Patient Oriented Research I: Allen McCutchan, M.D., M.Sc.

Objectives: This course will develop and apply the theory of clinical trial design and analysis, discuss the practical issues of financing and implementation of clinical trials, and describe issues of monitoring trials and working in cooperative groups. The scholar will design and present to a group of peers a concept sheet for a phase I/II and phase II/III clinical trial.

Course Content:

Topic Content

Hypothesis Generation and Formulation

Framework, Types, and Characteristics of Good Research Questions and formulating a testable hypothesis

Introduction to POR

Definition of POR; Origins & Scientific Basis; Causality; Validity of Inference; Classification of Clinical Research
Measurement Objectives, classes, and characteristics of measures in POR and their validity, accuracy, and precision

Fundamentals of Clinical Trials

Study Objectives, Target Populations, Comparison Groups, Blinds, Outcomes and Endpoints

Overview of the Process of Drug and Device Development

Preclinical and clinical development of drugs from an industrial prospective

Statistical Issues in Clinical Trials

Sample Size/ Power Analysis; Randomization Procedures; Analytic Strategies - Sequential, Interval and Terminal; Analysis Plan

Selecting and Measuring Outcomes and Study Implementation I

Outcomes: Selecting outcomes: who gets counted, clinical vs surrogates, and simple vs compound. Implementation: Defining, recruiting, consenting, initially assessing, tracking, and retaining participants; IRB submission.

Study Implementation II Assessing Adherence; Study Start-up Study Monitoring for adverse events and interacting with the IRB; Completing the study, DSMB review
Complex Trial Design Multi-centered studies, Crossover and Factorial Designs, equivalence vs superiority trials, and testing diagnostics