Multi-outcome trials with a generalised number of efficacious outcomes

Abstract: Existing multi-outcome designs focus almost entirely on evaluating whether all outcomes show evidence of efficacy or whether at least one outcome shows evidence of efficacy. While a small number of authors have provided multi-outcome designs that evaluate when a general number of outcomes show promise, these designs have been single-stage in nature only. We therefore propose two designs, of group-sequential and drop the loser form, that provide this design characteristic in a multi-stage setting. Previous such multi-outcome multi-stage designs have allowed only for a maximum of two outcomes; our designs thus also extend previous related proposals by permitting any number of outcomes.