Study Design
[Link]. HADEEL FAROUK
Cheap, Quick, Easy,
Simple, Unbiased, and
easily published
Stronger
Weaker
Medical Center Library, Duke University
EBP: Library Tools for Clinical Content. Christina L Wissinger, MS, MLIS, Clinical Informationist
Descriptive
Descriptive studies
• Describes specific characteristics in a population of
interest
• The most common forms are case reports and case series
• In a case report, we discuss our experience with the
patient’s symptoms, signs, diagnosis, and treatment
• In a case series, several patients with similar experiences
are grouped.
Case series and Case reports
It consists of collections of reports on the
treatment of individual patients or a report
on a single patient.
Because they are reports of cases and use no
control groups to compare outcomes, they
have little statistical validity.
Case reports
• Describe the experience of a single patient.
• Case reports document unusual medical occurrences
and can represent the first clues in the identification of
new diseases
Case series
• Collections of individual case reports.
• Investigation of the activities of the individual in case
reports can lead to the formulation of a hypothesis.
Analytical
Analytical Studies
• Analytical studies are of 2 types: observational and
experimental.
• Observational studies are studies that we conduct
without any intervention or experiment. In those studies,
we purely observe the outcomes.
• On the other hand, in experimental studies, we conduct
experiments and interventions.
Observational
Studies
Cross-sectional study
• This design is transverse where we take a specific sample at a
specific time without any follow-up
• It allows us to calculate the frequency of disease (prevalence) or the
frequency of a risk factor
• This design is easy to conduct
• For example:
• 1- if we want to know the prevalence of migraine in a population,
we can conduct a cross-sectional study whereby we take a sample
from the population and calculate the number of patients with
migraine headaches.
• 2- percentage of caries in children.
• 3- sugar intake and caries.
Cross-Sectional Survey
A study that examines the relationship between diseases (or other
health-related characteristics) and other variables of interest as they
exist in a defined population at one particular time (ie exposure and
outcomes are both measured at the same time). Best for quantifying
the prevalence of a disease or risk factor, and for quantifying the
accuracy of a diagnostic test.
Advantages:
- cheap and simple;
- ethically safe.
Disadvantages:
- establishes association at most, not causality;
- recall bias susceptibility;
- confounders may be unequally distributed;
- group sizes may be unequal.
Cohort study:
• We conduct this study by comparing two samples from the population:
one sample with a risk factor while the other lacks this risk factor
• It shows us the risk of developing the disease in individuals with the risk
factor compared to those without the risk factor (RR = relative risk)
• We may approach this study with 2 longitudinal designs:
▪ Prospective: we follow the individuals in the future to know who will
develop the disease
▪ Retrospective: we look to the past to know who developed the
disease (e.g. using medical records)
• This design is the strongest among the observational studies
• Data are obtained from groups who have been exposed, or not exposed, to
the new technology or factor of interest (eg from databases). No allocation
of exposure is made by the researcher. Best for studying the effect of
predictive risk factors on an outcome.
• For example:
to find out the relative risk of developing chronic obstructive pulmonary
disease (COPD) among smokers, we take a sample including smokers and
non-smokers. Then, we calculate the number of individuals with COPD
among both.
Cohort Study
Advantages:
- ethically safe;
- subjects can be matched;
- can establish timing and directionality of events;
- eligibility criteria and outcome assessments can be standardized;
- administratively easier and cheaper than RCT.
Disadvantages:
- controls may be difficult to identify;
- exposure may be linked to a hidden confounder;
- blinding is difficult;
- randomization not present;
- for rare diseases, large sample sizes or long follow-ups are necessary.
Cohort study (prospective) Cohort:
a group of people with a common
characteristic. Followed over time to
find how many reach a certain
outcome.
Study design:
➢Start by a disease-free population
➢identified by the exposure of
interest.
➢followed in time until the disease or
outcome of interest occurs
Retrospective cohort (or historical cohort)
• It Follows the same direction of inquiry as a cohort
study.
• Subjects begin with the presence or absence of an
exposure or risk factor and are followed until the
outcome of interest is observed.
• However, this study design uses information that
has been collected in the past and kept in files or
databases.
• Patients are identified for exposure or non-
exposures and the data is followed forward to an
effect or outcome of interest.
Case-Control Study:
• We conduct this study by comparing 2 groups: one group with the
disease (cases) and another group without the disease (controls)
• This design is always retrospective
• We aim to find out the odds of having a risk factor or an exposure
if an individual has a specific disease (Odds ratio)
• Relatively easy to conduct
• For example:
we want to study the odds of being a smoker among hypertensive
patients compared to normotensive ones. To do so, we choose a
group of patients diagnosed with hypertension and another group
that serves as the control (normal blood pressure). Then we study
their smoking history to find out if there is a correlation.
Case-control:
(Retrospective)
➢Cases: identified by outcome status at the start of the
investigation.
➢Controls: subjects without the outcome but from the same source
population (matching).
➢Data about exposure to a risk factor or several risk factors are
then collected retrospectively.
➢ Use interviews, records, or surveys.
Case-Control Studies
Advantages:
- quick and cheap;
- only feasible method for very rare disorders or those with long lag
between exposure and outcome;
- fewer subjects are needed than cross-sectional studies.
Disadvantages:
- reliance on recall or records to determine exposure status;
- confounders;
- selection of control groups is difficult;
- potential bias: recall selection.
Experimental
Studies
• Also known as interventional studies
• Can involve animals and humans
• Pre-clinical trials involve animals
• Clinical trials are experimental studies involving
humans
• In clinical trials, we study the effect of an intervention
compared to another intervention or placebo.
As an example, I have listed the four phases of a drug trial:
I: We aim to assess the safety of the drug (is it safe ?)
II: We aim to assess the efficacy of the drug (does it work ?)
III: We want to know if this drug is better than the old treatment (is it
better ?)
IV: We follow up to detect long-term side effects (can it stay in the
market ?)
• In randomized controlled trials, one group of participants receives
the control, while the other receives the tested drug/intervention.
Those studies are the best way to evaluate the efficacy of a
treatment.
Randomized Controlled Trial
An experimental comparison study in which participants are
allocated to treatment/intervention or control/placebo groups
using a random mechanism. Best for studying the effect of an
intervention
Advantages:
- unbiased distribution of confounders;
- blinding is more likely;
- randomization facilitates statistical analysis.
Disadvantages:
- expensive: time and money;
- volunteer bias;
- ethically problematic at times.
Crossover Design
A controlled trial where each study participant has both therapies, e.g., is
randomized to treatment A first, at the crossover point they then start
treatment B. Only relevant if the outcome is reversible with time, e.g.,
symptoms.
Advantages:
- all subjects serve as their own controls and error variance is reduced
thus reducing the sample size needed;
- All subjects receive treatment (at least some of the time);
- Statistical tests assuming randomization can be used;
- Blinding can be maintained.
Disadvantages:
-All subjects receive a placebo or alternative treatment at some point;
- washout period lengthy or unknown;
- cannot be used for treatments with permanent effects
