Study Designs in Medical Research

June 25, 2019

Classification sof Study Designs
• Observational
– Descriptive or Case Series
– Case Control Studies (retrospective)
– Cross sectional , surveys (prevalence)
– Cohort Studies (prospective)
– Historical Cohort Studies
• Experimental
– Controlled Trials
• Parallel or Concurrent controls
– Randomize
– Non randomized
» Self control
» Cross over

• Sequential Control
• External Controls
– Studies with No Controls
OBSERVATIONAL
 subjects are mere observed

 4 Main types

1. Case Series
o certain interesting characteristics of a group (in series)
of patients are described in a published result
o THE SIMPLEST DESIGN
o Frequently leads to the generation of hypotheses
2. Case control
- Begins with the presence or absence of an outcome
and then look backward in time to detect possible
cause or risk factors that they may have been
- Cases are from individuals selected on the basis of
some disease or outcome
- Retrospective Study “what Happened?”
- Attempts to explain
C. Cross Sectional
 surveys, epidemiologic studies and prevalence studies
 Descriptive of the time line and do not have the connotation
 Analyzes the data collected on a group of subjects at one
time rather than over a period of time
 Designed to determined “what is happening” right now!
Uses of Cross Sectional Studies
 Diagnosing or staging a Disease
 Evaluating Different Methods of Doing the Same
thing
 Establishing Norms
 Surveys
D. Cohort Studies

- A cohort is a group of people who have something in

common and who remain part of a group over an
extended time
- Subjects are selected by some defining characteristics
suspected of being a precursor to or risk factor for a
disease or health effect
- Ask question “WHAT WILL HAPPEN?”
- Sometimes called PROSPECTIVE STUDY
Outcome Assessment
 Patient outcomes to health care providers to
determine patients response to different
therapeutics and management given.
1. Functional status
2. Quality of Life
3. Patient Satisfaction
4. Cost-Effectiveness and Cost-benefit Analysis
Experimental Studies
 Controlled Trials
 Parallel or Concurrent controls
 Randomize
 Non randomized
 Self control

 Cross over

 Sequential Control
 External Controls

 Studies with No Controls

Experimental Studies
 Easier to identify
 To determine whether the intervention makes a
difference
 Clinical Trial – involves humans
A. Controlled trials
 studies in which experimental drug or procedure is
compared with another drug
 Greater validity
• Parallel or Concurrent controls
– Randomize
– Non randomized
» Self control
» Cross over
• Sequential Control
• External Controls
1. Randomized Control Trials
- Epitome of all research design
- Provides the strongest evidence fro concluding
causation
- Provides the best insurance that the result was due to
the intervention
2. Non-randomized
– Subjects are not always randomized to treatment
options
– Do not use randomized assignment
A. Trials with self controls
- Same groups of subjects
- Vulnerable to Hawthorne effcet
B. Trials with external controls
- Uses the result of another investigator research as a
comparison
B. Uncontrolled Studies
 By strict definition they are not really experiments
or trials
 Likely to be used when the comparison involves a
procedure than when it involves drug.
Meta-analysis
• A type of study that does not fit specifically in either
category of observation studies or experiments
• Uses published information from other studies and
combines the results so as to permit an overall
conclusion.
• Similar to review articles
• Includes a quantitative assessment and summary of
findings.
• Appropriate when the studies when the studies that
have been reported have small numbers of subjects or
come to different solutions.
Advantages and Disadvantages
of Study Design
Clinical trials
 Gold standard, strongest design
 Design against which other are judged because it
provides justification for concluding causality and is
subject to the least number of problems and biases
 Best to use when the objective is to establish the
efficacy of the treatment or a procedure
 Disadvantages : costly and long duration
Cohort Studies
 Design of choice for studying the causes of a
condition, the course of a disease or the risk factors
because they are longitudinal and follow a group
of subjects over a period of time
 But generally cannot prove CAUSATION
 Extended time period of study makes it costly
 Vulnerable to problems associated to patients
follow-up
Case Control Studies
 Appropriate for studying rare disease or events
and for conditions that develop over a longer
period of time and for investigating a preliminary
hypothesis.
 Quickest and the least expensive study
 Good choice for someone who needs to complete a
medical research project in a specific amount of
time.
 Largest number of biases and errors and
dependent on high quality existing records.
Cross sectional studies
 Best for determining the status quo of a disease or
condition such a sthe prevalence of HIV in given
population
 Quick to complete and relatively inexpensive
 They provide only a “Snapshot in time” of the
disease process which may result in misleading
information if the research question is really one of
the disease process.
Case Series Studies
 Easy to write
 Observations may be extremely useful to
investigators designing a study to evaluate causes
of explanations of observations
 Susceptible to many biases related to subject
selection and characteristics observed
Thank you