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descriptive study design
Describes only – does not examine relationships
• Surveillance data (study)
• Case reports
• Case series
analytic study design
Examines relationships / Tests hypotheses
analytic study design - observational
investigator observes
• Ecologic
• Cross-sectional
• Case-control
• Cohort
analytic study design - experimental
Investigator assigns exposure
• RCT
cross-sectional studies
• Snap-shot of population(s) at a single point in time
• Exposure and outcome data collected at the same time
• Participants selected without regard to exposure or disease status
point prevelance
from cross-sectional studies
number of people with an event at a specific time point divided by the size of the population
(disease status (top) vs exposure status (side))
disease prevelance
number of cases (diseased) at specified time/total population
exposure prevelance
number of people exposed at specified time/total population
cross-sectional studies strengths
good for generating hypothesis
temporality is not a problem for time invariant exposures
relatively low cost
does not require follow up
cross-sectional studies limitations
temporality (reverse causation bias)
subject to non-exchangeability
subject to selection bias
does not measure risk
not useful for rare diseases
prevelant cases may no be same as incident
case-control study
backwards directionality (tracing back to exposure status based on outcome)
(outcome staus (top) exposure status (side))
What measures of disease frequency can we estimate from a case-
control study?
prevalence of exposure and odds ratio (measure of association)
cases
sample (or all) people who developed outcome
controls
sample of people who did not develop outcome
case-control studies - advantages
good for rare outcomes
good for oucomes with long latency periods
study many (detailed) exposures
efficient with time and money
require smaller sample size
case-control studies - disadvantages
usually only one outcome per study
temporal ambiguity (exposure is measured after disease has occured)
inefficient for rare exposures (very few cases or controls will have rare exposure)
genealizability concerns (over-represent people who live longer with disease)
suceptible to selection and information bias (retrospective info)
cohort study
gold standard of observational study designs
follow a population of exposed and unexposed individuals at risk of developing an outcome to compare the development of hte outcome between groups
(disease (top) vs exposure status (side))
cumulative incidence
number of poeple who became newly ill during the time/number of people at risk during time
incidence rate
number of people who became newly ill during time/person time at risk
cohor studies - strengths
temporality
can estimate incidence of disease
can consider time-varying information
can study adverse exposures or those that cannot be randomzied
good for rare exposures
can study multiple outcomes/exposures
serve as the base population for other study designs
cohort studies - limitations
require long-term follow up
high cost
not practical for rare diseases
loss to follow-up
differences in those who are exposed and not can bias results
RCT study
gold standard of study designs
randomly assign people to either receieve or not receive the exposures
control group represents what would have happened without exposure/intervention/treatment
(disease status vs exposure)
intent to treat
Analysis in which data are evaluated based on the group that participants were randomly assigned, not in the group they eventually ended up in
*ITT answers the question, “What is the effect of being assigned to a treatment?
RCT - strengths
strongest evidence for causality
if blinded, less observer bias
calculate measures of disease incidence
randomization can minimize effects of confounding
unlikely misclassification of exposure
RCT - limitations
not “real life” so limitations of generalizability
high coset and very time consuming
could be inapporpriate or unethical
could be poor compliance and loss to follow-up
prevlalence ratio
prevalence of outcome in exposed/prevalence of outcome in nonexposed
What study designs can be used to calculate a prevalence ratio of the outcome?
• Cross-sectional study
• Cohort Study (after follow-up)
• RCT (after follow-up)
risk ratio
cumulative incidence of disease in exposed/cumulative incidence of disease in non-exposed
(same equation and values as prevalence ratio)
What study designs can be used to calculate a risk ratio?
• RCT
• Cohort study
rate ratio
incidence rate of disease in exposed/incidence rate of disease in non-exposed
disease and person time (top) vs exposure status (side)
What study designs can be used to calculate a risk ratio?
• RCT
• Cohort study
odds ratio
probability that an event will occur divided by the probability that an event will not occur
(#cases exposed/# cases not exposed)/(# controls exposed/#controls not exposed)
case and control top exposure statues sides
direction of measures of association
• Values = 1.0 means that there is no association
• Values > 1.0 indicate a positive association
• Values < 1.0 indicate a negative/inverse association
magnitude of measures of association
The value tells you how many times the (prevalence, risk) of the outcome the exposed group has vs. the unexposed group
confounder
associated with exposure (in base population)
associated with outcome (among unexposed)
not in causal pathway
statistical adjustment
include confounders as covariates in a regression model
stratification
analyze the exposure-outcome association within levels (strata) of a confounder
mediation
• Mediation explains how or why an exposure affects an outcome
• A mediator is a variable on the causal pathway between the exposure and the outcome (indirect)
effect modification
• Effect modification is when the relationship between exposure and outcome is different depending on a third variable.
quantitative effect modification
: Association between exposure and outcome is in same direction across strata but the strength of the association varies.
qualititative effect modification
: Association between exposure and outcome is in opposite direction across strata or is present in some strata but absent (null) in others