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Objectives
To understand the difference between
descriptive and analytic studies
To identify the hierarchy of study
designs, and the strengths and
weakness of each design
To be able to apply different study
designs to the same research question
Types of Studies
Descriptive Studies
Observational Analytic Studies
Cross Sectional studies
Case Control studies
Cohort studies
Experimental Studies
Randomized controlled trials
Descriptive
Case report
Case series
Survey
Analytic
Observational
Cross sectional
Case-control
Cohort studies
Experimental
Randomized
controlled trials
Descriptive studies
Getting a lay of the land
Surveys (NHIS, MCBS)
How many men in the U.S. filled Viagra
prescriptions in 2004?
Descriptive studies
Cannot establish causal relationships
Still play an important role in describing trends
and generating hypotheses about novel
associations
The start of HIV/AIDS research
Squamous cell carcinoma in sexual partner of Kaposi
sarcoma patient. Lancet. 1982 Jan 30;1(8266):286.
New outbreak of oral tumors, malignancies and infectious
diseases strikes young male homosexuals. CDA J. 1982
Mar;10(3):39-42.
AIDS in the "gay" areas of San Francisco. Lancet. 1983
Apr 23;1(8330):923-4.
Analytic Studies
Attempt to establish a causal link between
a predictor/risk factor and an outcome.
You are doing an analytic study if you have
any of the following words in your research
question:
greater than, less than, causes, leads to,
compared with, more likely than, associated
with, related to, similar to, correlated with
Descriptive
Case report
Case series
Survey
Analytic
Observational
Cross sectional
Case-control
Cohort studies
Experimental
Randomized
controlled trials
Research Question
Is the regular consumption of Red Bull
associated with improved academic
performance among U.S. medical students?
Rationale
functional drink designed for periods of
mental and physical exertion.
performance, concentration, memory,
reaction time, vigilance, and emotional
balance
Background
Seidl R, Peyrl A, Nicham R, Hauser E. A taurine and caffeinecontaining drink stimulates cognitive performance and well-being.
Amino Acids. 2000;19(3-4):635-42.
Study Design #1
Cross-sectional study of UCSF medical students
taking USMLE Step 2
Questionnaire administered when registering
for USMLE 2
Primary predictor: self-report of >3 cans Red Bull
per week for the previous year
Covariates: Age, sex, undergraduate university, place
of birth
USMLE Score
time
Cross-sectional Study:
Descriptive value:
How many UCSF medical students drink Red Bull?
What is the age and sex distribution of UCSF medical
students who drink Red Bull?
Analytic value:
Is there an association between regular Red Bull
consumption and test scores among UCSF med students?
Univariate
Multivariate (controlling for confounders)
Fast/Inexpensive - no waiting!
No loss to follow up
Measures of association
Risk ratio
(relative risk)
Disease
Yes
No
Yes
No
Risk
Factor
A
A+B
C
C+D
USMLE Score
time
time
Study Design #2
A case-control study
Cases: 4th year med students accepted to
residency in highly selective specialty X.
Controls: 4th year med students who applied
but were not accepted.
Predictor: self-reported regular Red Bull
consumption
Additional covariates (age, sex, medical school,
undergraduate institution)
ACTUAL CASES
TARGET
CASES
4th year UCSF students
Medical students accepted to
who matched in highly
highly selective residencies
selective specialty X
ACTUAL
CONTROLS
TARGET
CONTROLS
th
4
year students
who to
failed
All unsuccessful
applicants
highly
to
match
in
highly
selective
selective residency programs
specialty X
time
Case-control study-minuses
Sensitivity, specificity
Positive predictive value, negative predictive value
Measures of association
Disease
Yes
Sensitivity = A/A+C
Yes
No
Test
Specificity = D/B+D
PPV = A/A+B
NPV = D/C+D
No
Study design #3
Prospective cohort study of UCSF medical
students Class of 2009
All entering medical students surveyed
regarding beverage consumption and
variety of other potential covariates
Survey updated annually to record
changes in Red Bull consumption
Outcomes: USMLE Step 1 score, USMLE
Step 2 score, match in first choice
residency
Cohort studies
Framingham
Nurses Health Study
Physicians Health Study
Olmsted County, Minnesota
The future
Everyone else
time
Double cohort
Used to compare two separate cohorts with
different levels of exposure to predictor variable
(e.g., occupational groups)
Descriptive
Case report
Case series
Survey
Analytic
Observational
Cross sectional
Case-control
Cohort studies
Experimental
Randomized
controlled trials
Predictor
Outcome
Examples:
sex (men are more likely to drink red bull and men are
more likely to match in neurosurgery)
Undergraduate institution (students from northwest school are
more likely to drink red bull and also more likely to score higher on
USMLE)
Study design # 4
Randomized controlled trial of daily Red Bull
consumption among entering UCSF medical
students Class 2009
Randomized to daily consumption of Red Bull
vs. daily consumption of placebo
Outcomes: USMLE Step 1 score, USMLE Step
2 score, match in first choice residency
Sample
Sample
Treatment
Dx No Dx
Randomization
Randomization
Control
Placebo
Dx No Dx
Steps in a randomized
controlled trial
1. Select participants
high-risk for outcome (high incidence)
Likely to benefit and not be harmed
Likely to adhere
Steps in a randomized
controlled trial
4. Blinding the intervention
As important as randomization
Eliminates
co intervention
biased outcome ascertainment
biased measurement of outcome
5. Follow subjects
Adherence to protocol
Lost to follow up
6. Measure outcome
What
What is
is Blinding?
Blinding?
Single
Single blind
blind -- participants
participants are
are not
not
aware
aware of
of treatment
treatment group
group
Double
Double blind
blind -- both
both participants
participants
and
and investigators
investigators unaware
unaware
Triple
Triple blind
blind -- various
various meanings
meanings
persons
persons who
who perform
perform tests
tests
outcome
outcome adjudicators
adjudicators
safety
safety monitoring
monitoring group
group
Why
Why blind?:
blind?: Biased
Biased Outcome
Outcome
Ascertainment
Ascertainment or
or adjudication
adjudication
If
If group
group assignment
assignment is
is known
known
participants
participants may
may report
report symptoms
symptoms or
or outcomes
outcomes
differently
differently
physicians
physicians or
or investigators
investigators may
may elicit
elicit symptoms
symptoms
or
or outcomes
outcomes differently
differently
Study
Study staff
staff or
or adjudicators
adjudicators may
may classify
classify similar
similar
events
events differently
differently in
in treatment
treatment groups
groups
Problematic
Problematic with
with soft
soft outcomes
outcomes
investigator
investigator judgement
judgement
participant
participant reported
reported symptoms,
symptoms, scales
scales
Analysis of randomized
controlled trial
Analyzed like cohort study with RR
Intention to treat analysis
Most conservative interpretation
Include all persons assigned to
intervention group (including those who
did not get treatment or dropped out)
Subgroup analysis
Groups identified pre-randomization
Descriptive
Case report
Case series
Survey
Analytic
Observational
Cross sectional
Case-control
Cohort studies
Experimental
Randomized
controlled trials