Abstract
By the time a new drug moves into the confirmatory stage, its developer should in theory have a reasonable amount of information on the effect of the drug on several efficacy endpoints. Based on this assumption, we begin this chapter by assuming that some treatment effect information on the primary endpoint and for the appropriate patient population is available for planning a Phase 3 trial. We review how this information can be used to assess the probability of success of the study (POSS) and discuss how POSS can in turn help a sponsor assess the adequacy of the sample size calculated from the hypothesis testing perspective. In addition, we discuss factors that could affect POSS and how these factors should be incorporated into the planning of the confirmatory program. The chapter highlights the importance of a robust investment in Phase 2 development in order to achieve a desirable level of POSS at the Phase 3 stage.
Chance favors the prepared mind.
—Louis Pasteur
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Chuang-Stein, C., Kirby, S. (2017). Designing Confirmatory Trials with Desired Characteristics. In: Quantitative Decisions in Drug Development. Springer Series in Pharmaceutical Statistics. Springer, Cham. https://doi.org/10.1007/978-3-319-46076-5_9
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DOI: https://doi.org/10.1007/978-3-319-46076-5_9
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