The promise of precision

Programme stream(s): Treatment
Programme session type(s): Recent Advances session

Chair: Tim Maughan, University of Oxford, UK
Speaker: Cindy Billingham, University of Birmingham, UK
Speaker: Peter O'Dwyer, University of Pennsylvania, USA
Speaker: Richard Wilson, Queen's University Belfast, UK

11:00-12:30

Room: Boisdale

Precision medicine entails the delivery of treatment tailored to the individual based on genetic, clinical and other factors. Biomarker directed trials evaluate the efficacy of new therapeutic approaches in the patients considered most likely to benefit. The NCI Match trial is the most advanced example of this approach. This session will explore the biological underpinning, methodological approaches and the practical challenges of this clinical research strategy.

Methodological issues in precision oncology trials
Speaker: Lucinda Billingham
Affiliation: Cancer Research UK Clinical Trials Unit, University of Birmingham

Abstract:

In striving for precision oncology, master protocols provide an efficient framework in which to investigate the efficacy and safety of drugs for multiple biomarker-defined populations. Such protocols can take the form of an umbrella or basket design. Experience in the design and implementation of master protocols allows the challenges of such trials to become apparent. The National Lung Matrix Trial (NLMT) is a flagship trial in the United Kingdom being the first to combine the development of a technology platform that screens for multiple genetic aberrations in tumours (provided by the Cancer Research UK Stratified Medicine Programme 2) with testing of multiple novel genetic-marker-directed drugs. The trial is a phase II umbrella-basket trial in patients with advanced non-small cell lung cancer testing 7 different drugs targeting 20 molecular markers. In addition, patients with no actionable genetic change (NA) are included and treated with a sequential pipeline of drugs. The trial opened to recruitment in March 2015 and is ongoing. Challenges include: screening patients within standard NHS practice and translating them into trial patients; choosing a statistical design that is flexible and fit-for-purpose; enabling conclusions from very rare molecularly-defined cohorts alongside the more prevalent ones; adding new drug-biomarker cohorts into the trial; reporting trial results from drug-biomarker cohorts with varying recruitment rates.