Study Design
Dr/ Hanan Hasan Mohammed
Lecturer of public health and preventive
medicine
Objectives
• To understand the difference between descriptive
and analytic studies
• To identify the hierarchy of study designs, and the
strengths and weakness of each design
• To be able to apply different study designs to the
same research question
Study Design
interventional Observational
• clinical trials
•Lab
experiment
•Community Analytic Descriptive
intervention
• Cross sectional • Case report
• Case-control • Case series
•Quasi-
• Survey
randomized • Cohort studies
trials
Descriptive Analytic
Case report Cohort study
Study Designs
RCT
Case series Case-Control
study
Cross sectional
study Cross-sectional
study
Ecologic study
• Descriptive studies
– describe occurrence of outcome
• Analytic studies
– describe association between exposure
and outcome
Observational Studies
• non-experimental
• observational because there is no
individual intervention
• treatment and exposures occur in a “non-
controlled” environment
• individuals can be observed prospectively,
retrospectively, or currently
Observational/ Non- interventional study
• Descriptive study:
- Case report
- Case series
- Population study (survey)
• Analytical study:
- Cross-sectional
- Case-control
- Cohort
Descriptive Studies Develop
hypothesis
Increasing Knowledge of
Disease/Exposure
Investigate it’s
Case-control Studies relationship to
outcomes
Define it’s meaning
Cohort Studies with exposures
Test link
Clinical trials experimentally
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 and add to core
knowledge.
• Short time and low cost
• Disadvantages
• Cannot study cause and effect relationships
Analytical Studies
• Cross-sectional study
• Case control study
• Cohort study
Basic Question in Analytic Epidemiology
• Are exposure and disease linked?
Exposure Disease
Basic Questions in Analytic Epidemiology
• Look to link exposure and disease
– What is the exposure?
– Who are the exposed?
– What are the potential health effects?
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 Study
• Data collected at a single point in time
• Describes associations
• Prevalence
Prevalence vs. Incidence
Prevalence:
Point prevalence-
Number of all current cases (new & old) at a point in time
* 100
Population at the same point in time
Period prevalence
Number of all current cases (new & old) over a period of time
* 100
Mid year population at risk
Incidence rate
The no of new cases of a specific disease in a given time
Population at risk during that time * 1000
Cross-Sectional Study
Strengths
Prevalence (not incidence)
Fast/Inexpensive - no waiting!
May provide baseline data for cohort study
No loss to follow up
Associations can be studied
Weaknesses
Cannot establish cause-effect relationship (absence of
temporal relationship)
Weak evidence for association and worse for causation
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 Study
Disease
+ -
+ a b POR= ad
exposure bc
- c d
• Prospective study:
Forward looking study (Present ⇨ Future)
Risk factors/ Cause outcome of disease
• Retrospective study:
Backward looking study (Past ⇦ Present)
Disease Risk factors/ Cause
Case-Control Study
– Retrospective study
– Start with people who have a disease
– Match them with controls without disease
– Look back and assess exposures
Timeframe of Studies
• Retrospective Study - “to look back”,
looks back in time to study events that
have already occurred
time
Study begins here
Case-Control Studies: Strengths
• Good for rare outcomes: cancer
• Can examine many exposures
• Useful to generate hypothesis
• Fast & cheap
• Provides Odds Ratio
Case-Control Studies: Weaknesses
• High susceptibility to bias
• Uncertainty of temporal relationship between
exposure and outcome mainly in chronic diseases
with insidious onset
• Can only study one outcome
• Difficult selection of control group
• Ascertainment of exposure depends on recall
Measures of association
Disease
Yes No
Odds ratio:
Yes A B AD/BC
Risk 1= no diff.
facto
rs No C D case &
control
>1 = risk more
< 1 = risk less
Cohort Study
• Begin with disease-free patients
• Classify patients as exposed/unexposed
• Record outcomes in both groups
• Compare outcomes using relative risk
Timeframe of Studies
• Prospective Study - looks forward,
looks to the future, examines future
events, follows a condition, concern or
disease into the future
time
Study begins here
Prospective Cohort Study
Develop
Exposed disease
Stud to risk
y factors Do not develop
popu disease
latio comparison
n wo Develop
disea Not disease
exposed to
se
risk Do not
factors develop
disease
Present Future
Measures of Association
Disease Risk ratio (relative risk)
Yes No __ ___A___
_ A + B___
Yes A B _ ___ _C_____
Risk
Factor C+D
NO C D
RR = 4 means 4 times risk
in exposure than normal
Cohort Study: Strengths
• Strong design for confirming hypothesis
• Can calculate incidence data & relative risk
• Less bias than case control study
• Suitable for studying rare exposures
• Can measure multiple outcomes
• Can adjust for confounding variables
Cohort Study: Weaknesses
• Expensive
• Time consuming (long term study)
• Too long or too short follow up period
• Change of diagnosis along follow up
• Status change with long follow up
• Exposure may change over time
• Cohort attrition.
Experimental Studies
• treatment and exposures occur in a
“controlled” environment
• planned research designs
• clinical trials are the most well known
experimental design. Clinical trials use
randomly assigned data.
• Community trials use nonrandom data
Clinical Trials
This scientific study
Criteria
provides us ē the
information of the • Randomized
efficacy & usefulness of
a new drugs, vaccine, • Blinding
surgical procedures,
innovations & • Controlled
interventions etc.
What is Blinding?
• Single blind - participants are not aware of Rx group
• Double blind - both participants and investigators
unaware
• Triple blind – as persons who perform tests
–
Clinical Trial
R Treatment Outcomes
a Group
n
Study Co
d
Population mp
o
aris
m
on
i Control Outcomes
z Group
e
Phases of RCT (Drugs)
• Phase I: Healthy volunteers (limited no.)
• Phase II: On Patients (limited no.)
• Phase III: Large no of patients in multicenter evaluation
• Phase IV: Post marketing surveillance
Clinical Trials
Strengths:
– Best measure of causal relationship
– Best design for controlling bias
– Can measure multiple outcomes
Weaknesses:
– High cost
– Ethical issues may be a problem
– Compliance
Quasi study
• The interventional study does not fulfill the following
criteria:
- Randomization
- Controlled
- Blinding
## Chance of Biasness is more
Hierarchy of evidence
THANK YOU
