Experimental Study

Stefania Widya S., S.Gz, MPH

 Defining Experimental Study Design
A study in which a population is selected for
a planned trial of a regimen, whose effects
are measured by comparing the outcome of
the regimen in the experimental group
versus the outcome of another regimen in
the control group. Such designs are
differentiated from observational designs by
the fact that there is manipulation of the
study factor (exposure), and randomization
(random allocation) of subjects to treatment
(exposure) groups.
Experimental Process
Select and Define The Problem
Select Participants and Instrument
Selection of Research Plan
Execution of Research Plan
Analysis of Data
Formulation of Conclusion
Statement of a hypothesis
indicating a causal
relationship between
variables
• Random selection of a
sample of subjects from
a larger population
• Random assignment of
members of the sample
to each group
• Selection of valid and
reliable instruments
• Comparison of two
different approaches
• Comparison of new and
existing approaches
• Comparison of different
amounts of a single
approach
• Sufficient exposure to the
treatment
• Substantively different
treatments
 Why Performed?
1. Provide stronger evidence of the effect
(outcome) compared to observational designs,
with maximum confidence and assurance
2. Yield more valid results, as variation is
minimized and bias controlled
3. Determine whether experimental treatments
are safe and effective under “controlled
environments” (as opposed to “natural
settings” in observational designs), especially
when the margin of expected benefit is
doubtful / narrow (10 - 30%)
 RANDOMIZATION outcome
Intervention
no outcome
Study
Experimental Design

population
outcome
Control
no outcome
baseline
future

time
Study begins here (baseline point)
 Types of trials
Trial

Controlled Not controlled

Randomised Not randomised

Blinded Not blinded

 Selection of Subjects

• The criteria for determining who will or will not be

included in the study must be spelled out with great
precision, and in writing before the study is begun.
• the selection criteria must therefore be precisely stated
 Type of Control
• Historical Control
- comparison group from the
past
- Choosen by selecting subjects
with themsame disease who
were treated before the new
therapy became available.
- This type of design seems
inherently simple and
attractive.
• Simultaneoud randomized
control
- the assignment system was
predictable
- it was possible for the
researcher to know what the
assignment of the next
subjects would be.
- .
 Randomization

• Randomization is the best approach in the design of a

trial.
• Randomization means, in effect, tossing a coin to decide
the assignment of a patient to a study group.
• The critical element of randomization is the
unpredictability of the next assignment.
• Today, particularly for large trials, randomization is most
often carried out using a computer
 TREATMENT
• First, we must know to which treatment group the
patient was assigned.
• In addition, we must know which therapy the patient
actually received.
• It is important to know, for example, if the patient was
assigned to receive treatment A, but did not comply.
• A patient may agree to be randomized, but may later
change his or her mind and refuse to comply.
• Conversely, it is also clearly important to know whether a
patient who was not assigned to receive treatment A
may have taken treatment A on his or her own, often
without realizing it.
 OUTCOME
• The need for comparable measurements in all study
groups is particularly true for measurements of outcome.
• Such measurements include both improvement (the
desired effect) and any side effects that may appear.
• There is, therefore, a need for explicitly stated criteria for
all outcomes to be measured in a study.
• Once the criteria are explicitly stated, we must be certain
that they are measured comparably in all study groups..
 MASKING (BLINDING)
• Masking involves several components:
• First, we would like the subjects not to know which
group they are assigned to.
• This is of particular importance when the outcome is a
subjective measure, such as headache or low back pain.
If the patient knows that he or she is receiving a new
therapy, enthusiasm and certain psychological factors on
the part of the patient may operate to elicit a positive
response even if the therapy itself had no positive
biologic or clinical effect.
 CROSSOVER
- a new treatment is being compared with current
treatment.
- Subjects are randomized to new treatment or current
treatment
- After being observed for a certain period of time on one
therapy and after any changes are measured.
- the patients are switched to the other therapy
- Both groups are then again observed for a certain period
of time
 CROSSOVER
- a new treatment is being compared with current
treatment.
- Subjects are randomized to new treatment or current
treatment
- After being observed for a certain period of time on one
therapy and after any changes are measured.
- the patients are switched to the other therapy
- Both groups are then again observed for a certain period
of time
 FACTORIAL DESIGN

• Assuming that two drugs are

to be tested, the anticipated
outcomes for the two drugs
are different, and their modes
of action are independent, one
can economically use the same
study population for testing
both drugs.
1. Tujuan Penelitian
2. Desain peneliatan (gambarkan dalam bentuk diagram)
3. Treatment
4. Subyek (kriteria inklusi, eksklusi, dan randomisasi, serta drop off)
5. Hasil
6. Kesimpulan