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Randomized Controlled Trials

Key aspects include randomization to allocate subjects, having control groups, blinding to reduce bias, and comparing interventions to no intervention, placebos, or other interventions.

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Jerome G Thampi
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102 views22 pages

Randomized Controlled Trials

Key aspects include randomization to allocate subjects, having control groups, blinding to reduce bias, and comparing interventions to no intervention, placebos, or other interventions.

Uploaded by

Jerome G Thampi
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as PPTX, PDF, TXT or read online on Scribd
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Randomized Controlled

Trials

Dr. Jerome G Thampi


Assistant Professor
Department of Community Medicine
SVMCH & RC, Ariyur
Date: 26/09/2019
Introduction
• Observational Study
• Investigators use data observed in the population to make inference on
relationship between variables

• Experimental study
• The investigators intervene by actively altering one of the variables and then
making inference on the relationship between the variables based on the
outcomes

• Randomized Controlled Trials - Cornerstone of experimental epidemiology


“On the 20th of May 1747, I took twelve patients in the scurvy,
on board the Salisbury at sea. Their cases were as similar as I
could have them. They all in general had putrid gums, the
spots and lassitude, with weakness of their knees. They lay
together in one place, being a proper apartment for the sick in
the fore-hold; and had one diet common to all. … Two of these
were ordered each a quart of cider a day. Two others took
twenty-five gutts of elixir vitriol three times a day, … and so
on. They continued but six days under this course. … The
consequence was that the most sudden and visible good
effects were perceived from the use of oranges and lemons;
one of those who had taken them, being at the end of six days
fit for duty.” — James Lind, 1747
Randomization

• Process by which allocation of subjects to treatment groups is done by


chance, without the ability to predict who is in what group

• Avoids selection bias

• Improves comparability of intervention and control arm populations

• Individually randomized or cluster randomized trials


”Controlled” Trials
• Eligibility criteria

• Specified hypotheses

• Primary and secondary endpoints to address hypothesis

• Clear methods for enrollment and follow-up

• Rigorous monitoring

• Analysis plans and rules for stopping rules


Interventions that can be evaluated
• New drugs and new treatment modalities
• New medical and health care technology
• New methods of primary prevention
• New programs for screening
• New ways of organizing and delivering health services
• New community health programs
• New behavioural intervention programs
Groups that can be compared
• Intervention vs. no Intervention

• Intervention vs. placebo or sham

• Intervention A vs. Intervention B


Study Designs

• Parallel design vs Cross-over design

• Factorial designs e.g., intervention A, intervention B, intervention A+B vs control


Other Classifications

• Single intervention vs single control,

• Multiple interventions vs single control arm,

• Equivalency trials: head-to-head comparison of 2 or more


interventions, without a control group (e.g., contraceptive trials)
Why do RCTs?
• Observational studies are subject to potential bias and confounding:
• Self-selection
• Observer bias
• Secular trends

• RCTs provide the ”gold standard” for proof of a concept


Blinding
• Randomization eliminates influence of confounding factors of biases
at time of allocation

• Blinding controls post-randomization confounders

• Absence of blinding leads to:


• Performance bias
• Ascertainment bias
Techniques of Blinding
• Based on involvement of stakeholders

• Four types of stakeholders

• Study subjects
• Research investigators
• Outcome assessors
• Data analysts
Open Label Trials
• Open, non-blinded or unblinded

• All are aware of group assignment of participants

• Eg: Effectiveness of music therapy in reducing the anxiety of patients


undergoing Micrographic Mohs Surgery (MMS)
Single Blinded Trials
• Blinding of any one group of stakeholders

• Usually the study subjects or outcome assessors

• Eg: Effectiveness of imagination-induced relaxation intervention in


reducing post-operative pain among children aged 8-12 years; nurses
who collected the data were blinded
Double Blinded Trial
• Any two groups of individuals are blinded

• Typically recommended in drug trials

• Usually the investigators and study subjects

• Eg: placebo therapy vs Transcutaneous Electrical Nerve Stimulation


(TENS) on outcomes of patients with fibromyalgia; study subjects and
outcome assessors were blinded
Triple Blinded Trials
• Three groups of people are blinded to intervention assignments

• Usually the subjects, investigators and outcome assessors are blinded

• Eg: Efficacy of doxycycline and rifampin for patients with Alzheimer’s


disease – triple blinded for entire study period
Quadruple Blinded Trial

• All four groups are blinded

• Eg: effectiveness of peri-operative n-acetylcysteine in preventing renal


dysfunction among high-risk patients undergoing CABG
Conclusion

• Gold standard for proof of concept

• Randomization is the “heart”

• Blinding

• Best available evidence in Evidence-Based Medicine


Thank You

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