Design and Analysis of Precision Medicine trials


Precision medicine is about going beyond assessing whether a new treatment works on average to predicting which subgroups of patients receive benefit and to what extent.  When the subgroups, often defined by biomarkers, genetic, phenotypic or psychosocial characteristics, are associated with a treatment’s efficacy or toxicity, precision medicine offers substantial advantages to patients, trial sponsors, and the wider healthcare system.

In this course we introduce the concept of precision medicine and cover some innovative trial design and analysis approaches, including basket, umbrella, Bayesian hierarchical modelling, adaptive signature and adaptive enrichment designs. These approaches have all been developed to improve power and patient benefit provided by clinical trials. Examples from a wide variety of therapeutic areas will be discussed, with implementation in OpenBUGS and R software. Perspectives will be given on the future development of design, conduct and analysis of clinical trials in the field.

Presenters: Prof James Wason (Newcastle University) and Dr Haiyan Zheng (MRC Biostatistics Unit)