Study

Design
Chapter 6
 Study Design
A study design is the framework or strategy used to carry out
research in a systematic and structured manner to answer a specific
research question. It defines:
• How data will be collected • From whom it will be collected
• When it will be collected • How the data will be analyzed

The main purpose is to:

• Minimize bias • Efficiently use time and resources
• Ensure replicability • Ensure ethical standards are met
• Increase validity and reliability
Types of Study Design
Observational Study
An observational study design is a type of research in which the investigator
observes and analyzes outcomes without manipulating any variables or applying
interventions. It is commonly used in health, epidemiology, and social science
research when experimental methods are not feasible or ethical.

Types of Observational Study Designs:

1. Descriptive Studies 2. Analytical Studies
These describe the distribution of diseases These aim to find associations between
or health-related conditions. exposures and outcomes.

• Case Report • Case-Control Study

• Case Series • Cohort Study
• Cross-Sectional Study • Ecological Study
Prospective Study vs. Retrospective Study
 Prospective Study vs. Retrospective Study
Both prospective and retrospective studies are observational designs used to
investigate associations between exposures (risk factors) and outcomes (diseases or
events). The key difference lies in the timing of data collection relative to the outcome.

Prospective Study: A prospective Retrospective Study: A retrospective

study follows participants forward in study looks backward in time, using
time from the point of exposure to the existing data to examine exposures and
outcome. Data are collected in real- outcomes that have already occurred.
time as events unfold.
Example: Researchers review hospital
Example: Researchers enroll healthy records of lung cancer patients and
individuals and track their smoking compare their past smoking history to
habits over 10 years to see who non-cancer patients.
develops lung cancer.
Prospective Study vs Retrospective Study
Feature Prospective Study Retrospective Study

Time Direction Forward-looking Backward-looking

Data Collection Ongoing/future From past records

Cost Higher Lower

Duration Longer Shorter

Bias Risk Lower Higher

Example Cohort study on smokers Review of medical records

Strength Can determine temporal sequence Good for rare diseases/outcomes

Cross-Sectional Study:
A cross-sectional study is an
observational study design that collects
data from a population at a single point
in time to examine the prevalence of an
outcome, condition, or characteristic. It

Descriptive Studies
can also define as “A cross-sectional
study measures both exposure and
outcome simultaneously, providing a
“snapshot” of a population”.

Example: A survey conducted in Dhaka

in 2025 to determine the prevalence of
hypertension among adults and its A snapshot is taken of a particular group of
association with obesity. people at a given point in time
Cross-Sectional Study:
Key Features:
• Conducted at one time point.
• Measures prevalence, not incidence.
• Can study multiple variables (e.g., smoking status, BMI, disease).
• Does not establish causality (cannot determine which came first exposure or outcome).

Descriptive Studies
Uses of Cross-Sectional Studies: Limitations of Cross-Sectional Studies:

1. Estimate prevalence of diseases or health 1. Cannot determine causality (temporal

behaviors. ambiguity).
2. Assess the burden of health problems in a 2. May miss seasonal variations or trends
community. over time.
3. Generate hypotheses for further research. 3. Survivor bias: Only includes those alive
4. Plan health services and interventions. at the time of study.
5. Identify associations between risk factors 4. Not suitable for rare or short-lived
and health outcomes. conditions.
Case Report
A case report is the detailed presentation of a single patient's medical history,
clinical symptoms, diagnosis, treatment, and follow-up. It typically highlights
something unusual, rare, or novel in clinical practice.
Uses Limitations
•

Descriptive Studies
Based on one/few cases; may not
• Identifies new diseases and adverse effects
represent general trends
• Helps in diagnosis and treatment of unusual
• Cannot prove causality
cases

• Enhances medical education and discussion • No control group or statistical analysis

• Generates hypotheses for larger studies • Subject to bias and ethical concerns

Example: A case report on a 35-year-old female patient who developed Stevens-Johnson Syndrome after taking an
over-the-counter antibiotic, highlighting the rare reaction and the importance of drug vigilance.
Case Series
A case series is a descriptive observational study that tracks and reports
characteristics or outcomes of a group of patients who have a similar diagnosis,
treatment, or exposure. Unlike a case report (which focuses on a single patient), a
case series involves two or more patients.
Uses of Case Series:

Descriptive Studies
Limitations of Case Series:
• Describe new or rare conditions • No control group → Cannot assess
• Document side effects or complications cause-effect relationships
of new drugs or therapies • Low level of evidence
• Generate hypotheses for future research • Small sample size
• Provide insight into clinical management • Subject to selection bias
• Support medical education and training • May overestimate the importance or
frequency of findings
Example: A case series of 10 patients who developed liver toxicity after taking a specific herbal
supplement, detailing their symptoms, treatment, and recovery.
Case-Control Study
A case-control study is a type of
observational analytical study that
compares individuals who have a
specific condition or disease (cases)
with individuals who do not have the
condition (controls), to identify
possible past exposures or risk
factors.

Example: Researchers select a group

of patients with lung cancer (cases)
and a group without lung cancer
(controls) and then look back at their
history of smoking to see if there is an
association
Uses of case control study
1. Investigate Causes (Etiology): Explore possible risk factors or causative agents of a disease.
2. Study Rare Diseases: Efficient for diseases that occur infrequently, where large population studies
would be inefficient.
3. Examine Multiple Exposures: Helps identify various potential causes for a single outcome.
Example: Dietary habits, lifestyle, and environmental exposures in cancer patients.
4. Generate Hypotheses: Provides initial evidence to support or refute a possible cause-effect
relationship, which can later be tested in cohort or experimental studies.
5. Support Public Health Actions: Helps inform preventive strategies or health policies by
identifying modifiable risk factors.

Limitations of Case-Control Studies:

•Cannot measure incidence or prevalence.
•Prone to bias: 1. Recall bias: Patients may not accurately remember past exposures.
2. Selection bias: Choosing controls may not be truly representative.
•Cannot prove causality – only associations.
 Measurement of Case-Control Study
In a case-control study, the main statistical measure used is the Odds Ratio (OR), which estimates
the strength of association between an exposure and an outcome (disease).

Odds Ratio (OR) Not

Exposed Total
The odds ratio compares the odds of exposure among Exposed
the cases (those with the disease) to the odds of Cases
a b a+b
exposure among the controls (those without the disease). (Disease)
Controls
c d c+d
(No Disease)

Interpretation:
•OR = 1: No association between exposure and disease.
•OR > 1: Positive association (exposure may increase disease risk).
•OR < 1: Negative association (exposure may be protective).
Measurement of Case-Control Study
Example: Imagine a case-control study investigating the link between smoking and
lung cancer.

Smoker Non-Smoker
Lung Cancer (Cases) 80 20
No Lung Cancer (Controls) 40 60

Interpretation: Smokers are 6 times more likely to develop lung cancer than non-
smokers in this study.
Cohort Study
A cohort study is an observational analytical study in which a group of people (cohort) is followed
over time to assess how specific exposures affect the incidence of an outcome or disease.

Key Features:
•Can be prospective (followed into
the future) or retrospective (use
past records to follow outcomes).
•Participants are free of the
outcome at the start.
•Measures incidence (new cases
over time).
•Can study multiple outcomes from
a single exposure.

Example: Researchers follow a cohort of smokers and non-smokers over 20 years to see who develops
lung cancer.
Types of Cohort Studies
[Link] Cohort Study:
1. Participants are enrolled before developing the outcome.
2. Exposure is assessed, and they are followed into the future.
3. Example: Following healthy adults for 10 years to observe the effect of a high-fat diet on
heart disease.

[Link] Cohort Study:

1. Both exposure and outcome have already occurred.
2. Past records (e.g., medical, employment) are used.
3. Example: Using employee records from 2000 to 2010 to study chemical exposure and
cancer risk.
Uses of Cohort Studies
• Determine risk and incidence of disease.
• Study the natural history of disease.
• Explore causal relationships between exposure and disease.
• Assess the long-term safety or effectiveness of treatments or interventions.
• Evaluate multiple outcomes from a single exposure.

Limitations of Cohort Studies

• Time-consuming and expensive (especially prospective).
• Loss to follow-up can affect validity.
• Not suitable for rare diseases (better for common outcomes).
• May have confounding variables.
Measurement of Cohort Study
In a cohort study, the primary goal is to compare the risk of developing an outcome (like
disease) between exposed and unexposed groups over time. The main statistical measure used is
the Relative Risk (RR), also known as the Risk Ratio.
Not
Exposed Total
Relative Risk compares the probability (risk) of the Exposed
outcome occurring in the exposed group to the Cases
unexposed group. a b a+b
(Disease)
Controls
c d c+d
(No Disease)

Interpretation of RR:
Relative Risk (RR) Interpretation
RR = 1 No difference in risk between exposed and unexposed groups
RR > 1 Exposure is associated with increased risk of the outcome
RR < 1 Exposure is associated with decreased risk (may be protective)
Measurement of Cohort Study
Example: A cohort study follows smokers and non-smokers for 10 years:

Lung Cancer No Lung Cancer Total

Smokers 40 160 200
Non-Smokers 10 190 200

Interpretation: Smokers have 4 times higher risk of developing lung cancer compared
to non-smokers.
 Case-Control vs Cohort Study
Feature Case-Control Study Cohort Study

Retrospective (backward- Prospective or Retrospective

Study Direction
looking) (forward-looking)

Starts With Outcome (disease) Exposure (risk factor)

Cases (with disease) vs.

Groups Compared Exposed vs. Unexposed individuals
Controls (without disease)

Relative Risk (RR) and Attributable

Main Measure Odds Ratio (OR)
Risk
Time & Cost Usually quicker and cheaper Usually time-consuming and costly

Suitability Ideal for rare diseases Ideal for rare exposures

 Case-Control vs Cohort Study
Feature Case-Control Study Cohort Study

Can Study Multiple Exposures for one outcome Outcomes for one exposure

Incidence Measured ❌ Cannot measure incidence ✅ Can measure incidence

Risk Factor Stronger evidence for causality

Weaker evidence for causality
Identification (temporal relationship)
High (especially recall and
Bias Risk Lower (if well-designed)
selection bias)
✅ Requires follow-up
Follow-Up Required ❌ No follow-up needed
(prospective)
Smoking history in lung cancer Following smokers and non-
Example
patients smokers for lung cancer risk
 Experimental Studies
Experimental studies, also called interventional studies, are research designs in
which the investigator actively manipulates one or more variables (interventions) to
observe the effect on outcomes. These studies are commonly used in clinical trials
and laboratory research.

🔍 Key Characteristics:
•Researcher assigns participants to intervention or control groups.
•Involves manipulation, control, and randomization.
•Causal relationships can be assessed.
 Types of Experimental Studies
1. Randomized Controlled Trial (RCT)
• Gold standard in clinical research.
• Participants are randomly assigned to:
• Intervention group (receives the treatment)
• Control group (receives placebo or standard treatment)
• Minimizes bias and confounding.

2. Non-Randomized Controlled Trial (Quasi-Experimental Study)

• Participants are not randomly assigned.
• More prone to selection bias.
• Used when randomization is not feasible or ethical.

3. Pretest-Posttest Design
• Measures outcome before and after an intervention in the same group.
• No control group necessarily used.
 Types of Experimental Studies
4. Cross-Over Study
• Each participant receives both treatments (e.g., Drug A and Drug B) in
sequence, separated by a "washout" period.
• Each subject acts as their own control.

5. Factorial Design
• Tests multiple interventions simultaneously using combinations.
• Efficient for evaluating interactions between treatments.

6. Cluster Randomized Trial

• Groups (e.g., schools, hospitals) are randomized instead of individuals.
• Reduces contamination between subjects.
 Randomized Controlled Trial (RCT)
• An RCT is a prospective, comparative, interventional study in which participants
are randomly allocated to two or more groups to evaluate the effect of an
intervention.
• A Randomized Controlled Trial (RCT) is a type of experimental study where
participants are randomly assigned to either the intervention group (receives
the treatment) or the control group (receives a placebo or standard treatment). It
is considered the gold standard for testing the efficacy and safety of new
medical interventions.
For Example, A study to evaluate the effect of a new antihypertensive drug. 200
patients are randomly assigned to:
Group A: New drug
Group B: Placebo
Researchers measure blood pressure changes after 3 months.
 Randomized Controlled Trial (RCT)
Key Components of RCT:

Each participant has an equal chance of being assigned to any

Randomization
group
Control Group Receives placebo or standard treatment

Blinding Reduces bias—can be single-blind, double-blind, or triple-blind

Follow-Up Participants are monitored over a period to assess outcomes

Predefined primary and secondary outcomes (e.g., blood pressure,

Outcome Measurement
mortality)
 Randomized Controlled Trial (RCT)
Advantages of RCT: Limitations of RCT:
• Minimizes bias through randomization • Costly and time-consuming
and blinding • May raise ethical issues (e.g., withholding
• Can establish causal relationships treatment)
• High internal validity • Not always generalizable to the real-world
• Provides strong evidence for clinical population
guidelines • Loss to follow-up can affect the outcome

Measures Used:
• Relative Risk (RR)
• Absolute Risk Reduction (ARR)
• Number Needed to Treat (NNT)
• Hazard Ratio (HR) in time-to-event analysis
Types of Randomized Controlled Trials (RCTs)
1. Parallel Group RCT
• Each participant is randomly assigned to only one group (intervention or control).
• Groups are followed simultaneously but separately.
• Example: Group A receives Drug X; Group B receives placebo. Both are followed for 6 months.
• Most common and straightforward RCT design.

2. Cross-Over RCT
• Each participant receives both treatments (intervention and control), in a sequential order, with
a washout period in between.
• Each person acts as their own control.
• Example: Patient receives Drug A for 2 weeks → Washout → Then receives Drug B for 2 weeks.
• It reduces variability but not suitable for long-term or curative treatments.
Types of Randomized Controlled Trials (RCTs)
3. Cluster Randomized RCT
• Entire groups (clusters) like hospitals, schools, or communities are randomized, not individual
participants.
• Useful to prevent contamination between participants.
• Example: One clinic gives Drug A to all its patients; another clinic gives placebo.
• Ideal for public health interventions or vaccine trials.

4. Factorial RCT
• Tests two or more interventions simultaneously in a single study.
• Participants are assigned to multiple combinations.
• Example: Four groups:
Group 1: Drug A + Drug B
Group 2: Drug A + Placebo
Group 3: Placebo + Drug B
Group 4: Placebo + Placebo
• It is efficient for studying interactions but it requires a large sample size.
Types of Randomized Controlled Trials (RCTs)
5. Adaptive RCT
• The design adapts during the trial based on interim results.
• Can stop early for efficacy or adjust group sizes dynamically.
• Example: If Drug A is clearly superior halfway through, the trial may stop or shift focus.
• It is efficient and ethical but complex to design and analyze.

6. Pragmatic RCT
• Conducted in real-world clinical settings to assess effectiveness under usual conditions.
• Example: Testing a new diabetes drug in public hospitals with routine follow-up.
• It has high external validity but may have lower internal control.
 Why is an RCT considered the gold standard in clinical research?

A Randomized Controlled Trial (RCT) is considered the gold standard in clinical research
because it provides the most reliable evidence for assessing the effectiveness and safety of
interventions. The key strength of RCTs lies in randomization, which evenly distributes
confounding factors between the intervention and control groups, minimizing selection bias.

Blinding (especially double-blinding) reduces performance and measurement bias, while the
use of a control group ensures a valid comparison. RCTs also establish a clear temporal
relationship between the intervention and outcome, helping to demonstrate causality.

They employ precise statistical tools such as Relative Risk (RR) and Number Needed to
Treat (NNT), making their results highly credible for evidence-based practice and regulatory
approval.

In short, RCTs are valued for their ability to reduce bias, control confounders, and provide
high-quality causal evidence in clinical research.
 Non-Randomized Controlled Trial (Quasi-Experimental Study)
A quasi-experimental study evaluates the effect of an intervention by comparing outcomes
between groups, but without random allocation of participants. A Non-Randomized Controlled
Trial, also known as a Quasi-Experimental Study, is a type of interventional study in which the
researcher assigns an intervention to groups without using randomization. This means
participants are placed in either the intervention or control group using non-random methods
such as pre-existing groups, availability, or preferences.
Example: A study assessing the impact of a new health education program on smoking cessation,
where one community receives the program and another similar community does not, but
participants are not randomly assigned.

Advantages: Limitations:
• Ethically and practically feasible when • No randomization increases the risk of
randomization is difficult. bias.
• Useful in natural settings or large-scale • Greater chance of confounding variables
programs. affecting results.
• Can still suggest causal relationships • Weaker evidence for causality compared
when well-designed. to RCTs.
A picture can
always reinforces
the concept
Thank You