BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II
INTRODUCTION TO EPIDEMIOLOGY
Distribution and Determinants of Disease in
Humans
Chapter 5
Study Design
Introduction
• When interpreting results of studies,
consider:
-The magnitude of the hypothesized outcome
(disease)
-gaano ba ka dalang or heavy ang disease?
Eg. The knee injury (not so concerned because
it rarely happens) and diabetes mellitus (a major
concern)
-The population characteristics that the findings
apply to.
-How the data was collected.
(are the data collected properly?)
-we need to be careful in looking the findings
and data of a research study.
Study design affects ability to answer
research questions and to apply results
We need to be selective in order to have an
accurate study.
In epidemiology, we are more concern on
how to use the data for the benefit of the
population and community.
 A major part of study design is:
-Determining the desired characteristics of the
study population.
-Developing methods to acquire an appropriate
sample.
-Deciding how to collect data.
GOALS OF STUDY DESIGN
In order to perform a study, It must first be
designed
The goal of study design:
-Develop the methods necessary to accomplish
the goals of the study (See Table 5-1)
-will provide a blueprint and framework for the
research.
 Good study design starts with:
-Who (the persons)
-What (what specific disease are u going to
study)
-When (when, and look for the seasons and
peak of different diseases)
TABLE 5-1 Goals of Study Design
-Represent desired population.
-Allow appropriate determination of
outcome/disease.
-Eliminate and reduce bias.
-Identity confounders (confusers, not factor or
maybe considered a factor) and control for
confounding
-Test hypotheses.
-Find association or eight for causality.
-Evaluate the intervention.
Represent Desired Population
-Once the reference population (population na
pinag-aaralan) is identified, methods for
selecting subjects should be developed.
-Goal is to enroll a study group who will
represent identified population.
Allow Appropriate Determination of
Outcome/Disease
• Ensure methods are chosen that will allow the
appropriate determination of the outcome.
• Important to develop methods that can make a
valid determination if the study subjects have
the outcome or not. (through diagnostic testing)
Suspect case- individual who wasn’t tested and
confirmed with the outcome (disease).
Eliminate and Reduce Bias and Identify
Confounders
• Very important design goals are to:
-Collect valid data Recruit appropriate subjects.
- Collect data on the variables that may distort
the results of the study (confounders).
-Important that all methods address issues of
bias and confounding.
Test Hypotheses
-Enabling investigators to test a hypothesis is a
further goal of study design
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 BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II

-Study must be designed to allow comparisons Observational vs. Experimental

of subjects that have different levels of the Observational Experimental
independent variable (exposure) Intervenes with the
•Develop methods that ensure comparison only gathers data subjects in some way
groups are handled the same way. from the subjects (drug treatment,
-basically, an investigation. symposium, or
Investigators can be more confident that any vaccine)
differences between comparison groups are true
Without intervention With intervention
differences, and not due to improper methods
(test if that
intervention changed
their problem)
Find Associations or Evidence for Causality
Another goal of study design: Descriptive vs. Analytic
-to allow the ability to search for new Descriptive Analytic
associations More of the Generating
-May not start with "known hypothesis" distribution of the interpretations (link)
(Exposure should be known before the outcome) disease. (analytical
-Goal of study may be to look for characteristics (eg. Frequency) statistics; test
and outcomes that are related to each other. operation sheet.
-Evidence for causality may be provided by Test of comparison,
finding associations. association)
-Many discoveries in public health have come Knowing the disease Comparing several
from studies finding new associations. progression of an variables.
individual. Is there a significant
Evaluate the Intervention How can sex be a difference between
determined factor? the gender of the
-Pertains to studies with the goal of evaluating
Genetic or behavior population and their
an intervention such as a drug treatment,
Male or female geographical
surgical procedure, vaccine or education dominated behavior? locations in the
program. development of
(experimental research because it has diabetes mellitus?
intervention)
For example: looking at the effects of the given
intervention. The Perspective of Time in Collecting Data
-Most common way to label a study design
-These studies similar to those testing a is based on the timeframe of the study
hypothesis, experience of the comparison group - "Cornerstone" of a study is the "window"
is influenced by the investigators. (actual data collection period) during which the
investigator collects the data from subjects for
General Classifications of Study Design the FIRST TIME.
 Designs can be complex or simple; all -Timeframe of a study is dependent on the
have one characteristic in common: appropriate definition of this window.

Designs are a compilation of a number of Retrospective study Prospective study

methods that determine: Identify when we are Wait until 15 years
-How the data will be collected AND going to collect data before the patient
-The characteristics of the study sample (window) developed the
population. disease.
Usually in present The window time here
 General labels (male and female) are used time, for example the is 15 years.
diagnosis made 2 yrs
to describe the distinctions between designs
ago (the window time
here is 2 yrs)
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 BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II

Collecting Data Based on Exposure: episodes)

Cohort Design: Participants currently The disease is
-The "gold standard" of observational study do not have the already present
designs. disease. during your data
-if you want to establish causality, use this. collection.
-in cohort, there would be a comparison.

 Two possible timeframes:

Prospective cohort study:

Following outcome-free subjects into the future
to ascertain outcome.

-when we see the word cohort, remember the

exposure. Based on exposure.
does the exposure contributed to the
development of the disease.
-we are establishing causality (exposure first
before the outcome- should be the result of the
exposure)
Retrospective cohort study:
-Using exposure information of subjects from
the past and then ascertaining outcome from
the present.
-Designed to compare those with the exposure
of interest to those without the exposure of
interest.
-Must exclude any potential subjects who
already have the outcome. (prospective)
Exposed (taking aspirin)
Not exposed (not taking)
Outcome (have hemorrhagic episodes)
Without outcome (doesn’t have hemorrhagic
episodes)
Then we test for the mean and the significant
number.
Example: in 20 exposed, 11 has an outcome,
and 9 are without outcome.
Is 11 significant to establish that taking aspirin
causes hemorrhagic episodes in the future?

-Investigators watch subjects to see who Advantages of a cohort study design

develops the outcome (Prospective).
-Incidence rates (new cases) of outcome in both
groups are compared when study is over. (Still
in prospective)
1. Allows true determination of incidence rates of
outcome.
2. Allows attributes associated with the outcome
to be identified (pre-determined) as risk factors.

Prospective cohort Retrospective 3. Enables the highest quality research data and
study: cohort study: Information.
The exposure is The exposure is in
identified in the the past. 4. Allows the evaluation of interventions.
present.
The outcome is in the The outcome is in the 5. Allows the assessment and documentation of
future. present. natural history of disease.

The exposures are Although, the 6. Can assess multiple exposures and
identified and then outcome (peeps wt
outcomes.
there will be a follow diseases) is in the
up (eg. After 5 years, present, it is based on
check if the patient the exposures.
have hemorrhagic

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 BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II

Disadvantages of a Cohort Study Design

Problems with this design:
-Length of time and cost
-Prospective study requires ability to keep
track of study subjects during entire follow-up
period (eg. every 6 months)
-Possible for only a few (or none) of the
subjects to get the outcome during the follow
up period.

Enrolling Subjects in a Cohort Study

Options for enrolling subjects:
-Enroll subjects, then assess exposure and We can also compare based on incidence
divide them into exposure categories. densities (on person years- denominator)
-Enroll subjects after identifying their exposure Numerator- num of cases
Category.
-Enroll subjects only with a specified exposure Person years- number of individuals times
category, and compare to already known groups the number of years na nakasama sa study.
(it can be a population group).
Exposed:
Loss to Follow-Up
Subjects must be kept track of over the study
timeframe.

-Those that cannot be followed are "Lost to

follow-up.
-Methodology must be developed to minimize
risk of loss (incentives and compensation).
-Sufficient numbers should be enrolled so that
the expected number of loss can be
accommodated without damaging the study.

Time at Risk
-Defined as the time that everyone accumulates Not exposed:
between exposure and developing the outcome

-Can compare time at risk in two ways:

• Compare incidence rates of both groups
• Use incidence densities in both groups

-Demonstrates both the number of individuals

with outcome, as well as the amount of time at
risk.

Example of computation for time at risk:

O= outcome (individual who developed the
disease)
L=loss of follow-up

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 BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II

Summary of a Cohort Study Design Advantages of a Case Control Study

-Study design that is the most scientifically
desirable and allows for determining risk factors
and “cause”.
-Allows true determination of incidence of an
outcome.
-Can study multiple hypotheses/variables.
-Long time period necessary can increase
Costs.
Collecting Data Based on Outcome:
Case Control Study Design
Case control studies:
-investigators collect data in a group of subjects
with the outcome of interest (cases), as well
as in a group of subjects without the outcome
of interest (control)
-Subjects in both groups are compared Also
referred to as a retrospective design Subjects
with the outcome are cases", while those without
the outcome are "controls
remember:
Cohort: exposure,
Case control: Outcome (usually retrospective
design)
(kabaliktaran)
Example diagram:
-Ability to study rare diseases
-Quick and less costly to perform
-Requires fewer subjects
-Best design to use existing historical data
pertaining to both outcome and exposures
-Can study different hypotheses
Disadvantages of a Case Control Study
• Information bias is a big problem
-Accurate memory may be an issue. (the recall
of the participants)
-Participants may provide incorrect information
-Medical records may be outdated
-Some outcomes require more definitive
assessment when used as a research outcome
compared to a diagnostic outcome
Choosing the Control Group
• Potential for selection bias, especially in the
control group
-Control group must appropriately represent
source population (kung saan kumuha ng
cases, doon din kukuha ng control group).
-Controls should be as comparable to the cases
as possible (they almost have the same
characteristics except of the presence of the
disease, because control group doesn’t have
disease. But the age, sex, etc must be almost
the same characteristics).
-Sample of controls should be collected using
random selection procedures (to limit biases).
-Individuals eligible for enrollment need to be
“at risk” to be a participant.

Enrolling Subjects in a Case

Direction of investigation:
from the past exposure, but their condition is at
the present.
Control Study Design
• Collection of cases and controls can occur
in one of two ways:
-Cases selected first, then controls selected (fast
paced, kapag nagmamadali)
-Cases and controls selected simultaneously
(slow paced)
• Considerations about which sequence to use
depends on length of time needed to enroll
cases.

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 BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II
Summary of a Case Control Study
Design
-Makes sense to the general public
-Allows study of rare diseases (no longer have
to wait several years for the outcome)
-Can be done quickly and inexpensively
-Primary limitation is information bias
-If controls are not selected carefully, study may
not be “referable" (external validity; establish
first the link before you are going to
investigated whether the exposure is a
causative factor to the disease) to the
intended population.
Collecting Data for a Cross Sectional
Assessment
-Cross sectional study Investigates the
relationship between existing exposure
characteristics and existing outcome
information in a group of enrolled subjects.
-Subjects are assessed for all variables as
they exist at the time the data is collected.
-happens in the present time (both the
exposure and outcome are in the present;
happened the same time).
-now the problem of this is, we don’t know what
came first; the exposure or outcome?
Advantages of a Cross Sectional Study
Design
-Popular due to ease, speed and low cost
-Allows investigators to gather data on larger
numbers of subjects in a shorter period of time
-Allows investigators to study many attributes of
enrolled subjects.
Disadvantages of a Cross Sectional Study
Design
• Investigator cannot know whether the
prevalence of outcome in study is an
incidence rate
-Risk cannot be determined
-Not a good choice for rare diseases or
exposures
-we cannot establish causality.
-Does not allow investigators to determine
whether exposure occurred before the outcome
or not.
Selecting the Sample and Tools for Cross
Sectional Study Design
-Study sample should be collected so that the
study can be applicable to the intended
population.
-Tools used to gather information must be
scientifically valid and well developed.
Remember:
Study design and tools should be parallel/
aligned with research objectives.
Summary of Cross-Sectional Study Design
• Allows investigators to compare
prevalence of outcome in groups of the
exposure variables.
• Often used to search for hypotheses to be
investigated later.
• A valuable method to collect data: can be
very useful to identify important potential
risk factors.
Summary
-A design may gather data (observational), or
intervene with subjects (experimental)
-Cohort study is most scientifically desirable
-Case control design compares attributes of a
group with the outcome of interest to a group
without the outcome
-Cross sectional design collects only current
information about subjects for both outcome and
exposure.
Study design Direction
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 BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II

Cross-sectional Present-present
Both exposure and
outcome are in the
present.
Prospective cohort Present-future
study Exposure is from the
present and the
outcome will be on the
future.
Retrospective Past-present
cohort study Identify first the
exposure then the
outcome.
Past-present
Case-control Identify first the
outcome then the
exposure.
Present-future
Randomized Do
controlled trial intervention/inoculation
then check on the
future for the
changes .

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BIOSTATS AND EPIDEMIOLOGY
1st SEMESTER
Prof: Efren Deocades II

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