ABSTRACT
The preceding development of the efficiency of a Phase 2/3 clinical program did not address the use of a single-arm Phase 2 trial, that is, a Phase 2 trial without a control arm. The concern with using a single arm Phase 2 trial in a quantitative approach to identifying active drugs is that it is susceptible to selection bias, which can make the study results uninterpretable. In particular, selection bias can make a drug that is no different than a placebo appear to have promising activity. Here we present an approach to using Phase 2 single-arm trials that account for potential selection bias.
