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Study Designs
Individual
Group based
based

Ecological Observational Experimental

Descriptive Analytical Clinical Trials Program Trials

Cross-sectional Cross-sectional Longitudinal

Cohort Case-Control
1 (prospective) (retrospective) 2

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The major study designs differ in

several respects:
• Unit of observation
• Manipulation of exposure
• Randomization
• Direction of investigation
• Timing of data collection
• Data collection methods

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2012 Forrest & Miller.

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The major study designs differ in Unit of observation

several respects:
• Unit of observation Group based
• Manipulation of exposure (ecological)
• Randomization
• Direction of investigation
• Timing of data collection
• Data collection methods Individual
based
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ECOLOGICAL STUDY
BEWARE!!!

of

Ecological Fallacy
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Ecological Fallacy Hypothetical associations of income with

BMI within and between countries
“Mistaken assumption that a statistical
association observed between two group-level
variables is equal to the association between the
corresponding variables at the individual level”
(Gail & Benichou, 2001)

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Hypothetical associations of income with

BMI within and between countries

Individual-based Studies

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The major study designs differ in Manipulation of exposure

several respects:
• Unit of observation Experimental
Investigator observes
• Manipulation of exposure occurrence of condition/s in
Investigator exercises
• Randomization “self”-assigned groups of
control over allocation of
people
• Direction of investigation exposure
• Timing of data collection
• Data collection methods Observational
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Observational Studies Experimental Studies

• Investigator observes occurrence of condition/s in
“self-assigned” groups of people; exposure not
assigned
• Carried out in more natural settings - “natural
experiments”
• Often most practical and feasible
• Less control over study situation; results more
susceptible to distorting influence
• Investigator exercises control over
allocation of exposure
• More powerful than observational studies
for testing etiological hypotheses
• For ethical reasons the possibilities of
conducting experiments in humans is
limited

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• Is exposure of interest controlled by

The major study designs differ in investigator?
several respects: • In controlling the exposure, are study
participants randomly assigned to different
exposure conditions?
• Unit of observation
• Manipulation of exposure Exposure
Randomization? Study type
• Randomization control?
• Direction of investigation
• Timing of data collection
• Data collection methods

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• Is exposure of interest controlled by • Is exposure of interest controlled by

investigator? investigator?
• In controlling the exposure, are study • In controlling the exposure, are study
participants randomly assigned to different participants randomly assigned to different
exposure conditions? exposure conditions?

Exposure Exposure
Randomization? Study type Randomization? Study type
control? control?
N N Observational N N Observational

Y Y Experimental

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• Is exposure of interest controlled by Did investigator

investigator? assign exposures?
• In controlling the exposure, are study
participants randomly assigned to different
exposure conditions? yes no

Exposure Experimental Observational

Randomization? Study type
control? Study Study
N N Observational

Y Y Experimental
Quasi-
Y N
experimental
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Did investigator Did investigator

assign exposures? assign exposures?

yes
no
Experimental
Study
Experimental Observational
Study Study
randomize allocation?

yes no
Randomized Non-
Controlled Randomized
Trial Controlled Trial
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Did investigator Descriptive Study

assign exposures?
• Sets out to describe a situation
no ex. Distribution of depression in a population
Observational in relation to sex, age and other characteristics
Study

comparison group? Analytical Study

yes no • Sets out to test hypotheses or detect
Analytical Descriptive associations
Study Study ex. Identify factors that explain higher rates of
25 depression among women 26

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Did investigator assign exposures?

Descriptive Study no
• Often no a-priori hypothesis Observational Study

comparison group?

Analytical Study yes no

Analytical Descriptive
• Must have clear and measurable Study Study
hypothesis direction?
• At least 1 dependent (outcome) variable
and 1 independent (“exposure”) variable Cohort
Case- Cross-
Control sectional
Study Study
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Design of a PROSPECTIVE Study

Directionality of investigation
Time
retrospective (case-control )
Direction of inquiry
Exposure Outcome
START WITH:
prospective (cohort) D+
Exposed
D-
POPULATION
D+
Unexposed
D-
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Prospective Study Design of a COHORT Study

• Step 1 in a prospective study design: identify Time

relevant group/s of people and collect
Direction of inquiry
information about their exposure history
• Step 2: Follow these people over time and
D+
measure incidence of outcome/s of interest Exposed
D-
POPULATION
D+
Unexposed
D-
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Design of a COHORT Study 2*2

Time
if only life was so simple…

Direction of inquiry Outcome Status

D+ present absent
Exposed
Disease D-
POPULATION free exposed
people D+ Exposure
Unexposed
D- Status not
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Design of a RETROSPECTIVE Study Retrospective design

Time
Direction of inquiry Outcome Status

present absent Total

START WITH:
E+ CASES exposed
Exposure
(with
E- Status not
disease)
POPULATION exposed
E+ CONTROLS
(without Total
E-
disease)

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Did investigator assign exposures? Design of a CROSS-SECTIONAL Study

no (prevalence study)
Observational Study
START WITH:
comparison group?
DEFINED
yes no POPULATION
Analytical Descriptive
Study Study

direction? gather data on exposure and disease

Case- Cross- Exposed+ Exposed+ Exposed - Exposed -

Cohort sectional
Study
Control Diseased+ Diseased - Diseased+ Diseased -
Study Study

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Cross-sectional design Cross-sectional design

Outcome Status Outcome Status

present absent Total present absent Total

exposed exposed
Exposure Exposure
Status not Status not
exposed exposed

Total Total n

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Cross-sectional design Cross-sectional (prevalence) study

Outcome Status • Usually involves a representative (random)

sample of a dynamic population
present absent Total • Examines exposures and outcomes
simultaneously
exposed ? ? • Based on prevalence data
Exposure
Status not
exposed ? ?

Total n

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Design of a Clinical Trial

Relationship between prevalence and
incidence SAMPLE

non-participants

Randomization to groups (2…n)

Intervention Control
Group Group

Lost-to-follow-up
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Measure outcome Measure outcome

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Evaluation Studies Program Review

• Appraise the value of health care • Evaluates a specific program provided to a
specific population, community, group of
• 2 types: patients
– Program reviews
• Concerned with the “welfare” of that population
– Program trials and directed at guiding decisions regarding the
program under evaluation
• Similar to a physician’s review of treatment
given to an individual patient: concern for the
patient rather than testing the effect of treatment

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Program Review (cont.)
• Usually descriptive; no control group
• Assumes the program activities are beneficial
and evaluates whether activities are conducted
as planned
• Information on outcomes can be included under
the assumption that changes are a result of the
program
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Program Trial
• Evaluates a type of service provided to a
population, community, group of patients
• Concerned with the program’s “value” and
generalizibility of program’s effectiveness to other
populations
• Similar to a clinical trial of a new drug, findings
must be relevant not only to the participants of the
trial
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Program Trial (cont.)

• Focus on “Outcomes”
• Analytical investigation requiring a control
population in order to account for outside
influences
• Program planning and implementation must
allow for this from the outset

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