Part IV: 06 Sep 2023 Dr Kalyani Addya

Study Designs in Epidemiologic

Research
Descriptive Analytic

Case report Cohort study

RCT

Case series Case-Control

study
Descriptive
Epidemiology Case-Crossover
study

Cross-sectional
study

Before-After
study

Ecologic study
 Timeframe of Studies
• Prospective Study - looks forward/
future, examines future events, follows
a condition, concern or disease into the
future

time

Study begins here

 Timeframe of Studies
• Retrospective Study - “to look back”,
looks back in time to study events that
have already occurred

time

Study begins here

 Descriptive Studies Develop
hypothesis
Increasing Knowledge of

Investigate it’s
Disease/Exposure

Case-control Studies relationship to

outcomes (OR)

Define it’s meaning

Cohort Studies with exposures (RR)

Test link
Clinical trials Experimentally (RCT)
Descriptive Studies
 Case Reports
• Detailed presentation of a single case or
handful of cases
• Generally report a new or unique finding
• e.g. previous undescribed disease
• e.g. unexpected link between diseases
• e.g. unexpected new therapeutic effect
• e.g. adverse events
 Case Series
• Experience of a group of patients with a
similar diagnosis
• Assesses prevalent disease
• Cases may be identified from a single or
multiple sources
• Generally report on new/unique
condition
• May be only realistic design for rare
disorders
 Case Series
• Advantages
• Useful for hypothesis generation
• Informative for very rare disease with few
established risk factors
• Characterizes averages for disorder

• Disadvantages
• Cannot study cause and effect
relationships
• Cannot assess disease frequency
 Case Report One case of unusual
findings

Multiple cases of
Case Series findings

Descriptive Population-based
Epidemiology Study cases with denominator
Analytical Studies
 Analytic Epidemiology
• Observational Studies
– Group data
• Ecologic
– Individual data
• Cross-sectional
• Cohort
• Case-control
• Case-crossover
• Experimental Studies
– Randomized controlled clinical trials (RCT)
– Community trials
 Observational Studies
• non-experimental
• there is no individual intervention
• treatment and exposures occur in a
“non-controlled” environment
• individuals can be observed
prospectively, retrospectively, or
currently
 Cross-sectional studies
• An “observational” design that surveys
exposures and disease status at a single point
in time (a cross-section of the population)

time
Study only exists at this point in time
Cross-sectional Design
factor present
No Disease
factor absent
Study
population
factor present
Disease
factor absent

time
Study only exists at this point in time
 Cross-sectional Studies
• Used to study conditions that are relatively
frequent with long duration of expression
(nonfatal, chronic conditions)
• Measures prevalence of disease
• Example: community surveys
• Not suitable for studying rare or highly fatal
diseases/ disease with short duration of
expression
• Similar to cohort study but collects data
from one point of time for a short period
 Cross-sectional studies
• Disadvantages
• Weakest observational design,
( measures prevalence, not incidence of
disease). Prevalent cases are survivors
• The temporal sequence of exposure and
effect may be difficult or impossible to
determine
• Usually do not know when disease occurred
• Rare events/quickly emerging diseases are a
problem
 Epidemiologic Study Designs
• Case-Control Studies
– an “observational” design comparing
exposures in disease cases vs. healthy
controls from same population
– exposure data collected retrospectively
– most feasible design where disease
outcomes are rare
– E.g.- Association of colon cancer with
high fat diet
factor present
Cases
factor absent (disease)
Study
population
factor present Controls
(no disease)
factor absent
present
past

time

Study begins here

 Case-Control Study
• Strengths
– Less expensive and less time consuming
– Efficient for studying rare diseases
• Limitations
– Inappropriate when disease outcome for a specific
exposure is not known at the beginning of study
– Exposure measurements taken after disease
occurrence
– Disease status can influence selection of subjects
Hypothesis Testing: Case-Crossover Studies
• Study of “triggers” within an individual
• ”Case" and "control" component, but
information of both components will come
from the same individual
• ”Case component" = hazard period which is
the time period right before the disease or
event onset
• ”Control component" = control period which
is a specified time interval other than the
hazard period
 Epidemiologic Study Designs
• Cohort Studies
– an “observational” design comparing
individuals with a known risk factor or
exposure with others without the risk
factor or exposure
– looking for a difference in the risk
(incidence) of a disease over time
– best observational design
– data usually collected prospectively (some
retrospective)
 VIDEOS
• Confounding
• Chance
• Bias
 disease
Factor
Study present no disease
population
free of
disease Factor disease
absent
no disease
present
future

time
Study begins here
 Prospective Cohort study

Exposed Outcome

Measure exposure
and confounder
variables

Baseline Non-exposed Outcome

time

Study begins here ( follows a condition/ concern/ disease

into the future
 Retrospective Cohort study

Exposed Outcome

Measure exposure
and confounder
variables

Baseline Non-exposed Outcome

time
Study begins here , looks back in time to study events
that have already occurred
 Cohort Study
• Strengths
– Exposure status determined before disease
detection
– Subjects selected before disease detection
– Can study several outcomes for each exposure

• Limitations
– Expensive and time-consuming
– Inefficient for rare diseases or diseases with
long latency
– Loss to follow-up
 Experimental Studies

• treatment and exposures occur in a

“controlled” environment
• planned research designs
• clinical trials are most well known
experimental design.
• Clinical trials use randomly assigned
data.
• Community trials use nonrandom data
 Experimental Studies
• Types of experimental studies
• Clinical Trial (Therapeutic trial)
• Community Trial
• Field trial
 Experimental Studies
• The subjects who receive the treatment of
interest are called the treatment group.
• The subjects who receive no treatment or
a different treatment are called the
comparison group.
• Akin to laboratory experiments except
living populations are the subjects
 Epidemiologic Study Designs

• Randomized Controlled Trials (RCTs)

– Also called intervention / experimental studies

– the “gold standard” of research designs
– Subjects randomly assigned to “treatment” and
“comparison” groups
– provides evidence of relationship between exposure
and effect
– Not used to test effects of exposures that are
expected to be harmful, for ethical reasons
RANDOMIZATION outcome
Intervention
no outcome
Study
population
outcome
Control
no outcome
baseline
future

time
Study begins here (baseline point)
 Advantages
• Scientifically ideal method
• Removes a large number of biases related to
selection and measurement
• Controls for confounding through
randomization
• Ensures temporal relationship between
exposure and outcome
 Disadvantages
• Very expensive
• Study of risk / prognostic factors random
allocation of humans into two groups is not
possible
• Ethics becomes a very important issue
 When to use?
• Studying the efficacy of a preventive
procedure
• Studying the efficacy of a therapeutic
procedure
• Studying the efficacy of a health care
system
• Random allocation is
– Feasible
– Ethical
 Conducting a RCT
• Step 1: Clearly define the research question
and its background significance
• Step 2:
– Clearly define your reference population and
study population.
– Specify the general settings of the study
– Specify the time frame
 Conducting a RCT
• Step 3: Clearly specify the exclusion criteria
• Step 4: Specify the intervention and the scales of
measurement
• Step 5: Specify the outcome variable of interest
– Occurrence or non occurrence of an event
– Consider the entire spectrum of the disease
 Conducting a RCT
• Step 6: Specify the important potential
confounding factors
– Demonstrate no significant difference between
the groups in respect of imp confounders
• Step 7: Calculate the sample size
– Outcome on dichotomous scale
– Outcome on continuous scale
 Conducting a RCT
• Step 8: Sampling
– Select the study subjects by random sampling
• Simple random
• Systematic random
• Stratifies random
• Step 9:
– Subject the study sample to random allocation
 Conducting a RCT
• Step 10: Enunciate the stoppage rules
– Continuously analyse data
– Stop on clear statistical evidence
• Step 11: Organize for field work & data
collection
– Take administrative sanction
– Clearance from ethical committee
– Funds, logistics, equipment
– Training of data collectors
– Proper pretesting: Pilot Study
 Conducting a RCT
• Step 12: Take informed consent
– Clearly explain the purpose and scope
– Potential benefits and hazards
– Possibility of being assigned to study or control
group
– Consent without fear, prejudice, coercion
– Exclude those who don’t give consent
– Compare those who gave consent with those
who did not
 Conducting a RCT
• Step 13: Randomise
– Two groups: Drug and placebo
– Three groups: Drug, placebo, standard
treatment
– More groups: Different doses of drug
– Do a baseline comparison to check whether
there is any difference between the groups
 Conducting a RCT
• Step 14: Ensure Blinding
– Single: Subjects not aware in which gp
• Reporting bias removed
– Double: Subject & investigator not aware
• Reporting and ascertainment bias removed
– Triple: Subject, investigator and data analyser
not aware
 Conducting a RCT
• Step 15: Administer the intervention and
follow up
– Similar action for both intervention and placebo
– Duration of followup depends on outcome of
interest
– Constantly watchout for drop outs
– Lookout for side effects
 Conducting a RCT

• Step 16: Final assessment of outcome

– During followup and at the termination
– Positive results
– Negative results
DISCUSSION