Abstract
BackgroundMany recent Stroke trials fail to show a beneficial effect of the intervention late in the development. Currently a large number of new treatment options are being developed. Multi-arm multi-stage (MAMS) designs offer one potential strategy to avoid lengthy studies of treatments without beneficial effects while at the same time allowing evaluation of several novel treatments.In this paper we provide a review of what MAMS designs are and argue that they are of particular value for Stroke trials. We illustrate this benefit through a case study based on previous published trials of endovascular treatment for acute ischemic stroke.We show in this case study that MAMS trials provide additional power for the same sample size compared to alternative trial designs. This level of additional power depends on the recruitment length of the trial, with most efficiency gained when recruitment is relatively slow. We conclude with a discussion of additional considerations required when starting a MAMS trial.ConclusionMAMS trial designs are potentially very useful for stroke trials due to their improved statistical power compared to the traditional approach.
Highlights
Multi-arm trials A multi-arm trial compares several different experimental treatments against a common control group within a single study
While it is undesirable for every trial to conclude superiority of the experimental treatment – this would raise the question why such studies are done at all – it is widely agreed that these figures are unacceptably high
In this work we will discuss the potential utility of combining multiple experimental arms into a single multi-arm trial for improving evaluating treatments in stroke
Summary
It is well recognised that drug development is costly and time consuming [1] yet in recent years about half of Phase III trials and 80% of Phase II studies undertaken have been unsuccessful [2, 3]. The situation in stroke is no better with several recent studies failing to show superiority of the experimental treatment [4,5,6]. In this work we will discuss the potential utility of combining multiple experimental arms into a single multi-arm trial for improving evaluating treatments in stroke. We consider going further and considering an adaptive approach called a multi-arm multi-stage (MAMS) design, which allows elimination of ineffective treatments while reducing the number of patients allocated to a control treatment. We first consider these designs and their advantages in more detail. We end the paper with discussion of benefits and limitations of multi-arm and MAMS designs
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