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Study designs

Dr. Nadira Abbas AlBaghli


Experimental Studies
or Clinical Trials
Controlled trials
 are studies in which the
experimental drug or procedure is
compared with another drug or
procedure, sometimes a placebo
and sometimes the previously
accepted treatment
Randomized controlled
trial
 provides the strongest evidence
for concluding causation; it
provides the best insurance that
the result was due to the
intervention
 To reduce the chances that subjects or
investigators see what they expect to
see, researchers can design double-
blind trials in which neither subjects nor
investigators know whether the subject is
in the treatment or the control group.
When only the subject is unaware, the
study is called a blind trial.
Advantages of Clinical Trials

 the gold standard/ causality


 the least number of problems or
biases.
Disadvantages&

 great expense
 long duration.
Observational Studies

 case–control
 cross-sectional (including
surveys)
 cohort studies
 case–series
case–series study

 it is the simplest design in which the author


describes some interesting observations that
occurred for a small number of patients.
 not include control subjects
 generally not planned studies
 do not involve any research hypotheses
 important descriptive role as a precursor to
other studies
Advantages &
Disadvantages of Case–
Series Studies
 They are easy to write, and the
observations may be extremely useful
to investigators designing a study to
evaluate causes or explanations of the
observations.
Disadvantage
 case–series studies are
susceptible to many possible
biases related to subject selection
and characteristics observed
 hypothesis-generating and not as
conclusive
Case–Control Studies

 Case–control studies begin with


the absence or presence of an
outcome and then look backward
in time to try to detect possible
causes or risk factors
Advantages of Case–Control Studies

 appropriate for studying rare diseases or events


 the quickest
 least expensive studies
 ideal for investigators who need to obtain some
preliminary data prior to writing a proposal for a more
complete, expensive, and time-consuming study.
 good choice for someone who needs to complete a
clinical research project in a specific amount of time.
disadvantages
 Liable to selection/ recall bias
 they depend completely on high-
quality existing records. Data
availability
 Not for rare exposure
 No frequency measure
Cross-sectional studies
 analyze data collected on a group
of subjects at one time rather
than over a period of time.
 designed to determine "What is
happening?" right now. Subjects
are selected and information is
obtained in a short period of time
Advantages of Cross-Sectional Studies

 Feasible
 Cheap
 Quick
 For screening several dis/
exposures, health planning
disadvantage
 Temporal ambiguity
 Not for rare dis, short duration,
rapidly fatal
Cohort Studies

 A cohort is a group of people who


have something in common and who
remain part of a group over an
extended time.
 Cohort studies ask the question "What
will happen?" and thus, the direction in
cohort studies is forward in time
Advantages & Disadvantages of Cohort Studies

 No temporal ambiguity
 No selection/ recall bias
 Many outcomes.
advantage
 Costly
 Timely
 Problem of loss to f.u
Systematic review & meta- Analysis

 Summaries of evidence on a topic


 A review addressing a specific research
question ( on treatment, Dx, prognosis, or
etiology) using explicit methodology of
collecting, selecting, and appraising studies
and, whenever appropriate, synthesizing their
results quantatively.
 Meta-analysis:
 The statistical (quantitative) combination of
results from 2 or more studies addressing the
same research question.
Determining Q type
 Therapy: the effect of different therapy
 Harm: the effect of potentially harmful
agents.
 Diagnosis: establishing the power of an
intervention to differentiate between those
with or e/o a target condition or disease
 Prognosis: estimating the future course of a
patient’s disease.
Type of study
 Treatment:
 Randomized control study
 Harm: cohort study
 Diagnosis:
 Investigators begin by collecting gp of pts
suspecting of having the target condition.
These pts undergoes both the new diagnostic
and a gold standard
 Prognosis:
 Observational study for the prognostic factor
The hierarchy of levels of
evidence

 N of 1 randomized controlled trial


 Systematic reviews of randomized trials
 Single randomized trial
 Systematic review of observational studies
addressing important pt outcomes
 Physiologic studies ( studies of BP, cardiac
output, exercise capacity,..)
 Unsystematic clinical observation
The hierarchy of levels of evidence

  1.           
 A Systematic reviews/ meta-analyses
 B RCTs
 C Experimental designs 
 2.
 A Cohort control studies
 B Case-control studies 
 3.AConsensus conference
 B Expert opinion
 C Observational study
 D Other types of study eg. Interview based, local audit
 E Quasi-experimental, qualitative design 
 4.  Personal communication