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From Clinical Observations To Research: Dr. Dick Menzies June 10, 2005

This document outlines the hierarchy of study designs used in medical research, ranging from case reports and case series to randomized controlled trials. It describes the key aspects of observational studies like cross-sectional, case-control and cohort studies, as well as experimental studies including randomized trials and community trials. For each study design, the document discusses their advantages and disadvantages for investigating different research questions.

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Santi Padmasari
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28 views

From Clinical Observations To Research: Dr. Dick Menzies June 10, 2005

This document outlines the hierarchy of study designs used in medical research, ranging from case reports and case series to randomized controlled trials. It describes the key aspects of observational studies like cross-sectional, case-control and cohort studies, as well as experimental studies including randomized trials and community trials. For each study design, the document discusses their advantages and disadvantages for investigating different research questions.

Uploaded by

Santi Padmasari
Copyright
© © All Rights Reserved
Available Formats
Download as PPT, PDF, TXT or read online on Scribd
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From Clinical Observations

to Research
Hierarchy of Study Designs

Dr. Dick Menzies


June 10th, 2005
Overview of study designs
Observational Studies
Descriptive studies
Case reports
Case series
Reported data
Cross sectional studies
Ecologic studies
Analytic studies
Case control studies
Cohort studies
Diagnostic test evaluations
Experimental studies
Randomized control trials individual level
Field trials - community or group level
Step 1: Case Reports
Report of a single occurrence of new disease or
unusual occurrence of a known disease
Eg., Pneumocystis pneumonia in young homosexual men.
Pulmonary embolus in young woman on oral contraceptives
Myocardial infarction in a child
Strengths rapid and cheap
Useful to alert community to a new disease
This is useful if others are seeing the same thing.
Weaknesses rare events do happen!
Someone always wins the lottery
Clinicians have to recognize and diagnose the disease
Have to recognize that it is unusual.
Step 2: Small case series

This means 3 or more of the same condition


Unusual events - as for case reports
Generally adds a little more than case reports
More cases equals potentially more weight
But, rare events can happen - in clusters
3 cases of Angiosarcoma in workers from a PVC
factory
Three people on same street win lottery
Both of these occurred. Which do you think was
due to chance alone?
Step 3: After the case report/case series.
Need to review and understand disease
1. 1 Case definition
Who gets it, clinical features, outcomes?
The more detailed the better (autopsy)
2. 2 What is or appears to be the biology?
Apparent latency
Manifestations - what organs affected
Pathogenesis - probable or known
3. 3 Review the literature
This is often forgotten, or under-utilized
But is essential to avoid mistakes and
wasting time
3: Understanding the disease - latency
and duration

Latency refers to interval between exposure


and disease.
Short latency many infectious disease
Long latency many chronic diseases or
occupational diseases
Eg., cigarette smoking and lung cancer
Asbestos and mesothelioma
3: The relationship between latency and duration

Latency between exposure and disease onset


Short Long
Influenza Stroke
Short Food Poisoning Myocardial infarction
(acute) Trauma Herpes Zoster
Duration of
Illness
Chlorine gas Tertiary syphilis
Long
In-vitro teratogen COPD
(Chronic)
Rheumatic heart disease
Step 4a: Large Case Series
Description of a large number of patients with a new
disease, or receiving a new treatment or new operation
Can help to refine case definition.
No controls or comparison population
Implicit comparison with standard, or previous therapy
Historical controls or concurrent non-randomized controls
Advantages quick, easy and cheap
Comprehensive - Includes all cases
Disadvantages if results appear better can not be sure
it is due to:
Better results of new treatment , or, Better selection of patients
Eg: surgery for MDR-TB yields better survival than in patients
who did not have surgery
Step 4b: Reported Data - a form of
large case series
Reported data commonly used
(TB, HIV, Cancers)
Useful to define incidence/prevalence in a
population, and trends over time.
Some risk factors can be identified, if characteristics
of general population known
Description of clinical characteristics and outcomes
can be useful.
Most useful if reporting is very complete.
Step 4c: Ecologic Studies
General Design
Pick a condition or disease that is reported,or you
have information at a group level.
Eg., cancer rates by state or city
or, Complication or mortality rates by hospital
Get info re determinants or exposures at the
same group or community level
Eg Census data, Air pollution data, Climate,
Analyze association between disease or
condition and exposures - both at group level
4c: Ecologic Studies
Advantages
Usually very easy and quick studies
Take advantage of already reported data
And already gathered information about the
populations (eg., from census)
Disadvantages
Relationship may be due to completely unmeasured
factors
Substantial potential for confounding
Step 5 - Directly gathering your own
data: Prevalence or Analytic studies?

Prevalence surveys are the simplest to design


but can be harder to conduct
Analytic - Case-control are much harder to
design correctly, but can be easier to carry out
Analytic - Cohort studies - not-so-hard to design,
but very hard to carry out

So, lets start with a Prevalence study


Step 5 - Prevalence or cross-sectional
studies
Objectives:
Define risk factors (exposures) for disease
Also define occurrence/ importance in a population
General approach:
Pick a disease or condition (can pick several)
Identify the possible determinants or risk factors (can
pick several)
Pick a population, get them to agree (one time
survey)
Survey the population
Determine who has/has not the disease or condition
Identify who has/has not the possible determinants
Assess the relationship between disease presence and the
determinants
5: Cross-sectional or Prevalence Studies
(continued)

Advantages
Good for chronic diseases (prevalence)
Good for common diseases
Also good for fairly common exposures
Allows one to measure multiple disease or conditions
and multiple determinants or risk factors
Disadvantages
Measurement of exposure can be difficult
Recall problems if long latency
Accuracy if changes over time (Alcohol, smoking, blood
pressure)
Can not distinguish cause and effect (Tobacco Industry)
Step 6: Analytic Studies Case Control

General design
Identify a group of patients with disease, or
conditions = cases
Identify similar group but without disease or
conditions = controls
Measure risk factors or determinants in both
Assess if exposure more likely (odds > 1) in
cases than controls
6: Case Control Studies

Advantages
Relatively cheap and quick
Particularly useful for studying rare conditions
Or conditions with long latency
Disadvantages
Controls, Controls, Controls
Very difficult to select proper controls
This is the source of most problems in case control studies
And is why they are generally considered weak evidence.
Difficulties of retrospective exposure assessment
particularly if long latency
Step 7: Analytic Studies Cohorts

General design
Find a group of healthy people (without
condition/ disease)
Eg.: military, workforce, nurses
Measure their characteristics at baseline
Particularly exposures of interest
Follow them for a period of time
Measure occurrence of disease or condition
7: Cohort Studies

Advantages
Can measure many exposures or determinants
Can measure many diseases
Much better to know cause and effect
Disadvantages
Long and expensive (often very $$$)
Good for common diseases (some cancers,
cardiovascular)
Inefficient for rare diseases or with long latency
Also what if you fail to measure key determinants
(Solution = freezer)
Step 7a: Studies of Diagnostic Tests

General design
Usually prospective
Find group of patients with condition
Ideally when they are being investigated for it
Try new test & standard or reference tests
Establish a final accurate diagnosis in all
Need a GOLD standard.
Compare new test to old test(s)
agreement, sensitivity and specificity
7a: Diagnostic Test Studies

Advantages
Relatively cheap, and quick
If condition is reasonable common
Disadvantages
Must define final diagnosis correctly. Must have a gold
standard
Persons doing new test must be blinded
Population must be representative
eg. Patients with advanced disease vs. healthy volunteers
The final step: Experimental Studies
Randomized Trials
General Design
Pick an intervention usually a form of treatment
You can only pick one
Find a group of patients that agree to participate
Have to be representative of condition
Give the new treatment to some
Some get the old (or no) treatment
Do this randomly
Follow all to see outcomes
Experimental Studies Randomized Trials
Advantages
Best way to evaluate effect of an intervention
Best control of bias and confounding
Disadvantages
Not easy or feasible for all interventions
Not useful for studies or risk factors or natural history
Difficult to apply for most diagnostic tests
Substantial refusal or drop-out rates can restrict
generalizability
Population selected may not be representative
Young healthier adults
No pregnancy, no kids, no elderly PLEASE!
Experimental Community or Field Trials

General Design
Pick an intervention to be applied at a community
level
Fluoride in water, public education, vaccination
Find several communities or population groups
Apply intervention to some and not others
Randomly again
Measure outcomes at population or group level
Community or Field Trials
Advantages
Only way to study some interventions
May offer better assessment of likely impact of these
interventions
Disadvantages
All the same problems as ecologic studies
Also some important ethical issues (eg., fluoride)

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