School of Mathematical Sciences

Novel designs for trials with multiple treatments and biomarkers menu

Novel designs for trials with multiple treatments and biomarkers

J. M. S. Wason, University of Cambridge
Thu, 09/02/2017 - 16:30
Seminar series: 

Multi-arm trials are increasingly being recommended for use in diseases where multiple experimental treatments are awaiting testing. This is because they allow a shared control group, which considerably reduces the sample size required compared to separate randomised trials. Further gains in efficiency can be obtained by introducing interim analyses (multi-arm multi-stage, MAMS trials). At the interim analyses, a variety of modifications are possible, including changing the allocation to different treatments, dropping of ineffective treatments or stopping the trial early if sufficient evidence of a treatment being superior to control is found. These modifications allow focusing of resources on the most promising treatments, and thereby increase both the efficiency and ethical properties of the trial.

In this talk I will describe some different types of MAMS designs and how they may be useful in different situations. I will also discuss the design of trials that test efficacy of multiple treatments in different patient subgroups. I propose a design that incorporates biological hypotheses about links between treatments and biomarker subgroups effects of treatments, but allows alternative links to be formed during the trial. The statistical properties of this design compare well to alternative approaches available.