Carl-Fredrik Burman: Optimising designs of clinical trials – beyond optimal design theory

Optimal design theory is a fruitful area of mathematical research. The theory provides helpful tools for practical design decisions in many scientific disciplines, including clinical trials. However, there are many design issues that are not addressed by traditional optimal design theory. I will try to open up a discussion of some important design aspects for the practitioner. For example,

• What is the optimal sample size?
• How to design a trial with multivariate response, aimed at capturing both efficacy and safety?
• Can the trial design be optimised from an ethical perspective?
• Which population should be chosen?
• How to find the optimal trial duration?
• When and how to make interim analysis in a group-sequential trial?
• What is the optimal design of a dose-finding trial, considering the need for a later confirmatory trial?
• Can a trial programme, consisting of inter-related trials, be optimized?