Clinical trials are the gold standard study design for producing high-quality evidence and are a regulatory requirement for evaluating the safety and efficacy of drugs. They are very expensive and time-consuming to conduct and therefore design methods for making them more efficient are very important.
This course provides an overview of three main types of innovative study design:
1) adaptive trials;
2) master protocols (basket, platform, and umbrella designs);
3) designs that allow using external information to augment the trial, including synthetic control methods and Bayesian borrowing.
The course keeps statistical theory to an absolute minimum. Case studies are provided for each type of innovative design and focus placed on operational and regulatory issues as well as statistical issues.