Study Design

in
Research
Dr.G.Pitchaimani B.P.T., M.Sc., PhD
Assistant Professor
Department of Allied Health Sciences
Sri Ramachandra Institute of Higher Education and Research
STUDY DESIGN

DESCRIPTIVE STUDIES
 Descriptive Studies

Case Reports

Case Series

Population Studies
 Includes activities related to characterizing the
distribution of diseases within a population

Person

Time Place

Descriptive
Study

Think of this as the standard dimensions used to

track the occurrence of a disease.
 Descriptive Studies
 Other Name Case-Series/Population

 Unit of Study Case/Individuals

 Study Question What is happening

 Direction of Inquiry -
 Descriptive Research Design
 Describe facts
 Discover new facts
 Not invent new theory and methods
 Largest effort given on data collection
 It answers questions: satisfy curiosity
 Solve problems
 Case Series
Advantages
 Easy to do
 Excellent at identifying unusual situation
 Good for generating hypotheses
Disadvantages
 Generally short-term
 Investigators self-select (bias!)
 no controls
STUDY DESIGN

ANALYTICAL STUDIES
 Analytical Observational Studies
 Cross-Sectional Study

 Case-Control Study

 Cohort Study
 Cross-Sectional Study
 Data collected at a single point in time (Snapshot in
time)
Example:
Cholesterol measurement and ECG measured at
same time
 Describes associations
 Determines prevalence at a point in time
 Therefore, C-S is a prevalence study
 Cross-Sectional Study

 Other Name Prevalence Study

 Unit of Study Individual

 Study Question What is happening

 Direction of Inquiry -
 Cross-Sectional Study
Study Design
Exposed to Factor
Diseased
Not Exposed to
Factor
Population
Exposed to Factor
Non-Disease
Not Exposed to
Factor
 Cross-Sectional Study

Disease
Exposure Yes No Total
Yes a b a+b
No c d c+d
 Cross-Sectional Study

Defined Population

Regular Meditation Not doing Meditation

Prevalence of DM Prevalence of DM

Time Frame = Present

 Cross-Sectional Study
Example:
Out of 1000 population if 100 were doing meditation
regularly & out of that only 2 were having DM.
Remaining 900 were not doing meditation at all, out of
that 220 were having DM.

DM
Meditation Yes No Total
Yes
No
 Objectives of a
Cross-Sectional Study

To find out association

 Advantages of
Cross-Sectional Study
 Short term
 Fewer resources required
 Less statistical analysis
 More easily controlled
 Design less complex
 Provide relationship between attributes of disease and
characteristics of various groups, e.g. elderly group
 Data is useful for planning of health services and medical
programs
 Disadvantages of
Cross-Sectional Study
 Represent only those who are surveyed
 Identify prevalence, not incidence necessarily
-excludes cases that died before study was done
 Show association with survival - not risk of development
 People who are ill may not show up for survey
-Healthy Person Effect
 Often, not possible to establish temporal relationship
between exposure and onset
-e.g. does high cholesterol precede CHD?
 Not too effective if disease levels are low, as difficult to
establish a causal relationship
ANALYTICAL STUDY

CASE CONTROL STUDY

 Case-Control Study
Frequently used study design
Participants selected on the basis of whether or not
they are DISEASED (remember in a cohort study
participants are selected based on exposure status)
Those who are diseased are called CASES.
Those who are not diseased are called CONTROLS.
Look back and assess exposures
 Case-Control Study
Control
A control is a standard of comparison (confounded
with variability but without effect) for
–Effects
–Variability
 Case-Control Study

Other Name Retrospective Study

Unit of Study Cases/Control

Study Question What has happened

Direction of Inquiry =F O
 Case-Control Study
Study Design

Exposed
Cases
Not Exposed

Exposed
Control

Not Exposed
 Case-Control Study
Regular Cases
Meditation
Patients
No with DM
Meditation

Regular Control
Meditation Person
without DM
No
Meditation

Past Present
MATCHING
CHARACTERISTICS OFTEN USED
– age
– gender
– body mass index (BMI
– smoking status
– marital status
 Objective of a
Case-Control Study

To find out association

To assess Risk Ratio

 Case-Control Study: Strengths
Good for rare outcomes: cancer

Can examine relation of exposures to disease

Useful to generate hypothesis

Fast

Cheap

Provides Odds Ratio

 Case-Control Study: Weaknesses
Cannot measure

– Incidence

– Prevalence

– Relative Risk

Can only study one outcome

High susceptibility to bias

 Example: Hypothetical data

Cases Control
Exposed 141 133
Unexposed 1250 4867
Total 1391 5000

ODDS RATIO = 141 * 4867 = 4.13

133 * 1250
Interpretation of the Odds Ratio…
If:
OR = 1 then exposure is NOT related to
disease
OR>1 then exposure is POSITIVELY related
to disease
OR <1 then exposure NEGATIVELY related
to disease
 Interpretation
The odds that those with the outcome had
the exposure is 4.13 times greater than those
who do not have the outcome
ANALYTICAL STUDY

COHORT STUDY
 Cohort Study
Begin with disease-free individuals

Classify patients as exposed/unexposed

Record outcomes in both groups

Compare outcomes using relative risk

When is a cohort study warranted?

When good evidence suggests an association

of a disease with a certain exposure or
exposures.
 Cohort Effect
Changes and variation in the disease or health
status of a study population as the study group
moves through time.
“Generation effect”
 Types of Cohort Study
Prospective (concurrent)

Retrospective (historical)

Restricted (restricted exposures)

 Types of Cohort Study
 Prospective
- cohort characterized by determination of exposure
levels (exposed vs. not exposed) at baseline (present)
and followed for occurrence of disease in future
- Groups move through time as they age
 Retrospective
- makes use of historical data to determine exposure
level at some baseline in the past and then determine
subsequent disease status in the present.
 Restricted
- limited exposure, narrow behaviour (e.g. military)
 Types of Cohort Study

 Other Name
Prospective Study / Follow-up Study/Incidence Study

 Unit of Study Individual

 Study Question What is happening

 Direction of Inquiry =F O
 Types of Cohort Study
Study Design Diseased
Exposed to
Factor
Non-
Diseased
Cohort
Diseased
Not Exposed
to Factor
Non-
Diseased
 Cohort Study: Strengths
Can measure multiple outcomes

Can adjust for confounding variables

Can calculate Attributed Risk

 Cohort Study: Weaknesses
Expensive

Time consuming

Cannot study rare outcomes

Confounding variables
 Steps for Cohort Study
Identify geographically defined group
Identify exposed subjects and not exposed subjects
Follow over a specific time
Record the fraction in each group who develop the
condition of interest
Compare these fractions using RR, AR or OR
 Objectives of a Cohort Study
 To find out association

 To assess Risk Ratio

 To find out Relative Risk

 To find out Attributed Risk

 Prospective Cohort Study

DM
No Meditation
No DM

Cohort

DM
Regular Meditation
No DM

Present Future
Measurements of Association

Cohort Study Case-Control Study

Significance Test Significance Test

ODD’s Ratio ODD’s Ratio

Relative Risk

Attributable Risk
STUDY DESIGN

EXPERIMENTAL STUDIES
 Experimental Studies
 Clinical trials provide the “Gold Standard” of
determining the relationship between factor and
the event.
 It is a collection of research designs which use
manipulation and controlled testing to understand
causal processes.
 Generally, one or more variables are manipulated
to determine their effect on a dependent variable.
 Aim
The aim of experimental research is to investigate
the possible cause and effect relationship by
manipulating one independent variable to influence
the other variable in the experimental group and by
controlling the other relevant variables and
measuring the effects of the manipulation by some
statistical means.
 Types of Experimental Study

As per Randomization:

 Randomized Control Trials (RCT)

 Non – Randomized Trials

As per Construction:
– Simple
– Cross-Over Study Design
As per Study Area:
– Field Trials
– Clinical Trials
– Laboratory Trials
 Blinding
Good practice: factors that can affect the evaluation
of outcome should not be permitted to influence
the evaluation process
Single-blind
 Patient or evaluator (either of one) is blinded as
to intervention
Double-blind design
 Neither patient nor outcome evaluator knows
treatment to which patient was assigned
 Experimental Study
 Other Name Intervention Study
 Objective
To know the effect of intervention
 Unit of Study
Individual meeting entry criteria
 Study Question
What is happening after intervention in both groups
 Direction of Inquiry I E
 Experimental Study

Positive
Group 1 Outcome
Intervention
Cases Negative
Outcome
Experimental
Study
Positive
Group-2 Outcome
Placebo
Control Negative
Outcome
 Cross Over Design
Cases Meeting
Group 1 Group 2
Entry Criteria

Intervention 1 Intervention 2

Positive Negative Positive Negative

Outcome Outcome Outcome Outcome

Cross Over

Group 2 Group 1

Intervention 1 Intervention 2

Positive Negative Positive Negative

Outcome Outcome Outcome Outcome
Steps of Experimental Study
Drawing up a Protocol

Reference Population

Sample Population

Exclusions

Randomization

Experimental Group Control Group

Manipulation / Intervention

Follow-Up

Assessment of Outcome
 Characteristics or Features of
Experimental Design

1. Manipulation
2. Control
3. Randomization
 Types of Experimental Designs

True-Experimental (Simple)

Quasi-Experimental

Pre-Experimental
 Types of Experimental Designs

Randomization, Control and Manipulation

True experimental : All 3 R C M

Quasi experimental: M + R or C

Pre experimental : M, no R & no C

 Experimental Study
Advantages
Best establishes cause-and-effect relationships

Disadvantages
 Artificiality of experiments
Feasibility
Ethical Issues (Unethical)
Experimental Studies

RANDOMIZED CONTROLLED TRIALS

 Randomized Controlled Trials
 Similar groups of individuals from same source
population are allocated at random to receive or
not to receive an intervention, then observed for
occurrence of outcome(s).

Subjects Experiment
Outcome
with al Group
condition Control
of interest Outcome
Group
 Randomized Control Trials (RCT)
Before and After Comparison

Comparison with Placebo

Comparison Of two medicine/procedure/tests

Comparison Of > two medicine/procedure/tests

Steps:
1.Select a sample from the population
2.Measure baseline variables
3.Randomly assign two active interventions
4.Apply interventions blindly
5.Follow-up the cohorts
6.Measure outcome variables blindly
 Randomized Controlled Trial :
Advantages
 Comparability due to randomization and same
effect of known and unknown confounders gets
eliminated
 Experiments provide strong evidence of cause and
effect.
 Allows standardization of eligibility criteria,
manoeuvre and outcome assessment.
 Allows use of statistical methods with few inbuilt
assumptions.
 Randomized Controlled Trial :
Disadvantages
 May be expensive in terms of time, money and
people.
 Many research questions are not suitable due to
ethics, likely co-operation or rarity of outcome.
 To a greater or lesser extent RCT tends to be an
artificial situation.
Thank You
QUALITATIVE RESEARCH
 Qualitative Research
 Not every thing can be quantified.
 Some valuable ideas, opinions, perceptions,
experiences, behaviours, qualities can be described
only in words
 These subjective things are shared between people,
but the meanings may be distorted in the process of
communication and recording
 Although subjective, these aspects often add richness
and depth
 The art of the doctor and the experience of being
human are aspects that need a qualitative approach
to investigate/research properly.
 Definitions
Quantitative Research - investigation in which the
researcher attempts to understand some larger
reality by isolating and measuring components of
that reality without regard to their contextual
setting.
Qualitative Research - investigation in which the
researcher attempts to understand some larger
reality by examining it in a holistic way or by
examining components of that reality within their
contextual setting.
 Qualitative Research
‘Qualitative Research… involves finding out what
people think, and how they feel - or at any rate,
what they say they think and how they say they
feel. This kind of information is subjective. It
involves feelings and impressions, rather than
numbers’
 Qualitative Research
 Human understanding and interpretation define
reality
 Complex reality can be understood and not as
simply a sum of its parts
 Goal of research is to examine complex phenomena
to define the reality within
 To be meaningful, inquiry must be holistic and
contextual
Qualitative Research
 Characteristics of
Qualitative Research
 Purpose is understanding
 Oriented toward discovery
 Uses subjective data
 Extracts meaning from data
 Interprets results in context
 Focus is holistic
 Advantages of
Qualitative Research
 In-depth Examination of Phenomena
(Phenomenological Study/Research)
 Uses subjective information
 Not limited to rigidly definable variables
 Examine complex questions that can be impossible
with quantitative methods
 Deal with value-laden questions
 Explore new areas of research
 Build new theories
 Disadvantages of
Qualitative Research
 Subjectivity leads to procedural problems
 Replicability is very difficult
 Researcher bias is built in and unavoidable
 In-depth, comprehensive approach to data
gathering limits scope
 Labour intensive, expensive
 Not understood well by “classical” researchers