Efficient trial designs to evaluate treatments in a public health emergency

Babiker, A.

In global health pandemics caused by novel infectious pathogens entering the human population, there is an acute need for identifying effective treatments very rapidly. However, traditional phase 3 clinical trials are inefficient. They usually focus on addressing a single primary question, take a very long time to implement from conception to publication of findings, and are very costly. There is a need for improved designs for clinical trials to enable rapid, efficient and better evaluation of treatments; both a need to identify effective treatments, and equally important, to identify ineffective ones early, in order to direct resources to the most promising interventions. It is increasingly recognised that efficiencies could be gained by asking multiple questions in a single protocol. Adaptive platform trials with multi-arm multi-stage (MAMS) designs offer a mechanism for the systematic evaluation of several investigational agents simultaneously and the abandonment of those that do not demonstrate sufficient activity, thereby significantly speed up the rate at which answers can be achieved. This talk will review some general features of adaptive Platform Trials, their advantages and challenges in their design and implementation in a pandemic setting, drawing from experience and lessons learned in conducting such trials in COVID-19.