Optimized adaptive enrichment designs.

ABSTRACT: Based on a Bayesian decision theoretic approach, we optimize frequentist single- and adaptive two-stage trial designs for the development of targeted therapies, where in addition to an overall population, a pre-defined subgroup is investigated. In such settings, the losses and gains of decisions can be quantified by utility functions that account for the preferences of different stakeholders. In particular, we optimize expected utilities from the perspectives both of a commercial sponsor, maximizing the net present value, and also of the society, maximizing cost-adjusted expected health benefits of a new treatment for a specific population. We consider single-stage and adaptive two-stage designs with partial enrichment, where the proportion of patients recruited from the subgroup is a desi

SUBMITTER: Ondra T 

PROVIDER: S-EPMC6613177 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature