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Clinical Trials Fundementals Flashcards

Introduction to Clinical Trials

  • Course Name: MEDC0014

  • Session Title: Fundamentals of Clinical Trials

  • Instructors: Jemima Thompson & Hannah Plant

  • Date: 25-Feb-2025

Who are We?

  • Institutions: UCL

  • Team Members: Jemima Thompson, Hannah Plant, Giulia Pelizzari

Learning Objectives

  • Recognize, define, and distinguish between different types of clinical studies

    • Interventional Studies

    • Observational Studies

  • Define the phases of clinical trials and understand the aims of each phase

  • Describe the fundamental principles of clinical trials

Types of Studies

  • Two Main Categories:

    • Interventional Studies: Involves testing an intervention on participants.

    • Observational Studies: Involves monitoring participants without intervention.

What is a Clinical Study?

  • Research that uses human volunteers aimed at adding to medical knowledge.

  • Main Types:

    • Interventional Studies: Investigates the effect of an investigative treatment.

    • Observational Studies: Observes outcomes without manipulation.

Interventional Studies

  • Definition: Tests if an intervention affects an outcome.

  • Example: Administering beta-blockers to see if it reduces blood pressure.

  • Focuses on health-related events as outcomes.

Observational Studies

  • Definition: Observes the course of a disease or relationships between risk factors and outcomes without intervention.

  • Example: Investigating how lifestyles affect blood pressure in older adults.

Hierarchies of Evidence

  • Levels of Evidence (from highest to lowest):

    • Meta-analysis & Systematic Reviews

    • Randomized Controlled Trials (RCT)

    • Cohort Studies

    • Case-Control Studies

    • Case Series, Case Reports

    • Editorials, Expert Opinion

Types of Observational Studies

  • Cross-sectional Studies: Snapshot of a population.

  • Cohort Studies: Follow subjects over time based on exposures.

  • Case-Control Studies: Compare cases and controls based on outcomes.

  • Ecological Studies: Measure variables at a group level to study correlations.

Cross-Sectional Study

  • Aim: Describe population regarding outcome and risk factors.

  • Typically carried out via surveys to gather prevalence information.

Cohort Study

  • Definition: Study subjects over time, comparing outcomes based on exposure.

  • Selection based on characteristics relevant to the condition being studied.

Case-Control Study

  • Definition: Compares a group with an outcome to a group without.

  • Example: Investigating tobacco smoking's relationship with lung cancer.

Ecological Study

  • Measures variables at the group level to analyze relationships between exposures and outcomes.

  • Example: Variance in treatment patterns across populations.

Clinical Trials Definition

  • A type of interventional study involving human participants aimed at evaluating interventions.

  • Must undergo rigorous testing through phases post-laboratory and animal studies to determine safety and efficacy.

Randomized Controlled Trials (RCT)

  • Considered the gold standard for intervention evaluation.

  • Randomization: Keys to mitigate selection bias by randomly assigning participants to groups.

Bias in Trials

  • Definition: A tendency to deviate from the true value in estimates.

  • Affects objective analysis and can lead to under or overestimation of effects.

Clinical Trial Phases

  • Phases involve:

    • Phase I: Initial safety on <100 healthy volunteers.

    • Phase II: Tests on a larger group (100-500) for safety and activity.

    • Phase III: Efficacy and safety assessed on 1000s of patients for regulatory approval.

    • Phase IV: Post-marketing studies to find long-term effects in various populations.

Characteristics of Good Clinical Trials

  • Inclusion of a control group for comparison.

  • Randomization and blinding (when appropriate) to minimize bias.

  • Adequate sample size to detect meaningful differences.

Control Groups

  • Participants not receiving the treatment serve as a baseline to evaluate treatment effectiveness.

Randomization Mechanism

  • Allocates treatment randomly to avoid bias from systematic differences.

Blinding in Trials

  • Definition: Patients and/or evaluators unaware of treatment assignments.

  • Aims to minimize bias in evaluation.

Importance of Sample Size

  • Sufficient size enhances statistical power to detect differences.

  • Ethical implications if too large or insufficiently powered.

Summary of Clinical Trials

  • Can find new treatments/strategies that improve outcomes or reveal no benefits or harm.

  • Essential for evidence-based practice in healthcare to avoid ineffective or harmful treatments.

Future Sessions

  • Overview of topics for sessions 2-10, including outcome measures, ethics, and effective management of trials.

Assessments

  • Formative Assessment: Multiple-choice questionnaire due March 11, 2025.

  • Summative Assessment: Combines MCQ and group presentation due May 20/22, 2025.

Expectations from Students

  • Strive for 100% attendance.

  • Engage actively in discussions and question sessions.

Questions and Communication

  • Utilize discussion forums on the module Moodle page for peer support.

  • Direct inquiries to instructors via email for unsolved issues.