UJI KLINIS RCT dan

UN-RCT
Kursus Metodologi
Penelitian PPDS

PREPARING THE RESEARCH DESIGN

Research design is the conceptual structure within
which research would be conducted.
The function of research design is to provide for the
collection of relevant information with minimal
expenditure of effort, time and money.
The preparation of research design, appropriate for
a particular research problem, involves the
consideration of the following :
1. Objectives of the research study.
2. Method of Data Collection to be adopted
3. Source of informationSample Design
4. Tool for Data collection
5. Data Analysis-- qualitative and quantitative

What Is Clinical
Research?
Research that takes place in clinical
settings
Case studies
Case series
Descriptive & retrospective studies
Clinical trials
Randomized controlled trials

What Is A Clinical Trial?

A research study in which a specific
treatment is compared to a control
condition
Control Condition

No treatment
Pre-treatment (pre- vs. post-)
Placebo
Standard care
Another experimental intervention

Types of trials
T r ia l
C o n tr o lle d
R a n d o m is e d
B lin d e d

N o t c o n tr o lle d

N o t r a n d o m is e d

N o t b lin d e d

Types Of Research

Descriptive vs. Analytical

Applied vs. Fundamental
Quantitative vs. Qualitative
Conceptual vs. Empirical
Some Other Types of Research

Quantitative research
Quantitative research often aimed at
testing a research hypothesis. Involve
the analysis of numerical data.
Hypotheses = propositions about
relationships between variables or
differences between groups
Eg. Do patients treated with drug A show
greater improvement than those treated with
Drug B

Other quantitative studies aim to describe

characteristics of a sample
Eg. health needs of a local community

Quantitative techniques
Two main approaches:
Surveys and questionnaires
Experimental designs and
intervention

Staging Clinical Trials

Efficacy vs. Effectiveness
Stages of Development
Discovery & pre-clinical studies
Phase I, II, III, IV

Efficacy and effectiveness

Efficacy Does the intervention work
in tightly controlled conditions?
Strict inclusion/exclusion criteria
Highly standardized treatments
Explicit procedures for ensuring
compliance
Focus on direct outcomes

Efficacy and effectiveness

Effectiveness Does the intervention
work in real world conditions?
Looser inclusion/exclusion criteria
Treatments carried out by typical clinical
personnel
Little or no provision for insuring
compliance
Focus on less direct outcomes (e.g.,
quality of life)

Efficacy and effectiveness studies optimize different

aspects of validity

clinical research [intervention] studies

continuum:
Internal
External
Validity

Validity
Efficacy

Experimen
tal (RCTs)

Effectiveness

Quasiexperimental/
Observational
designs

Most studies fall somewhere in between these anchors

From: Winstein & Lewthwaite, Eugene Michels Forum: CSM, Nashville TN, February

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.

Experiments
Enables researchers to demonstrate
how manipulating one set of
variables (independent variable)
produce systematic changes in
another set of variables (outcome or
dependent variables)

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%)

outcome

RANDOMIZATION

Intervention

Experimental
Design

no outcome
Study
population

outcome
Control
no outcome

baseline
future

time

Study begins here (baseline point)

Experimental Design
Advantages
Best establishes cause-and-effect
relationships

Disadvantages
Artificiality of experiments
Feasibility
Unethical

Characteristics of True
Designs
Manipulation (treatment)
Randomization
Control group