Study Designs in Clinical Research
Study Designs in Clinical Research
Study Designs in Clinical Research
IN CLINICAL
RESEARCH
Types of Research Studies
Architecture of Clinical Research
Learning Objectives
• Be familiar with the types of research
study designs
• Be aware of the advantages,
disadvantages, and uses of the various
research design types
• Recognize a study
Studies in Medical Literature
• Two main categories:
1. Observational:
• Studies in which subjects are observed
2. Experimental:
• Studies in which the effect of an intervention is
observed
Observational Studies
• Case Series
• Case-Control
• Cross Sectional
• Cohort
Observational Studies
• Case Series
– What are they?
• Author describes some interesting or intriguing
observations that occurred to a small number of patients
– Characteristics:
• The simplest design
• Descriptive
• Lead to hypotheses subsequently investigated by other
types (Case-Control, Cross-Sectional or Cohort study)
• Generally over short period of time
• Generally no controls
Case Series & Case Reports
Observational Studies
• Case-Control
– Retrospective
– Question answered: “What happened?”
– Matching needed for controls
– Might be difficult to differentiate from Case
Series (Both are after the fact)
• Ask if the goal was to describe a phenomenon, if
description is the goal Case Series
Case Control Studies
Case-control Studies: research in reverse
?
Choose groups with and without disease, look
back at what different exposures they may have
had
Observational Studies
• Cross Sectional
– AKA Surveys, epidemiologic or prevalence
studies
– What are they?
• Analyzes data collected on a group of subjects at
one time
– Question answered: “What is happening
now?”
– Short time
Cross Sectional
• Possible uses of Cross Sectional studies:
– Diagnosing or staging a disease
– Evaluating different methods of doing the same thing
• e.g. Examining the relationship between histology slides and
MRI findings of diseased carotid arteries
– Establishing norms
• e.g. Establishing normal lab values from normal subjects
– Surveys
• May use preexisting validated surveys
• To learn what people think
• Sometimes combined with interviews
Observational Studies
• Cohort
– Question answered: “what will happen?”
– Prospective/forward
– e.g. Framingham study of cardiovascular disease:
Started in 1948, 6000 citizens participated, followed
for 20 years (study in 1970 by Gordon and Kannel)
– Possible uses:
• Typical cohort study
• Outcome assessment (patient outcomes: economic,
functional, satisfaction, QOL, ..)
• Historical Cohort studies/AKA Retrospective cohort: Relies
on prospective records collected (If accurate) – still
forward in time in the past
Cohort Studies
Cohort studies: marching
towards outcomes
The defining characteristic of all cohort studies is that they track people forward in
time from exposure to outcome. Data collection may be prospective or
retrospective. Ex. Contraceptives and DVT.
Cohort study design
(Prospective)
Exposure Observer Disease
“Longitudinal Studies”
Notion of time
Observational Studies
Longitudinal Studies
RCT Non-randomized
Systematic Reviews &Meta-
analyses
RCT RCT
SYSTEMATIC
REVIEW
Meta-analysis
RCT RCT
“STRONGER” EVIDENCE
Study Pyramid
Best
Worst
Classification of types of clinical research
DAR...
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Cohort Studies
• Advantages:
– Design of choice for studying cause of a
disease, course, risk factors
• Disadvantages:
– Cannot be used to prove causation
– Long studies can be costly
– Vulnerable to patient attrition, migration
Case-Control Studies
• Advantages:
– Quickest
– Least expensive
– Good for rare diseases and diseases that take long
time
– Good for investigation of a preliminary hypothesis
– Time factor research
• Disadvantages:
– Large biases
– Difficult to find matching controls
Cross Sectional Studies
• Advantages:
– Best for:
• Determining status quo of a disease
• Prevalence of disease
• Evaluation of diagnostic procedures
– Relatively quick and inexpensive
• Disadvantages:
– Provide only a snapshot in time
Case Series
• Advantages:
– Easy to write
– May be extremely useful to investigators
looking for causes of the observation
• Disadvantages:
– Subject to many biases in patient
selection
– Should be viewed as hypothesis
generating, not conclusive