Design and Interpretation of Clinical Trials

Clinical trials are experiments designed to evaluate new interventions to prevent or treat disease in humans. The interventions evaluated can be drugs, devices (e.g., hearing aid), surgeries, behavioral interventions (e.g., smoking cessation program), community health programs (e.g. cancer screening programs) or health delivery systems (e.g., special care units for hospital admissions). We consider clinical trials experiments because the investigators rather than the patients or their doctors select the treatment the patients receive. Results from randomized clinical trials are usually considered the highest level of evidence for determining whether a treatment is effective because trials incorporates features to ensure that evaluation of the benefits and risks of treatments are objective and unbiased. The FDA requires that drugs or biologics (e.g., vaccines) are shown to be effective in clinical trials before they can be sold in the US. The course will explain the basic principles for design of randomized clinical trials and how they should be reported. In the first part of the course, students will be introduced to terminology used in clinical trials and the several common designs used for clinical trials, such as parallel and cross-over designs. We will also explain some of the mechanics of clinical trials, like randomization and blinding of treatment. In the second half of the course, we will explain how clinical trials are analyzed and interpreted. Finally, we will review the essential ethical consideration involved in conducting experiments on people.

• Types of Trial Designs
• This week, we explore different types of trial designs, including parallel, crossover, group allocation, factorial, large simple, equivalency, non-inferiority, and adaptive designs.
• Randomization and Masking
• This week we discuss two key design features of randomized clinical trials used to protect against bias, randomization and masking.
• Outcomes and Analysis
• This week focuses on a key design issue - selecting the primary outcome. We will also cover the gold standard for analysis of clinical trials, which is including all the participants in the analysis regardless of their actual treatment.
• Ethics
• This week focuses on a key issue in the field of clinical trials, the ethics of experimentation in humans.
• Reporting Results
• This week, we focus on reporting results of clinical trials in publications. We cover the Consolidated Standards of Reporting Trials (CONSORT) guidelines.
• Randomized Clinical Trials
• This week, we focus on whether RCTs are still the gold standard for evaluating evidence.