It is well established that the Phase 3 clinical trial failure rate is still very high in many therapeutic areas. As a result, both financial and intellectual resources that could have been applied to other therapies or experiments may often result in minimal return. One driver of this paradigm is that major investment or strategy decisions are often made in the presence of less robust early/mid-stage data containing considerable uncertainty while trying to address multiple important research questions. Statisticians play an important role in helping to quantify decision making and risk from either a development or a regulatory perspective.

In this roundtable discussion, industry, academic and FDA researchers are welcomed to share perspectives and examples related to decision making in early stage development. A few example topics for discussion: 1) how to frame a prospectively defined robust and statistically grounded Go/No-Go (interim or final) decision making; 2) what are the key success factors related to decision making during early stage development; 3) what are the novel methods or ideas for optimizing Phase 2/3 seamless designs.