Abstract
With current success rates of confirmatory studies being only around 50%, new approaches to drug development are paramount. Many trials fail simply because ineffective treatments are identified too late. In this paper, we discuss the utility of multi-arm studies with treatment selection as a potential strategy that can reduce the high attrition rate. We illustrate the large gains in efficiency that are possible based on an example in Alzheimer’s disease while outlining the additional challenges that need to be overcome to implement such studies.
Highlights
Thomas JakiThe development of medicinal products and before embarking on such a design. We health technologies is time consuming and provide an illustrative example highlighting very costly
Because treatments are removed from consideration early and the trial can be stopped before the maximum number of patients are recruited, the required sample size of such studies will typically be smaller than a multi-arm study without treatment selection
Besides the added efficiency that will be illustrated below, a further advantage of a multi-arm study with treatment selection, illustrated in Figure 2, is that one can use an interim analysis to mark the end of Phase II and beginning of Phase III thereby allowing a seamless Phase II/III study which removes the white
Summary
The development of medicinal products and before embarking on such a design. We health technologies is time consuming and provide an illustrative example highlighting very costly. Group-sequential designs that allow the study to stop early, either because the evidence is already sufficient to claim superiority of the treatment over control or because it is unlikely to reach such a claim, have been developed [7,8] and are routinely used in practice. Multi-arm studies can be made more efficient by adding interim analyses that allow early stopping because the evidence collected is already sufficient to conclude that one or more treatments is superior to control or to stop because none of the experimental treatments looks sufficiently promising. Because treatments are removed from consideration early and the trial can be stopped before the maximum number of patients are recruited, the required sample size of such studies will typically be smaller than a multi-arm study without treatment selection. Besides the added efficiency that will be illustrated below, a further advantage of a multi-arm study with treatment selection, illustrated in Figure 2, is that one can use an interim analysis to mark the end of Phase II and beginning of Phase III thereby allowing a seamless Phase II/III study which removes the white
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