Single arm trials are the bane of my life: a seminar by Adrian Mander

This event has ended.

The single-arm trial is a very simple design: everyone gets a treatment (mostly within oncology) and wait to see if it works, in terms of a binary response. It assumes that those not treated will not get better or have a small chance (p0) of getting better with the hope that the treatment improves response to the chance p1. One improvement to this design is an adaptive version where an interim analysis is carried out after observing the outcomes of the first, n1 participants. The intention of the interim analysis is to stop the trial early as the treatment is not working. This adaptive version of the trial design is often labelled Simon’s two-stage design and it is perhaps the most used adaptive design in practice. However, from results of a review it shows how a design can be abused with poor reporting and adherence to a design.  I will describe different ways of improving this design such as doing many interims, being Bayesian, adding in biomarkers and comparing this approach to randomised designs. Perhaps in the end I might grow to like this design.