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Research Design:

Choosing the Study Design


Kimberly R. Barber
Director of Research
McLaren Regional
Medical Center
Assign Exposure?

Yes No

Experimental Observational

Random allocation Comparison group

Yes No Yes No

RCT Non- Analytic Descrip


RCT study study

Direction?

Exposure Outcome Exposure & Outcome


Simulteneous

Cohort Case- Cross-


Control Section

Exposure Outcome
Design to Employ

 Dependent on the hypothesis posed.


– Is your intent to observe, associate factors, or
show cause and effect?
– Are exposure or outcome factors common or
rare?
– Are your resources many or constrained?
Types of Study Designs

 Observational Designs
– Descriptive
– Analytic
 Cross-sectional, Case-control, Cohort
 Experimental Designs
– Quasi experimental
 Non randomized or non control trial
– True experimental
 Randomized Controlled Trial
Descriptive Studies

 No assignment of exposure or risk factor.


 Intent is to observe and record.

 Record events or activities.


– Single event or case = Case Report.
– Several events or cases = Case Series.
Cross-Sectional Studies

 Measurement of risk and outcome at the


same time.
Risk factor

Outcome
Cross-Sectional Design
 The only study capable
of calculating
prevalence.
– Proportion of the
population with
the outcome at any
point in time.
Cross-Sectional Studies (cont)

 Most useful if exposure continues right up to


time that outcome is recognized.

 Often conducted as screening and


classification preamble to prospective studies.
Application Exercise:
Cross-Sectional Study

 Design a cross-sectional study that examines


the relationship between dietary sodium and
hypertension in middle-aged men.

 What research question can we answer?


Cross-Sectional Studies
Advantages

 Cheap and quick studies.


 Data is frequently available through current
records or statistics.
 Ideal for generating new hypothesis.
Cross-Sectional Studies
Disadvantages

The importance of the relationship between the


cause and the effect cannot be determined.
 Temporal weakness:
– Cannot determine if cause preceded the effect or
the effect was responsible for the cause.
– The rules of contributory cause cannot be fulfilled.
Case-Control Studies
(Retrospective)

 Case/Control Studies.
– Group subjects according to the presence or
absence of the outcome.
?
?
Risk Disease

– Review past histories of the subjects for the


occurrence of suspected risk factors.
Case / Control Studies (cont)

 Case / Control studies have two main


purposes:
 Descriptive
– Describe the risk factor profile for an outcome.
 Analytic
– Analyze associations between outcome and risk
factors.
Application Exercise:
Case / Control Study

 Describe a case/control study on the


relationship between childhood obesity,
smoking history, and occurrence of
hypertension in middle-aged men.

 What research question can we answer?


Case / Control Studies
Advantages

 Are good initial explanatory studies.


– Investigators can explore multiple risk factors
simultaneously for one outcome.
 Are efficient, relatively cheap, and quick.
– Data available through
chart review.
Case / Control Studies Advantages
(cont)

 Well suited for rare diseases.


– Since the study begins with subjects who already
have the outcome, it is easier to accumulate
enough subjects for significant results.
Case / Control Studies
Disadvantages

 Data Quality
– Data with inadequate detail, questionable
reliability, or use a different standard to judge
disease severity.
 Recall bias
– Subjects who have unpleasant experiences may
recall past differently than control subjects.
Retrospective Studies
Disadvantages (cont)

 Sampling bias
– Sample usually not representative of all subjects
who could be included.
 Other
– Capable of studying only one outcome at a time
– Cannot calculate prevalence or incidence.
– Subject to confounding factors.
– Cannot prove contributory cause.
Cohort Studies
(Prospective)

 Subjectsidentified according to the presence


or absence of the risk factor.

 Followedover time until the outcome occurs


or becomes evident.
Cohort Studies (cont)
 Subjects with and without the suspected risk
factor are followed for the development of the
outcome.
Disease
R
i ?
s ?
k
– The frequency of the outcome is compared
between the two groups.
Cohort Studies (cont)

 Cohort studies have two main purposes:


 Descriptive
– Describe the incidence of outcome over time.
 Analytic
– Analyze associations between risk factors and
outcome.
Historical Cohort Studies
(Non concurrent)

 The following all happen in the past:


– Assembly of the cohort according to the presence
of absence of risk factors.
– Baseline measurements
– Follow-up and outcomes
?
?
Exposure Disease
Historical Cohort (cont)

 Are not retrospective studies.


– Subjects are grouped according to the presence
or absence of risk factors (past or present).

– Subjects in retrospective studies are grouped


according to the presence or absence of the
outcome.
Cohort Studies
Advantages

 More powerful design for defining incidence.


 Powerful design for associating the cause
with the effect.
– Can suggest that the cause precedes the effect.
– Data can be collected in a comprehensible and
uniform fashion.
 Exception:
historical cohort with same measurement
weaknesses as the case/control.
Cohort Studies
Advantages (cont)

 No recall bias.
 Cohort designs can examine many outcomes
for potential risk factors under investigation.
Cohort Studies
Disadvantages

 Expensive in time, money, and number of


patients necessary to demonstrate significant
differences between groups.

 Less
likely than retrospective studies to
uncover new risk factors.

 Also subject to confounding.


Cohort Studies
Disadvantages (cont)

 Loss of valuable information due to patient


attrition.

 Patients may change their behaviors or risk


factors after the initial grouping of subjects
resulting in misclassification and study
error.
Clinical Trial

 Experimental study
– Unique features:
– Intervention in the subject’s care for the
purposes of the study.
– Randomization of subjects.
– Control group comparison.
 Placebo or treatment
Clinical Trial Design

Randomized Outcome

Intervention/
Effect
Clinical Trial (cont)

 Randomization
– Subjects are randomly assigned to control or experimental
group.

– Groups are similar in every way except for the intervention


under study.

– Each subject has equal probability of being placed in


either group.
Clinical Trials
Advantages

 Subject to the fewest methodological biases


of all study designs.

 Most powerful study designed to show


contributory cause.
Clinical Trials
Disadvantages

 Is the most expensive study design in terms


of money, time, and number of patients.
– Issues of patient attrition and compliance may
invalidate the results.
– Can be problematic for ethical reasons.
 Use of placebo
 Harm outweighing benefits
 Zero tolerance for some exposures
Choosing the Specific Design

 Studydesign is highly dependent on the type


of analysis. (3)

 Typeof analysis is dependent on the


hypothesis posed. (2)

 Thehypothesis is dependent on the intent of


your research. (1)
1. Research Study Intent
 Know the problem.

 Determine what you


want to conclude.

 Formulate the question.


Examples of Intent
Iintend to show that aspirin resistance is
associated with the severity of heart disease.
– I will compare levels of aspirin resistance among
patients with differing severity of heart disease.
I intend to show that breast feeding is
protective against allergies developing in
infants.
– I will compare rates of allergies among women
who breast feed and those who do not.
2. Research Hypothesis
 Know the question you
want answered.

 Restate the question into


terms of Ho and Ha.

 Think about
corresponding analysis.
Examples of Hypotheses

 Is aspirin resistance associated with heart


disease?
– Aspirin resistance increases the risk of heart
attack.
 Is breast feeding associated with allergies?
– Breast feeding decreases the risk of allergies in
babies.
3. Statistical Plan of Analysis
 Correlation?

 Comparison?

 Association?

 Difference?
Examples of Analysis

 Thelevel of aspirin resistance is compared


between those with heart attack and those
without.
– Differences in resistance scores between two
groups (Student T-test)
 Therate of infant allergies is compared
among women who breast feed and those
who do not.
– Relative risk association (Chi square analysis)
Choose Appropriate Design
 Cross-sectional.

 Case-control.

 Cohort.

 Clinical Trial.
Apply the Best Design
 Think about the
measures to be used.

 Know the analysis


required.

 Rethink desired
conclusions.
Summary

 Study Design key to determining cause or just


association.
 Each design has unique strengths &
weaknesses.
 The design drives the conclusions.