Program Overview

Precision Medicine Clinical 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 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. However, a barrier to realising the promise of precision medicine is the inappropriate use of traditional clinical trial design and analysis, which rely on estimates of population-averaged effects.

Course Overview

In this course, we introduce the concept of precision medicine and cover innovative approaches. This includes novel designs (biomarker-guided, basket, umbrella, adaptive signature, adaptive enrichment designs and Sequential Multiple Assignment Randomized Trials) as well as efficient analysis approaches (e.g. Bayesian hierarchical modelling, identifying patient subgroups using high-dimensional genetic data) to help estimate sub-group specific treatment effects. These approaches have all been developed to improve the power, quality of information, and patient benefit provided by clinical trials. Examples from a wide variety of therapeutic areas will be discussed, with implementation in R software. Perspectives will be given on the future development of design, conduct and analysis of clinical trials in the field.

Course Topics

• Novel designs:
  • Biomarker-guided designs
  • Basket designs
  • Umbrella designs
  • Adaptive signature designs
  • Adaptive enrichment designs
  • Sequential Multiple Assignment Randomized Trials
• Efficient analysis approaches:
  • Bayesian hierarchical modelling
  • Identifying patient subgroups using high-dimensional genetic data
• Implementation in R software
• Perspectives on the future development of design, conduct and analysis of clinical trials in the field

University Information

Newcastle University
Newcastle upon Tyne
NE1 7RU

Other Relevant Courses

• Leveraging External Information in Clinical Trials
• Adaptive Designs and Multiple Testing Procedures