Advanced Clinical Trial Design

About

This course will provide an introduction to advanced topics of clinical trial design. The focus will be on trial planning especially sample-size calculations within a hypothesis testing framework and implications for study design. Topics are selected based on practical experience of the faculty and relevance over recent years. In the course we will cover platform trials, pragmatic trials including routine-data supported or registry trials, cluster-randomized trials, non-inferiority (combined with superiority) study questions, multi-arm versus sequential multiple assignment randomized trials, studies within a trial (SWATs), and estimands. The faculty reserves the right to change focus regarding some of the topics based on developments over the coming months.

Course leader

PD Dr. Sven Trelle CTU Bern, University of Bern, Switzerland Prof. Matthias Briel Department of Clinical Research, University of Basel, Switzerland

Target group

Clinical researchers, (clinical) epidemiologists, medical statisticians, or any research stakeholder with an interest in trial methodology. Participants should have some experience with developing and conducting randomized clinical trials in their professional role.

Students should bring their own portable computers. A course license for Stata® will be available, to be installed before arrival. University of Bern IT staff onsite can provide help upon request per e-mail (it@ispm.unibe.ch)

Course aim

By the end of this course participants will have:
- An understanding of the concept of platform trials
- An understanding of what constitutes a pragmatic trial and how it differs from an efficacy trial
- An understanding of the potential and limitations of routinely-collected data for randomized trials
- An understanding of the specifics of non-inferiority and cluster-randomized trials
- An understanding of trial designs that aim to test multiple hypotheses
- An understanding of the implications of these design choices on sample-size calculation and how to perform them in practice
- An understanding of the concept and value of SWATs
- An understanding of different estimands and the implications for trial planning

Fee info