MIT World Peace University

Research Methods

Research Designs-Types
Shamim Mohammad, M.Phil., PhD.
1. To refresh different types of epidemiological
research designs
2. Merits and demerits of these research designs
 Study Designs
Observational Quasi
Experimental Experimental

Descriptive Analytical

Case report Case control

Case series Cohort

Cross section

Ecological
 Observational studies

• Descriptive studies:
• Describe occurrence of outcome.

• Analytic studies:
• Describe association between exposure and outcome.
 Cross-sectional Studies

• It describes the relationship between diseases and other

factors at one point in time in a defined population.

• It is just like a “snapshot”.

• It lacks any information on timing of exposure and outcome.

• It used mainly in the prevalence of diseases.

 Disadvantages Cross-sectional Studies

• Weakest observational design, (it measures prevalence, not

incidence of disease).
• The temporal sequence of exposure and effect may be difficult
or impossible to determine.
• Usually give no evidence about when the disease occurred.
• Not suitable for quickly emerging diseases and rare events.
 Case Report Study

• It is a detailed report of the symptoms, signs, diagnosis,

treatment, and follow-up of an individual patient.

• It describes an unusual or novel occurrence.

• It is uncontrolled observations of patient.

• Usually consists of 1-3 patients.

• E.g. Frederick Treves reported on "The Elephant Man"
Case Report Study Cont.

• 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 Study

• It the same as case report but consists of collections of

reports of group of patients ( usually more than 3).

• Experience of a group of patients with a similar diagnosis.

 Case Series Study Cont.

• 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.
 Ecological Studies

• They are studies of risk-modifying factors on health or other

outcomes on defined populations (geographically or temporally).

• John Snow and cholera outbreak in London. He used a map of

deaths from cholera to determine the source of infection.

• The data are analyzed at the population or group level, rather than
individual level.
Analytic studies
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?
• What approach will you take to study the
relationship between exposure and effect?
 Case-Control Studies

It is an “observational” design comparing exposures in

diseased cases versus healthy controls from same population.

It is the most feasible design where disease outcomes are

rare.
 Case-Control Design
Factor Present
Cases
Factor Absent (disease)
Study
Factor Present groups
Controls
(no disease)
Factor Absent

past present

Time

Study begins here

 Example of Case Control Study

Occupational exposures to vapors, gases, dusts, and fumes

(VGDF) increase risk of chronic obstructive pulmonary disease
(COPD).

This case-control study estimated the risk of COPD attributable

to occupational exposures to (VGDF) among construction
workers.
 Advantages of Case Control Studies

• Less expensive and time consuming.

• Efficient for studying rare diseases.

 Disadvantages of Case Control Studies

1.Can not calculate incidence rates.

2.Not useful in rare exposure.

3.Liable to bias- recall bias

4.Difficulty in finding control

5.Do not demonstrate causation

 Types of Bias in case control studies

Selection bias. Recall bias Information bias

Poor selection Poor memory- Poor designed

criteria selective recall tools for
collecting data
 Cohort Studies
• A cohort is a group of people who share a common
characteristic or experience within a defined period.
• It compares individuals with a known risk factor or
exposure with others without the risk factor or exposure.
• Two groups of subjects:
• Exposed: having the exposure under consideration;
• Non-exposed: not exposed to the factor under
consideration.
 Cohort Studies cont.

• Subjects are then followed prospectively over time to

identify those who develop the disease.
• Incidence will be compared between the two groups
to assess the association between the exposure & the
outcomes.
• Data usually collected prospectively (sometimes it can
be retrospective).
 Cohort
Defined
population Outcome (+)

Exposed

Outcome (-)
Sample
Outcome (+)
Not exposed

Outcome (-)

Direction of Study
Time
 Cohort Studies - Examples

In 1951, a British Study, a cohort that included

both smokers (the exposed group) and non-
smokers (the unexposed group) and followed up
to see the outcome (lung cancer).
The findings:
The exposure to smoking was associated with
lung cancer.
 Cohort Studies Advantages

• Results can be analyzed with well known

statistical tools.
• Populations of participating individuals are
clearly identified.
• Can study several outcomes for each exposure.
 Cohort Studies Disadvantages

• Expensive in terms of time and money.

• Does not reveal causation but build temporal
relationship.
• Problems of loss to follow-up
• Not appropriate for rare outcome (disease).
Experimental Studies
 Experimental Studies
• The investigator can “control” the exposure.
• Treatment and exposures occur in a “controlled”
environment.
• It is a planned research design.
• Clinical trials are the most well-known experimental
design.
• Clinical trials use randomly assigned data.
• Community trials use nonrandom data.
 Experimental Studies Cont.

• In an experiment, we are interested in the consequences of some

treatment on some outcome.

• The subjects in the study who actually receive the treatment of

interest are called the treatment group.

• The subjects in the study who receive no treatment, or a different

treatment are called the comparison group.
 Experimental
Studies

Outcome (+)

Intervention
No outcome
Study
Randomization
population
Outcome (+)
Control
Baseline No outcome

Future
Study begins here Time
(baseline point)
 Experimental Studies
These studies evaluate the effects of an intervention
• Types of interventions:
• Behavior modification (eg. a walking program to
improve weight loss)
• Drug (eg. a new investigational drug or studying a
drug for off-label use – subject to FDA regulations)
• Device (eg. a new investigational stent)
• Strength: Can demonstrate causality
 Phases of experimental studies
• Phase I:
Unblinded studies of a small number of healthy volunteers to test safety of
treatment (can sometimes use people with the disease)
• Phase II:
Randomized studies of relatively small number of people with the disease to test
dose ranges and/or efficacy of treatment
• Phase III:
Randomized studies of large number of people with the disease to test efficacy of
treatment on pre-selected outcomes
• Phase IV:
Large experimental studies or observational studies conducted after treatment has
been approved by the FDA to assess performance of treatment (called Post-Market
Studies)
 Phase 1
•Drug is tested for its interaction with the human system.
How is the drug absorbed
How is the drug distributed in the body
How is the drug metabolized by the body

•Trials usually involve normal, healthy volunteers and take

about a year to complete.
 Phase 2

• Pilot to begin to define the effectiveness and

safety of the drug in patients with the disease or
condition to be treated, diagnosed or prevented.
• Testing the various doses of the drug and dosing
regimens
 Phase 3

• Expanded clinical trials

• Designed to …
Gather additional evidence of effectiveness for
specific interactions
Better understand safety and drug-related
adverse effects
 Phase 4
Studies that occur after a drug has received approval from
the U.S. Food and Drug Administration to be marketed
Performed to determine the incidence of adverse
reactions
Determine the long-term effect of the drug
To study a patient population not previously studied
For marketing comparisons against other products and
users
Testing on Humans
 Post Marketing Surveillance
Monitor the ongoing safety of marketed drugs by reassessing
drug risk based on …
 New data collected after the drug is marketed
 By recommending ways of trying to most appropriately
manage that risk
 Includes adverse reaction reporting by the medical
community of the pharmaceutical company that
markets the drugs
 Periodic sampling and testing of the drug
 Periodic inspections of the manufacturing and
distribution process
 Randomized Controlled Trials (RCTs)

• It is the “gold standard” of research designs.

• A design with subjects randomly assigned to “treatment” and
“comparison” groups.
• It provides most convincing evidence of relationship between
exposure and effect.
• It is not possible to use RCTs to test effects of exposures that are
expected to be harmful, for ethical reasons.
 Advantages of Randomized Controlled Trials (RCTs)

• This study design can make causal inferences, i.e. it is the

strongest empirical evidence of a treatment's efficacy
• Considered the gold standard.
Disadvantages
Disadvantagesof
ofRandomized
RandomizedControlled
ControlledTrials
Trials(RCTs)
(RCTs)

• It is very expensive.
• Not appropriate to answer certain types of questions.
• It may be unethical, for example, to assign persons to certain
treatment or comparison groups.
 Comparison of epidemiological studies
Case-
Cohort control Cross- Ecological Applications
sectional
- +++++ - ++++ Investigation of rare disease (outcome)
+++++ - - ++ Investigation of rare cause (exposure)
+++++ ++ - + Testing multiple outcomes
+++ ++++ ++ ++ Study of multiple exposures

+++++ - - ++ Direct measurement of incidence

- +++ - - Investigation of long latent periods

+ indicates the degree of suitability

 Timeframe of Studies

• Prospective Study:

• It looks forward, looks to the future, examines future events,

follows a condition, concern or disease into the future.

Time

Study begins here

 Timeframe of Studies Cont.

• Retrospective Study:

• It looks backward in time to study events that have already

occurred.

Time

Study begins here

 Epidemiological
Studies
Descriptive Case-control Cohort Clinical trials
Studies Studies Studies

Develop Investigate it’s Define it’s Test link

hypothesis relationship to meaning experimentally
outcomes with exposures