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.