Novel trial designs in precision medicine

Category: Laboratory Studies

Location: University of Birmingham, Occupational Health Building (Y16), Birmingham, B15 2TT | Date: 3 May 2019



The day will include a talk by Prof James Wason, submitted presentations from attendees and will be followed by discussion of novel trial designs in small groups.

We encourage participants to send details of short presentations they would be willing to share to facilitate discussion.


Speaker: James Wason, Newcastle University and MRC Biostatistics Unit, University of Cambridge
Title: Novel precision medicine trial designs for incorporating patient subgroups prospectively
There is an increasing interest in designing clinical trials to enable precision medicine. Precision (also known as stratified or personalised) medicine is about going beyond whether a treatment works on average to finding which patient groups it works particularly well or poorly for.
The traditional randomised controlled trial can provide good information through subgroup analysis. However by utilising patient subgroups, which are thought to be associated with treatment effect, prospectively in the trial design we may improve the power and patient benefit provided by the trial.
In this talk I will cover a range of novel trial designs that have been proposed to help enable precision medicine. I will cover:

  1. Basket trials, where a treatment is tested in multiple subgroups or related conditions with adaptive borrowing of information possible
  2. Umbrella trials, where multiple treatments are tested within multiple patient subgroups, often with an adaptive design to best match treatments with groups
  3. Adaptive signature design, where a large number of potential biomarkers (e.g. genomic information) can be used to form a signature predicting patients who receive a lot of benefit from a new treatment.

I will aim to give background and some details on these approaches, and lay out future research needed to realise their potential.

Join the NIHR Statistics Group - Subscribe for the latest news, updates and event information.