Randomized Clinical Trials

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 3 0/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 1

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 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 2

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 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 3 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 . 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 4

5. Steps in conducting a RCT 30/9/2022 3 www.clinosol.com | follow us on social media @ clinosolresearch 5

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 3 0/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 6

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 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 7

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 3 0/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 8

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 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 9

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 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 10

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 Single blind trial –participants is not aware whether he belongs to study or control group Double blind trial – neither the participant nor the investigator is aware Triple blind trial – the participant, investigator and person analyzing data all are blind 30/9/2022 3 www.clinosol.com | follow us on social media @clinosolresearch 11

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 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 12

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. 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 13

14. References Park’s textbook of preventive and social medicine, park. K. Leon Gordis . Epidemiology. Elseiver : 2004 Wikipedia 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 14

15. Thank You! www.clinosol.com (India | Canada) 9121151622/623/624 [email protected] 30/9/2023 www.clinosol.com | follow us on social media @clinosolresearch 15