MDAndersonX: CERTaIN: Pragmatic Clinical Trials and Healthcare Delivery Evaluations

• Describepragmatic clinical trial designs
• Identify and classify clinical trial designs
• Describe the statistical methods used to analyze pragmatic clinical trial data

Overview of Design Options for Pragmatic Clinical Trials

• Types of trial designs including randomized clinical trials
• Sources of errors that could lead to erroneous trial results

Outcome Measures in Clinical Trials

• Measuring health status and disease
• Characteristics of a good outcome measure
• Types of outcome measures in clinical trials

Non-inferiority Trials

• Non-inferiority trial designs and how they differ from other designs
• Interpretation and reporting of results

Basic Analytic Methods

• Matching research questions with statistical analysis methods
• Interpreting results
• Distinguishing intent to treat from per protocol analyses
• Problems with missing data

Basic Power and Sample Size Calculations

• Relevant issues to estimate sample size in clinical trials
• Interpreting study power
• Calculating sample size using online calculators

SMART: Adaptive Treatment Strategies

• Research questions which can benefit from an adaptive design
• Analytic methods for adaptive designs
• Interpreting results of adaptive designs

Introduction to Bayesian Methods

• Statistical inference
• Comparison of frequentist and Bayesian approaches and inference

Bayesian Designs

• Bayesian method and adaptive design introduction
• Adaptive randomization and predictive probability
• Bayesian design trial interpretation
• Software tools for conducting Bayesian designs

Quasi-Experiment in Health Services Research

• Need for quasi-experiments and their limitations
• Difference-in-Differences (DID) estimators and models

Adaptive Trial Design

• Limitations of randomized traditional trials
• Common adaptive approaches and their challenges
• Covariate and outcome adaptive randomization

Logistics of Clinical Trials

• Assessing impact, feasibility and funding
• Scientific review process
• Study initiation, continuing review and audits
• Working with sponsors