Conditional assurance: the answer to the questions that should be asked within drug development.

ABSTRACT

In this paper, we extend the use of assurance for a single study to explore how meeting a study's pre-defined success criteria could update our beliefs about the true treatment effect and impact the assurance of subsequent studies. This concept of conditional assurance, the assurance of a subsequent study conditional on success in an initial study, can be used assess the de-risking potential of the study requiring immediate investment, to ensure it provides value within the overall development plan. If the planned study does not discharge sufficient later phase risk, alternative designs and/or success criteria should be explored. By transparently laying out the different design options and the risks associated, this allows for decision makers to make quantitative investment choices based on their risk tolerance levels and potential return on investment. This paper lays out the derivation of conditional assurance, discusses how changing the design of a planned study will impact the conditional assurance of a future study, as well as presenting a simple illustrative example of how this methodology could be used to transparently compare development plans to aid decision making within an organisation.