Study Designs

Most problems in studies are due to poor design (not due to poor analysis)
 LEARNING OBJECTIVES
By the end of this lecture, you will be able to:

•Distinguish between experimental and observational studies.

•Identify the design of a particular study.

•Explain your choice of the appropriate study design to achieve the stated objectives
Types of study designs
•Studies can be classified as either:

• Observational and

• Experimental
 Types of Study Designs

Meta-
analyses
Randomised
Controlled, Field and
Community trials
gth
Cohort Study
en
Str

Case control

Cross-sectional

Case report and case series

Descriptive study designs
 Case Reports
Detailed presentation of a single case or handful of cases

Generally report a new or unique finding for example:

◦ previously undescribed disease

◦ unexpected link between diseases
◦ unexpected new therapeutic effect
◦ adverse events
Case Series
•The primary purpose of a case series should:

• illustrate a new finding,

• emphasize a clinical principle, or

• generate new hypotheses

•Usually cannot identify risk factors or the cause (no control or

comparison group)
 Cross-sectional study
A cross-sectional study examines the relationship between
disease (or other health related state) and other variables
of interest as they exist in a defined population at a single
point in time or over a short period of time (e.g. calendar
year).
 Types of cross-sectional study

Descriptive
This type is used to assess the burden of a particular disease in a defined
population.

Analytic
to investigate the association between a risk factor and a health outcome.

Risk factors and outcome are measured simultaneously, and therefore it may be
difficult to determine whether the exposure proceeded or followed the disease
(cause and effect).
Strengths

•Relatively quick and easy to conduct (no long periods of follow-up).

•Data on all variables is only collected once.

•Able to measure prevalence for all factors under investigation.

•Multiple outcomes and exposures can be studied.

•Good for descriptive analyses and for generating hypotheses.

 Weaknesses

•Difficult to determine whether the outcome followed exposure in time or exposure resulted
from the outcome.

•Not suitable for studying rare diseases or diseases with a short duration.

•As cross-sectional studies measure prevalent rather than incident cases, the data will
always reflect determinants of survival as well as aetiology1.

•Associations identified may be difficult to interpret.

•Susceptible to bias due to low response and misclassification due to recall bias.
Analytic study designs
 Case Control Study
•Case-control studies start with the identification of a group of cases
(individuals with a particular health outcome) in a given population and a
group of controls (individuals without the health outcome) to be included in
the study.

•It is essential that the case definition is clearly defined at the

outset of the investigation to ensure that all cases included in the
study are based on the same diagnostic criteria.
 Case control design
Direction of enquiry

Time
End of study Follow up Start of study
What is odds ratio?

• An odds ratio (OR) is a measure of association between an exposure

and an outcome.

• OR is sometimes called the Cross-product ratio.

• The OR represents the odds that an outcome will occur given a

particular exposure, compared to the odds of the outcome occurring
in the absence of that exposure.
Example 4:
A case-control study of 1700 participants looked at the association
between Tamoxifen and uterine cancer. The study included 689 cases.
There were 139 of the cases and 58 of the controls were taking Tamoxifen.

Fill in the 2x2 table below

Uterine Cancer
Tamoxifen Yes No

Yes A = Disease + Exposure B = No disease + Exposure

No C = Disease + No Exposure D = No Disease + No Exposure
Calculate the odds ratio of the above study
 Strengths
•Cost effective relative to other analytical studies such as cohort studies.

•Case-control studies are retrospective, and cases are identified at the beginning of the study;
therefore there is no long follow up period (as compared to cohort studies).

•Efficient for the study of diseases with long latency periods.

•Efficient for the study of rare diseases.

•Good for examining multiple exposures.

Weaknesses
•Particularly prone to bias; especially selection, recall and observer bias.

•Case-control studies are limited to examining one outcome.

•Unable to estimate incidence rates of disease (unless study is population based).

•Poor choice for the study of rare exposures.

•The temporal sequence between exposure and disease may be difficult to determine.
Cohort Study
•The cohort studies consist of two groups based on exposure status.
Exposed vs unexposed.
•The incidence of disease in exposed is compared to incidence in
unexposed group.
•All study participants must be free of the outcome under investigation
and

•have the potential to develop the outcome under investigation.

 Cohort design

Time
Start of study Follow up End of study
What is risk ratio?
• Risk ratio (also known as a relative risk) tells us how much more likely
the outcome is among those exposed compared to those unexposed
and is calculated as follows:
Practical 2:

• A study enrolled 100 smokers and 100 non smokers. They followed for
20 years to see if they develop lung cancer.

• The study found 30 smokers and 10 non smokers developed lung

cancer.

• Using the 2 x 2 table calculate the relative risk.

 Lung cancer

Smoking Yes No

Yes 30 70

No 10 90
Strengths
•Multiple outcomes can be measured for any one exposure.

•Can look at multiple outcomes.

•Exposure is measured before the onset of disease (in prospective cohort studies).

•Good for measuring rare exposures, for example among different occupations.

•Demonstrate direction of causality.

•Can measure incidence and prevalence.

 Weaknesses

•Costly and time consuming.

•Prone to bias due to loss to follow-up.

•Prone to confounding.

•Knowledge of exposure status may bias classification of the outcome.

•Poor choice for the study of a rare disease.

•Classification of individuals (exposure or outcome status) can be affected by changes

in diagnostic procedures.
 Quick Comparison of Cohort and Case-control Studies

Cohort Study Case-Control Study

Can calculate incidence rate and relative risk Only estimates Odds Ratio
Potentially greater strength for causal investigations Potentially weaker causal investigation
Expensive Inexpensive
Long-term study Short-term study
Large sample size required Can be powerful with small sample of cases
Efficient design for rare exposure
Efficient design for rare disease
Good for multiple outcomes
Good for multiple exposures
Less potential for recall bias
More potential for recall bias
More potential for loss-to-follow up
Less potential for loss-to-follow up
Possibly generalizable
Probably not generalizable
Allows examination of natural course of disease, survival
Does not allow examination of natural course of
disease, survival
Experimental
research
Purpose of the experimental research is to

• Test the effect(s) of at least one independent variable on one or more

dependent variables

• Major characteristic of experimental research that distinguishes it

from all other types of research is that researchers manipulate* the
independent variable.

*They decide the nature of the treatment (that is, what is going to happen to the subjects of the study), to whom it is to be applied, and to what extent.
Characteristics of Experimental Research

• Usually involves two groups of subjects:

• An experimental group and

• A control or a comparison group

RANDOMIZATION

Random selection: NOT really applicable for an Experimental Study Every

member of a population has an equal chance of being selected to be a
member of the sample.

Random assignment: Applicable for Randomized Control Trials RCTs

• Every individual who is participating in an experiment has an equal
chance of being assigned to any of the experimental or control
conditions being compared.
 General outline of a Randomized Controlled Trial
(RCT)
Intervention studies and Randomized Controlled Trials
(RCTs)

Interventional studies are often performed in laboratories and clinical

studies to establish beneficial effects of drugs or procedures.

They are considered to provide the most reliable evidence in

epidemiological research.

They are either preventative or therapeutic

Therapeutic trials
Therapeutic trials are conducted among individuals with a particular disease to
assess:

• The effectiveness of an agent or procedure

• To diminish symptoms, prevent recurrence, or

• Reduce mortality from the disease.

Preventative trials
Preventative trials are conducted:

•to evaluate whether an agent or procedure reduces the risk of

developing a particular disease among individuals free from that disease
at the beginning of the trial, for example, vaccine trials.

•Preventative trials may be conducted among individuals or among entire

communities
 Strengths of a randomized controlled trial
• A well designed randomised control trial provides the strongest evidence of any epidemiological
study design

• A RCT provides the best type of epidemiological study from which to draw conclusions on causality.

• Randomisation provides a powerful tool for controlling for confounding,

• Clear temporal sequence: exposure clearly precedes outcome.

• Provides a strong basis for statistical inference.

• Enables blinding and therefore minimizes bias.

• Can measure disease incidence and multiple outcomes.

Weaknesses of a RCTs

•Ethical constraints - for example, it is not always possible or ethical to manipulate

exposure at random.

•Expensive and time consuming.

•Requires complex design and analysis if unit of allocation is not the individual.

•Inefficient for rare diseases or diseases with a delayed outcome.

 What is meta-analysis?
Meta-analysis is a statistical technique for combining the findings from
independent studies.

Meta-analysis is most often used to assess the clinical effectiveness of

healthcare interventions; it does this by combining data from two or more
randomised control trials.

Meta-analysis of trials provides a precise estimate of treatment effect, giving

due weight to the size of the different studies included.
What is meta-analysis?
The validity of the meta-analysis depends on the quality of the systematic
review on which it is based.

Good meta-analyses aim for complete coverage of all relevant studies, look for
the presence of heterogeneity, and explore the robustness of the main findings
using sensitivity analysis.
 11.3 Study Design
•Justify why you have chosen this study design.

•Mention the advantage of this study design and how it is related to your
study topic

•Remember: Chart Review is NOT a study design

 You need to specify the type of study design when you are doing a
chart review:
- Case Series, Cross-sectional survey, Case Control, Cohort study
Proposal section 11.3
11.3 Study Design: (Instructions: Mention the type of study design e.g. cross-sectional,
case-control, intervention study, etc.)

It is an observational cross-sectional study in which we draw inferences from a sample to a

population. Data will be collected, analysed, and represented at a specific point in time. As the
study aims to assess the level of knowledge and awareness of selected physicians thus cross
sectional study design best suits the objectives. Also sectional study are less time consuming
and cost effective that’s why this stud design was chosen.
Example
11.3 Study Design: (Instructions: Mention the type of study design e.g. cross-sectional, case-
control, intervention study, etc.)

An analytical cross sectional survey will be conducted to assess the prevalence of social anxiety
and obsessive compulsive disorder among multiple sclerosis patients and investigate the
association between the EDSS disability scores and the presence of social anxiety and obsessive
compulsive disorders. This study design is most suitable for our objectives and the initial
assessment of the problem.
Thanks