Choosing the Appropriate

Study Design
and
Protocol Development

Dr. Padma Bhatia

Associate Professor,
Community Medicine, GMC
Bhopal
Study design: Definition

A study design is a specific plan

or protocol for conducting the
study, which allows the investigator
to translate the conceptual
hypothesis into an operational one.
 USUALSTUDYDESIGNS
OBSERVATIONAL EXPERIMENTAL
STUDY STUDY

CLINICAL COMMUNITY
DESCRIPTIVE TRIAL
STUDY TRIAL

CROSS-SECTIONAL

CASE CONTROL

ANALYTIC STUDY
COHORT
Study Design Sequence
Hypothesis formation

Descriptive
Case reports Case series
epidemiology

Analytic Animal Lab

epidemiology study study

Clinical
Hypothesis testing
trials

Cohort Case- Cross-

control sectional
 Difference Between
Study Designs

Study Design Intervention Temporal Sequence Sampling

Risk Factor and

Cross Sectional Absent Outcome at same time. Yes

Outcome first, then risk

Case Control Absent factor (retrospectively). Matching

Risk Factor first, then

Cohort Absent Maybe
Outcome.
Clinical/ Intervene, then measure
Community Trial Present Outcome. Randomisation
Which design is appropriate for
your study?
 Usual study design
Commonly chosen : Specific for Patho/Diagnostic
• Clinical trial • Diagnostic testing
• Cross-sectional • Sensitivity,
• Case-control Specificity & ROC
• Cohort
 Which one?
• If you have an intervention, then it is a clinical
trial.
• If there is no intervention, then it is one of the
observational studies, usually cross-sectional.
• If the disease is rare, less than 5%prevalence
rate, then it is a case-controlstudy.
• If you are looking forward in time for new
incidence of the disease, then it is a cohort
study.
 Clinical Trials

If you have an intervention, then

probably it is a clinical trial.
 Clinical Trial

Intervention Outcome
Look forward
in time

Confounders
Starts with Intervention, then measure the futureOutcome
 EXPERIMENTALSTUDY
C+

T+
C-
Study Eligible
Population Participants
C+

T-
Selection
Randomisation C-

Future
 Clinical Trial
Intervention Outcome-Improved?

Improved (75%)

Drug Feelgood

Sample No improvement (25%)

Depressed ratio (1:1)
patients
Improved (70%)

Drug Sogood

No improvement (30%)
Time Future
Starts with Intervention, then measure the futureOutcome
CROSS-SECTIONALSTUDY

Also known as Prevalence Study

or Survey. Easiest study design.
 Cross-Sectional Study
• Measures the relationship of variables in a
defined population at one particular time
• Both risk factors (exposure) and disease
outcome are observed at the same (point in)
time in a sample (or the entire population) of
subjects.
• i.e. Studying the effect of overweight onthe
prevalence rate of diabetesmellitus.
 Exposure & Outcome

Risk Factor Outcome

Confounders
 Cross-Sectional Study

Risk Factor Outcome

Confounders
Both Risk Factor & Outcome measured at the sametime.
 Reminder
• If the prevalence/incidence rate is below 5%,
please do not choose a cross-sectional study
design. Your number of cases would be too low to
do any analysis.
• For example, doing a cross-sectional study of HIV
cases among pregnant mothers of Bhopal
district. The rate of HIV+ is less than 0.5%.
Therefore even with 10,000 samples, you will
only get less than 50 HIV+.
CASE-CONTROLSTUDY

For rare diseases. Consider if

prevalence rate below 5%.
 CASECONTROLSTUDY
- CONCEPT
• Comparison of group of diseased person
(cases) with another group ofnon-diseased
person (control) for past exposure to a
suspected cause of thedisease.
• Arises because of hypothesis that the
risk factor (exposure) causes thedisease
 Case-Control Study

Exposure Outcome
Look back in
time

Confounders
Starts with Outcome, then trace the retrospective exposure
 CASESELECTION
• Determine clear and reproducible definitions of
the health problems to be studied (avoid
misclassification bias)
• source of cases
All persons with the disease seen inparticular
facility(ies) in a specified period of time.
All persons with the disease found ingeneral
population.
• Incidence cases (newly diagnosed cases)
preferred
 CHOICEOFCONTROLS
• Controls should ideally be selected from thesame
population gave rise to cases
• Similar to cases in regard to past potentialexposure
• Free from study disease
• If controls are patient with other diseases then
select only diseases that are not known to have
relationship with factors under study.
 ADVANTAGES
• able to study rarediseases
• can explore multiple exposures
• relatively inexpensive
• can calculate Odds Ratio
• can support causation but not proveit
• easy to get cases
 COHORTSTUDY
- ignore, none of you willdo this.
 COHORTSTUDY
BASIC CONCEPT
• Group or groups of individuals are
studied over time as to onset of new
cases of disease and factorsassociated
with the onset of disease.
• Synonyms : incidence study,
longitudinal study, prospectivestudy.
 Cohort Study

Exposure Outcome
Look forward
in time

Confounders
Starts with Risk Factor, then measure the future Outcome
 COHORTSTUDY
FOLLOW-UP

DISEASE

EXPOSED GROUP
NONDISEASE
Free from
disease

DISEASE

UNEXPOSED GROUP
NONDISEASE
 Protocol
Development

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 Research Protocol

Protocol is a comprehensive yet concise

statement regarding the proposal.
Protocol is the BACKBONE that supports
research in all steps of it’s execution.
Sufficient thought must be given to it’s
preparation
Contents/Elements of a medical
research protocol

Title • Ethics
Investigator(s) • Statistical evaluation
Abstract • Limitations
Introduction • Logistics
Review of literature • References
Objectives and • Appendix
hypothesis
• Dissemination
Methodology

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 Protocol Format

• Why?
• What?
• How/ When/ Where?
• At What Cost?

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 Title

Clearly worded but concise title that

aptly describe the gist of study
 Investigator(s)

Name, qualification, affiliation,

supervisor/adviser, degree, year
etc

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 ABSTRACT

•An abstract is a brief summary.

•Provides the reader with the main points and
conclusion of your proposal.
•Descriptions of the method may include the design,
procedures, the sample and any instruments that will be
used.
•It should stand on its own, and not refer the reader to
points in the project description.
 Introduction
Background and Rationale
•Review of previous studies and critique
•Data gap / unclear areas
•Your hypothesis
•How it helps fill data gaps
•Why resources should be directed to your project
(Introduction in a protocol should be short, not
exceeding one page)
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 Review of literature

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 Review of literature

 Critical appraisal of the findings of others that could

have bearing on this research.
It sets the context for your research to help the reader
understand the questions and objectives.
 Must be focused on local environment so that the
relevance is clearly established
 Confine it to topic and include latest developments.
37
 Objectives

“Clear worded short statement of

what exactly is intended to be
achieved by this research”

•General objectives
•Specific objectives

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 General/broad objective is generally one
that specify the area of research.
Specific objectives delineate the specific
aspect of problem.

39
 General/broad objective -
“ To assess whether a new diagnostic modality
is better than existing one”

Specific objectives –
i. Positive and negative predictivity
ii. Safety in case it is invasive procedure or
side effect
iii. Feasibility – field , clinic and hospital
settings
iv. Acceptability by medical community and
patients
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v. Cost – effectiveness
 Methodology
•Study Design
• Sample size
•Subjects

Definition of subjects/controls
Exclusion / Inclusion criteria
Sampling methods
Compliance / dropouts (expected)

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 Methodology ….
•Blinding or other strategies to reduce bias
• Matching criteria
•Specification of intervention
•Definition of variables
•Confounders and their control
•Method of eliciting information
•Validity and reliability of devices

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 Collection of Data
• Source of data
• Data collections procedures
• Measurements
• Instruments

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 Analysis of Data

 “Clean” your data

 “ Use appropriate statistical tests”

for comparison

44
 Limitations

“Conditions or groups to which the

results would not apply”

45
 Logistics

Arrangements of resources, assignment

of duties, sharing of responsibilities and
training

46
 A time line / Gantt
Analysis and
chart
submitting
report
Data entry

Post intervention
assessment

Education
intervention
Initial
assessment
Pre-test
questionnaire
Questionnaire
preparation
Clearance
from principal
Months 1 2 3 4 5 6 7 8 9 10 11 12
 References / Bibliography ?

48
 References / Bibliography ?

All REFERENCES in the list must have been cited

in the text.
BIBLIOGRAPHY is a list of all related literature –
cited or not.

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 Appendix

Forms such as questionnaires, schedule, or

proforma to be used: structured or open, pre-
coded or not , pre tested or not, Consent form,
patient information sheet etc.

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 Conclusions
 Do not make conclusions beyond the study

– Make realistic statements

 Some studies generate hypothesis

 Translate the experience into policy

“ CAN WE MAKE A DIFFERENCE”

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 DISSEMINATION

How do you propose to share the findings of

your study with professional peers, practitioners,
participants and the funding agency?
 SUMMARY

 Protocol is Chief architect of the study

and provides support in case of any
confusion or doubt arises.
 It should be clearly worded and should be
prepared with utmost care.

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