Design your scientific research

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 Research Methodology

Observational Study Designs:

Descriptive Studies:
Case reports
Case series
Cross-sectional surveys
Analytical Studies:
Prospective Cohort studies
Retrospective Case-control studies
Experimental Study Design:
Intervention studies
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Case Reports:
Unusual occurrence
Or
Rare incidence

Useful in recognition of new entities

Case Series:
Collection of individual case reports.
Help formulate a hypothesis concerning
possible risk factors.

no control group  no statistical validity

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 Cross Sectional Studies

* Conducted among individuals in a well-

defined population.
* Conducted in a specified time period.

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 Prospective Cohort Study
• Group of individuals
– sharing same experience
– followed up for specified period of time

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 Cohort Studies

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 Cohort studies
exposed

Incidence among
exposed

unexposed

Incidence among
unexposed

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Retrospective Case Control Studies

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 Experimental Study Design

Construct hypothesis

Design experiment

Perform experiment

Record and analyze results

Draw and Publish Conclusions

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 Experimental Study Design
• Hypothesis-driven Experimentation
which
– tests an intervention
– follows a protocol that specifies in
detail
• hypothesis that is being tested
• eligibility to enter the study
• application of the intervention
• collection and analysis of data

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 Randomized controlled studies
Are experimental studies that
traditionally have been considered
the gold standard in establishing the
efficacy of an intervention in treating
or preventing a clinical disorder.

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 Randomized controlled studies
Involve the random allocation of
different interventions (treatments or
conditions) to subjects. As long as the
numbers of subjects are sufficient,
randomization is an effective method
for balancing confounding factors
between treatment groups.

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 Confounding factor
Is an extraneous variable in a statistical
model that correlates (positively or
negatively) with both the dependent
variable and the independent variable.
Leads to 'false positive' conclusion that the
dependent variables are in a causal
relationship with the independent
variable.

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 Confounding factor
Thus, confounding is a major threat
to the validity of inferences made
about cause and effect, i.e.
internal validity, as the observed
effects should be attributed to the
confounder rather than the
independent variable
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 Confounding factor
Assume that a child's weight and a
country's gross domestic product (GDP) rise
with time. A person carrying out an
experiment could measure weight and GDP,
and conclude that a higher GDP causes
children to gain weight, or that children's
weight gain boosts the GDP. However, the
confounding variable, time, was not
accounted for, and is the real cause of both
rises.
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 Confounding factor
• By definition, a confounding variable is
associated with both the probable cause and
the outcome.
• The confounder is not allowed to lie in the
causal pathway between the cause and the
outcome:

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• Being female does not always lead to
smoking tobacco, and smoking tobacco
does not always lead to cancer.
Therefore, in any study that tries to
elucidate the relation between being
female and cancer should take smoking
into account as a possible confounder. In
addition, a confounder is always a risk
factor that has a different prevalence in
two risk groups (e.g. females/males).
(Hennekens, Buring & Mayrent, 1987).
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 Possible confounder

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 Randomized controlled studies
Participants are randomly assigned into the
control group or the investigational group.

The control group receives the typically used or

approved treatment

The investigational group receives the treatment

or intervention being studied.

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 Placebo

• Definition: Latin for “I shall please”

• A preparation with no effective or
active ingredient. It should resemble
the actual therapy in every aspect
(shape, colour, smell and taste)

• Placebo effect:
• 1. the psychological theory: it's all in
your mind
• 2. the process-of-treatment theory
• 3. the nature-taking-its-course theory
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 Types of control groups
• Placebo control group

• Dose-response control group

• Active control group

• No treatment control group

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 TYPES OF STUDIES

• Open trial
• Blind trials
1. Single-blind trial
2. Double-blind trial
3. Triple-blind trial
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 Open trial

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 Blinding or Masking
• Hides the specific
intervention to which
each subject is
assigned

• May apply to either

subjects or
investigators (single
blinding) or both
(double blinding)

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 Triple-blind trial
The subject, researcher and person
administering the treatment (often a
pharmacist) are blinded to what is
being given. Alternately, it may mean
that the patient, researcher and
statistician are blinded

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 ACTIVE CONTROL STUDIES
• when a treatment exists that is clearly
better than doing nothing for the
subject ( giving them the placebo), the
alternate treatment would be a
standard-of-care therapy.
• The study would compare the 'test'
treatment to standard-of-care therapy.

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 Systematic reviews
Often coupled with meta-analyses of
randomized controlled trials have
emerged during the EBM movement
to assume their position atop the
evidence pyramid.
• levosimendan
• Thrombolytic therapy

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 Meta-analysis

• This type of study involves

analysis of multiple similar
studies (already completed)

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 Common Errors in Research

1. Selective observation.

2. Inaccurate observation.

3. Overgeneralization.

4. Made-up information.

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 Common Errors in Research

5. Illogical reasoning.

6. Ego involvement in understanding.

7. Premature closure of inquiry.

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