In the pursuit of advancing medical knowledge and improving patient care, randomized clinical trials (RCTs) stand as the gold standard for rigorous and unbiased research. They are the backbone of evidence-based medicine, offering invaluable insights into the effectiveness and safety of medical interventions. Let's delve into the world of RCTs, exploring their significance, key principles, and their critical role in healthcare. The Foundation of Evidence-Based Medicine RCTs are the linchpin of evidence-based medicine, a paradigm that emphasizes clinical decisions based on empirical evidence and scientific inquiry. The fundamental premise of RCTs is to provide a structured and unbiased way to evaluate the efficacy and safety of medical treatments, interventions, or drugs. Key Principles of Randomized Clinical Trials: Randomization: Participants are randomly allocated into two or more groups, ensuring that each group is comparable at the outset. This minimizes the risk of bias in group assignment, enhancing the reliability of the results. Control Group: RCTs typically include a control group that receives either a placebo or an existing standard treatment. The experimental group receives the new intervention under investigation. Blinding: To minimize observer and participant bias, RCTs often employ blinding. Single-blind studies conceal information from either the participants or the investigators, while double-blind studies conceal information from both. Outcomes and Endpoints: RCTs define specific outcomes or endpoints, such as disease progression, side effects, or mortality rates, to measure the intervention's impact.

1. Welcome
Randomized Clinical Trials
G.Naga malleshwari
B. Pharmacy
Student ID – 182/092023
30/9/2023
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2. Contents
 Introduction
 Importance
 Steps in RCT
 -Protocol design
 -Selecting reference and experimental population
 -Randomization
 -Intervention
 -Follow up
 -Assessment of outcome
 Types of RCT
 Conclusion
 References
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3. INRODUCTION
DEFINITION:
“An epidemiological experiment in which subjects in a population are randomly
allocated into groups usually called study and control groups to receive and not receive
an experimental prevention or therapeutic procedure, maneuver, or intervention”.
- John M. Last, 2001
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RCTs are quantitative, comparative, controlled experiments in
which investigators study two or more interventions in a series
of individuals who receive them in random order. (Stedman’s
medical dictionary)
 The RCT is one of the simplest and most powerful tools in
clinical research.
 To provide high-quality evidence for decision-making in
healthcare
History

4. Importance of randomized clinical trials
• Gold Standard: RCTs are considered the gold
standard for evaluating the effectiveness of medical
interventions.
• Evidence-Based Medicine: They form the basis for
evidence-based medical practice.
• Regulatory Approval: RCTs often play a crucial
role in gaining regulatory approval for new
treatments.
• Improved Patient Care: RCTs lead to improved
patient outcomes and healthcare decision-making.
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5. Steps in conducting a RCT
Designing a protocol
Selecting reference and experimental population
Randomization
Manipulation and Intervention
Follow up
Assessment of outcome
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6. Designing a protocol
-Aims and objectives of the study
-Research Questions to be answered
-Criteria for selection, Sample size, Procedure
for allocation of study and control groups
-Patient consent, adverse events
-Treatments to be applied
-Responsibilities of people involved in trial
 Once a protocol as been made its should be
strictly adhered
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7. Selecting reference and experimental
population
 Reference or target population: population to which the
findings of trial, if found successful, are expected to be
applicable(eg. Drugs, vaccines, or any other procedures)
 Experimental or study population: actual population that
participates in the experimental study
Criteria – must give informed consent
-eligible for the trial
-belong to reference population
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8. Randomization
• Procedure; participants are allocated into study and control groups
• It is done to Eliminates bias and allows comparability
• Both groups should be alike with regards to certain variables that might affect the
outcome of the experiment
• Best done by using table of random numbers
Methods of randomization should always be reported(CONSORT).
Types:
Stratified - randomly assigning participants to treatment groups based on predefined
and objective characteristics
Blocked –participants are randomly assigned to treatment groups in small blocks of
four or six participants
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9. Manipulation/ Intervention
 Having formed the study and control groups, the next step is
to manipulate the study groups by withdrawal or reduction of
the suspected causal factor (eg: this may be drug, vaccine
etc.) as laid down in the protocol
 It creates an independent variable
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10. Follow up
Implies examination of the experimental and control group
subjects
-at defined intervals of time,
-in a standard manner, with equal intensity, under the same given
circumstances
Attrition: some loses to follow up are inevitable due to factors like
death, migration or loss or interest
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11. Assessment of results
Positive results- benefits such as incidence or severity of disease
Negative results- severity and frequency of side effects,
complications , including death
 Biases: subject variation, observer bias, evaluation bias
Can be minimized by blinding
o Single blind trial –participants is not aware whether he
belongs to study or control group
o Double blind trial – neither the participant nor the
investigator is aware
o Triple blind trial –the participant, investigator and person
analyzing data all are blind
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12. Types of randomized clinical trials
Clinical trials
-immunization, drug treatment
Preventive trials
-vaccine trials
Risk factor trials
-control of BP in preventing CHD
Cessation experiments
-smoking and lung cancer
Trial of etiological agents
-retrolental fibroplasia
Evaluation of health services
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13. Conclusion
 Randomization is the heart of Randomized clinical trials.
 RCT is a gold standard in terms of identifying causality.
 Provides the strongest evidence.
 Randomization is an attempt to eliminate bias and allow for
comparability.
 Randomization is useful in hiding the knowledge of particular
treatment (blinding).
 Applications- evaluation of treatment, prevention and health
care services.
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14. References
 Park’s textbook of preventive and social medicine, park.
K.
 Leon Gordis. Epidemiology. Elseiver: 2004
 Wikipedia
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15. Thank You!
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(India | Canada)
9121151622/623/624
info@clinosol.com
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