Futility Interim Analyses - A Plea for Simplicity

Abstract

Futility interim analyses remain a topic of anxiety for most clinical drug development teams. While they can significantly reduce the risk involved in the development of innovative therapies and do so at a minimal cost, they are not used frequently enough. Development teams often shy away due to the fear of terminating a potentially successful therapy by mistake. We believe these risks are overestimated and hence necessitate a simple and effective communication to be better understood. The planning of futilities often evolves into complex statistical modeling exercises where various scenarios are simulated and multiple risk metrics are calculated. To assist teams in such discussions we propose a straightforward strategy for establishing a range for the futility threshold and an intuitive futility risk measure to evaluate the associated risks.

Keywords: Clinical drug development; Futility interim analysis; Futility risk; Operating characteristics; Retrospective futility analysis.