can lead to hypothesis
generation:
• identification of the disease
• identification of specific risk factors
2. Case Reports & Case Series
• examples:

• Infantile Amoebiasis (Iran, 2012)

• Occupational Asthma & Glutaraldehyde Exposure (Singapore, 2004)
2. Case Reports & Case Series
• limitations:
• cannot be used to test for the presence of a valid statistical
decision
• based on experience of a single person/ group of persons only
• interpretability of information is severely limited by lack of
appropriate comparison group
Analytic studies
• Objective:
• to test a hypothesis; a statement about the relationship between 2
variables
• Types:
• observational
• experimental
• Key strategy
• use of a comparison group
3. Cross-sectional studies
• EXPOSURE (E) and OUTCOME/ DISEASE (D) variables are measured at
one point in time or over a very short period of time

• Prevalence study/ survey: use to estimate prevalence

**difference from descriptive type: presence of comparison groups
3. Cross-sectional studies
• uses:
• provides information about the frequency and characteristics of
the disease by furnishing a “snapshot” of the health experience of
the population at a specified time
• provides information on the prevalence of disease or other health
outcomes
• monitor changes in population over time
• make inference about risk of developing disease
• determine association between coexisting variables
3. Cross-sectional studies
• steps:
1. Choose study population
2. Draw an adequate # of subjects by scientific sampling
techniques
3. Collect data
4. Analyze data
3. Cross-sectional studies
3. Cross-sectional studies
• example:
• A sample of 5, 891 randomly selected males were examined and
201 were found to have liver cancer. The investigators wished to
assess whether alcohol consumption was associated with the
prevalence of liver cancer. Among 3, 247 alcohol drinkers, 124 had
liver cancer; on the other hand, of 2, 644 non- alcohol drinkers, 77
had liver cancer.
3. Cross-sectional studies
• Contingency table:
disease disease
present absent
exposure
present a b ∑𝑬 +

exposure
absent c d ∑𝑬 −

∑𝑫 + ∑𝑫 −
3. Cross-sectional studies
• Example:

D+ D-

E+

E-
3. Cross-sectional studies
• Analysis:
• Measures of disease occurrence
• prevalence proportion
• disease/ prevalence odds

• Measures of association
• prevalence difference
• prevalence ratio
• odds ratio
3. Cross-sectional studies
• Analysis: Prevalence Proportion

!"#
• Prevalence in the sample/ population = x 100
!"$"#"%

!
• PE+= !"$ x 100

#
• PE- = x 100
#"%
3. Cross-sectional studies
• Analysis: Prevalence Proportion

!"#
• Prevalence in the sample/ population = x 100 =
!"$"#"%

!
• PE+= !"$ x 100 =

#
• PE- = x 100 =
#"%
3. Cross-sectional studies
• Interpretation: Prevalence Proportion

• P = 3.4%

• PE+= 3.8%

• PE- = 2.9%
3. Cross-sectional studies
• Analysis: Prevalence Difference

• PD = PE+ - PE-

• PD = =

• Interpretation: Prevalence Difference

3. Cross-sectional studies
• Analysis: Prevalence Ratio

PE+
• PR = P =
E−

• Interpretation: Prevalence Ratio

3. Cross-sectional studies
• note: PR interpretation
• 1.0 is the null value of PR
• suggest that exposure is not associated with disease
• if PR is > 1
• exposure is positively/ directly associated with the disease
• if PR is < 1
• exposure is negatively/ indirectly associated with the disease
3. Cross-sectional studies
• advantages:
• resource-efficient

• does not suffer from lost to follow-up

• generizable
3. Cross-sectional studies
• limitations:
• difficult to establish the temporal relationship between study
variables

• unable to establish CAUSALITY

• lacks information on past exposure

• may miss disease of short duration or with periods of remission
3. Cross-sectional studies
• limitations:
• suffer from selection bias
• response bias
• survival bias
4. Cohort studies
• cohort
• group of people who share a common experience during defined
time period

• cohort study
• group or groups of persons are defined on the basis of presence or
absence of exposure to a suspected risk factor of a disease
• investigator starts with group of individuals apparently free from
the disease of interest
4. Cohort studies
• groups of individuals are divided into who are exposed and those not
exposed to a suspected risk factor

• followed during a period of time to determine and compare the

occurrence of outcome among exposed and unexposed
3. Cross-sectional studies
4. Cohort studies
• general feature:
• proceeds from a suspected cause or etiological agent to the
disease outcome

• also known:
• follow-up studies, incidence studies, prospective studies,
longitudinal studies, panel studies
4. Cohort studies
• uses:
• to identify risk factors
• to identify protective factors against diseases
• to identify prognostic factors for outcome of disease
• to describe the natural history of disease
• to project incidence/ proportion/ number of new cases of disease
over a period of time; data useful for planning acute rare services
• to assess effectiveness of preventive programs/ measures
4. Cohort studies
• types are based on:

• time relationship between initiation of study

• conceptual sense
• temporal sense

• occurrence of disease
• prospective
• retrospective
 4. Cohort studies
• types: occurrence of disease

Information needed to Type of cohort design

classify Prospective Retrospective
Occurrence Has E occurred? Yes/ No Yes
of events Has O occurred? No Yes
Data on Do records of E data exist? Maybe Yes
E&O Do records of O data exist? No Yes
4. Cohort studies
• sources of cohort groups:
• representative of the general population or geographically defined
groups

• special exposure groups

• special resource groups

4. Cohort studies
• selection of comparison groups:
• should be similar to the study group in all respects except the
exposure
• information obtained from the non-exposed groups is adequate
for comparison with the exposed group
• types:
• internal comparison group

• external comparison group

4. Cohort studies
• sources of data:
• exposure data:
• pre-existing records
• direct physical examinations and/ or laboratory test of cohort
members
• self reports of cohort members through interviews
• environmental measurements
4. Cohort studies
• sources of data:
• outcome data:
• obituaries and death certificates
• periodic exams of the participants
• health records
4. Cohort studies
• example:
• Cohort Study of Oral Contraceptive Use and Bacteuria among
Women Aged 16-49 Years Old
Bacteuria
OC use Total
Yes No
Yes 27 455
No 77 1831
Total
4. Cohort studies
• Analysis:
• Measures of disease occurrence
• incidence proportion or cumulative incidence
• incidence density
• mortality rates

• Measures of association
• ratio measures: Relative risk
• difference measures: Attributable risk
4. Cohort studies
• Contingency table:
disease disease
present absent
exposure
present a b ∑𝑬 +

exposure
absent c d ∑𝑬 −

∑𝑫 + ∑𝑫 −
4. Cohort studies
• Analysis: Cumulative incidence

!"#
• Cumulative incidence/ incidence proportion = x 100
!"$"#"%

!
• CIE+= !"$ x 100

#
• CIE- = x 100 =
#"%
4. Cohort studies
• Analysis: Cumulative incidence

!"#
• Cumulative incidence/ incidence proportion = x 100 =
!"$"#"%

!
• CIE+= !"$ x 100 =

#
• CIE- = x 100 =
#"%
4. Cohort studies
• Interpretation: Cumulative incidence

• CI = 4.35%

• CIE+= 5.60%

• CIE- = 4.04%
4. Cohort studies
• Analysis: Relative risk

• Risk ratio (RR) or Cumulative Incidence ratio (CIR)

&'!" !"! #.
• RR or CIR = = =
&'!( !"! %

• Interpretation: Relative Risk

4. Cohort studies
• Analysis: Attributable risk
• Risk difference (RD) or Cumulative Incidence Difference (CID)
• RD or CID = CIE+ - CIE- = - = ______

• Interpretation: Attributable risk

4. Cohort studies
• Analysis: Attributable risk
!"! # $ !"%$ &'! ".
• % attributable risk = = = =
!"! # &'! (

• Interpretation: Attributable risk

4. Cohort studies
• advantages:
• yield information on the incidence of the disease
• possible to compute for the relative risk
• temporal relationship between exposure and disease is clearly
defined
• design is particularly efficient for studies involving rare exposure
factors
• strongest observational design for establishing cause-effect
relationship
4. Cohort studies
• limitations:
• time consuming
• often a large sample size
• expensive
• not efficient for the study of rare diseases
• losses to follow-up may diminish validity
• changes over time in diagnostic methods may lead to biased
results
4. Cohort studies
• Sources of bias:
• selection bias
• misclassification bias
• confounding
5. Case-control studies
• Objective:
• to show that the probability of E is greater than in those with D+
than D-
3. Cross-sectional studies
5. Case-control studies
• Steps:
1. Define and select cases
a. Establish objective criteria
• diagnostic criteria for the disease
• eligibility criteria

• may be problematic if diagnostic procedure is expensive

5. Case-control studies
• Steps:
1. Define and select cases
b. Select cases
• sources:
• hospitals
• secondary or case-defined base
• population
• primary study base
• types: prevalent or incident cases
• methods of selection: total enumeration or random sampling
5. Case-control studies
2. Definition and selection of controls
a. Define control group
• comparable to the source of population cases

• matching: to achieve comparability

• 1-1 matching
• category matching
5. Case-control studies
2. Definition and selection of controls
b. Select control
• get controls from the same source population as the case

• source: hospital, genera population, special groups

• methods: random sampling or paired sampling (matched)
5. Case-control studies
3. Ascertainment of exposure
• operational definition of exposure variable
• sources: subjects or medical records
• methods of data collection: same for the 2 groups
• reference point should be identified: basis on which as individual
should be considered exposed
5. Case-control studies
4. Analysis

• Measures of association
• Odds ratio (OR)
5. Case-control studies
• Contingency table:
disease disease
present absent
exposure
present a b ∑𝑬 +

exposure
absent c d ∑𝑬 −

∑𝑫 + ∑𝑫 −
5. Case-control studies
4. Analysis: Odds ratio (OR)
!/# !%
• OR = $/% = $#
!
• ODE+ = #
$
• ODE- = %
5. Case-control studies
• example:
1. State the hypothesis in clear and specific terms

“Living with a sputum positive adult Ptb case for one year is associated
with development of Ptb in children years old and below”.
5. Case-control studies
• example:
2. Define study variables operationally

• Exposure variable:

• Outcome:
5. Case-control studies
• example:
3. Define study population

≤ 7 year old children residing in barangay X

5. Case-control studies
• example:
4. Define and select a case and control
• Case: ≤ 7 year old children who has Ptb based on WHO criteria of the
disease

• Control:
5. Case-control studies
• example:
5. Data collection
• history of exposure to the factor
• exposure should have occurred at least 12 months before the
diagnosis of Ptb---reference point
• method
• face-to-face interview
5. Case-control studies
• example:
6. Analysis

Disease
Factor Total
Yes No
Yes 20 3
No 80 97
Total
5. Case-control studies
• example:
6. Analysis: Odds ratio
!/# !% !/#####
• OR = = = =
$/% $# $/%

• Interpretation: Odds ratio

• 1: no association
• >1: association, risk factor
• <1: association, protective factor
5. Case-control studies
• example:
6. Analysis: Odds ratio
!/# !% +, - ./
• OR = = = = 8.0
$/% $# 0 - 1,

• Interpretation: Odds ratio

• 1: no association
• >1: association, risk factor
• <1: association, protective factor
5. Case-control studies
• advantages:
• quick and inexpensive
• suited to disease with long latency
• optimal for rare disease
• can examine multiple etiologic factors for a single disease
•
5. Case-control studies
• limitations:
• inefficient for rare exposure
• cannot generate incidence of disease
• difficult to establish temporal sequence
• prone to bias
6. Experimental studies
• investigator manipulates the exposure assigned to the participants in
the study
• investigators intervenes in one group and withholds intervention in
another group
• aim is to reduce variation in the outcome attributable to extraneous
factors and accounting accurately for the remaining extraneous
variation
6. Experimental studies
• Characteristics:
• analytical study, involves a test hypothesis
• independent and dependent variables
• exposure and outcome
• treatment and effect
• Prospective
• individuals are enrolled on the basis of their exposure status
• entire group is followed up and monitored
6. Experimental studies
• Characteristics:
• random allocation of study subjects
• investigators allot exposure by applying randomized allocation
scheme
• purpose is to create groups that differ only randomly at the
time of allocation with regard to subsequent occurrence of the
study outcome
• comparison group
• intervention group
• control group
6. Experimental studies

population population

random random
sample sample

volunteers volunteers

non-
intervention control exposed exposed
random observation
allocation
outcome outcome outcome outcome

RCT Prospective Observational Study

6. Experimental studies
• Classification:
• according to type of subjects/ unit of analysis
• according to purpose
• according to treatment modality
• according to phase of development of a treatment (RCTs)
6. Experimental studies
• Classification:
• according to type of subjects/ unit of analysis
• Randomized clinical trials
• patients as subjects
• Field trials
• interventions assigned to individual community members
• subjects are not defined by presence or absence of the disease
but by the initial occurrence of the disease
• Community intervention trials
• interventions assigned to whole communities
6. Experimental studies
• Classification:
• according to purpose
• therapeutic (secondary prevention)
• conducted among patients with a particular disease to
determine the ability of an agent or procedure to diminish
symptoms, prevented occurrence or decrease risk of death from
the disease
• Preventive (primary prevention)
• evaluation of whether an agent or procedure reduces the risk of
developing the disease among those free from the disease at
enrollment
6. Experimental studies
• Classification:
• according to treatment modality
• between subjects design (parallel group design)
• effects on the dependent variable for one subjects or group
of subjects are compared with the effect for another subject
o another group of subjects
6. Experimental studies
• Parallel design
6. Experimental studies
• Classification:
• according to treatment modality
• within subjects design (cross-over design)
• effects on the dependent variable for an experimental
condition and for the controlled condition are compared
within one person
• applicable for outcomes that are reversible
6. Experimental studies
• Cross-over design
6. Experimental studies
• Classification:
• according to treatment modality
• same group of subjects (factorial design)
• used to test two or more hypothesis simultaneously
• subjects are first randomized to treatments A & B to address
one hypothesis and then, within each treatment group
there is further randomization to treatments evaluate a
second question
6. Experimental studies
• Classification:
• according to phase development of a treatment (RCTs)
• Phase I
• Phase II
• Phase III
• Phase IV
Thank you…..