Population (P)

Outcomes (O)
Interventions (I)
or Exposures (E)

Centre for Evidence Based Medicine,

Oxford, UK www.cebm.net
• Observational
• Cross sectional, case series, case-control studies, cohort studies
• Identify participants
• Observe and record characteristics
• Look for associations

• Experimental
• Randomized clinical trials
• Identify participants
• Place in common context
• Intervene
• Record data/evaluate effects of intervention
• Compare patients who have a
disease or outcome of interest
(cases) with participants who
do not have the disease or
outcome (controls).
• Look back (retrospectively) to
compare how frequently the
exposure to a risk factor is
present in each group to
determine the relationship
between the risk factor and
the disease.
• Examples …
Advantages:
• Quick and cheap as it requires fewer participants than cross-
sectional studies
• The only feasible method for very rare disorders or those with long
lag between exposure and outcome
Disadvantages:
• Reliance on recall or records to determine exposure status
• Confounders
• Selection of control groups is difficult
• Potential bias: recall, selection
• An observational study that looks at data at a certain point of time
• The investigator measures the outcome and the exposure in the study participants at the
same time
• Examples …
Advantages:
• Cheap and simple
• Ethically acceptable
Disadvantages:
• Establishes association at most, but not causality
• Researcher’s bias
• Group sizes may be unequal
• Confounders may be unequally distributed
• Are longitudinal studies that follow participants over a period of time
• Can be retrospective or prospective. Retrospective cohort studies are
NOT the same as case- control studies.
Retrospective cohort Prospective cohort

• The exposure and • The exposures are

outcomes have defined before looking
already happened. at the existing outcome
data.
• They are usually • Identify whether
exposure to a risk
conducted on data factor is associated
that already exists. with a statistically
significant difference in
the outcome
development rate.
• Prospective cohort studies are more common than
retrospective.
• Participants are recruited into cohort studies
regardless of their exposure or outcome status
(important strength).
• Participants are often recruited because of
their geographical area or occupation for
example, and researchers can then measure
and analyze a range of exposures and
outcomes
• Examples …
Advantages:
• Ethically safe
• Participants can be matched
• Can establish timing and directionality of events
• Eligibility criteria and outcome assessments can be standardised

Disadvantages:
• Controls may be difficult to identify
• Exposure may be linked to a hidden confounder
• Blinding is difficult
• For rare disease, large sample sizes or long follow-up necessary
• Prospective intervention of treatment studies
• An experimental comparison study where
participants are allocated to treatment and/or
intervention or control/placebo groups using
a random mechanism.
• Best for studying the effect of an intervention
• Examples …
• P Who are the patients, and what is the
problem?
• I What is the intervention or exposure?
• C What is the comparison group?
• O What is the outcome or endpoint?
Advantages:
• Unbiased distribution of confounders
• Blinding more likely
• Randomization facilitates statistical analysis

Disadvantages:
• Expensive: time and money
• Volunteer bias
• Ethically problematic at times
Are controlled clinical trials in which all participants
receive the same intervention (two or more
therapies), but of different randomized order.

Examples …
Advantages:
• All participants serve as their own controls and error variance is
reduced, thus reducing sample size needed
• All participants receive treatment (at least some of the time)
• Statistical tests assuming randomization can be used
• Blinding can be maintained .
Disadvantages:
• All participants receive placebo or alternative treatment at some
point .
• Cannot be used for treatments with permanent effects
• CONSORT for randomized controlled trials
• STARD for diagnostic accuracy studies
• STROBE for observational studies
• PRISMA for systematic reviews of trials
• MOOSE for meta-analyses of observational studies

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