Clinical trials are considered the gold-standard study design when testing for a causal effect of a new treatment or intervention. There are, however, many challenges to be faced in the future. The high, and increasing, cost of clinical trials has caused the drug development process to become more and more expensive (DiMasi et al., 2016), meaning new design and analysis methods to increase efficiency are greatly needed. The barriers to using adaptive clinical trials are lifting, which is leading to an increasing uptake (Hatfield et al., 2016). Members of the BSU are working on several current and future trials that are implementing novel methods. This portfolio of trials forms a strong basis for our future methodology research plans. We also have the capacity, skills and track-record to push the methodology forward to tackle even more ambitious problems. These include dealing with patient heterogeneity captured by multiple ’omics platforms, exploiting multivariate outcomes in dose-finding/ranging trials, using historical data, patient preference designs, applying bandit methodology to improve patient benefit in trials, and conducting trials within large cohorts.

Members of the DART theme have been active participants in the MRC HTMR Network, in particular, the adaptive designs and stratified medicine working groups. As an example of an interaction, we are currently involved with writing several papers to help clinical teams implement adaptive designs, and to give guidance on dose-escalation studies. A future collaboration is the Adaptive designs Consolidated standards of reporting trials Extension (ACE) group which is developing guidance for reporting of adaptive trials.

Currently there are many clinical trials units in the UK but few groups in the UK actively research novel designs. Our strengths lie in the development of novel trial designs and analysis within the areas of early phase experimental studies and biomarker stratified designs. Due to being based in a centre of excellence for biostatistics research, we are uniquely placed to collaborate with experts in stratified medicine, evidence synthesis and genomics research: all areas of importance in designing and analysing novel trials. As clinical trials units often lack the capacity for methodological knowledge of novel trials, we have several collaborations to help CTUs run adaptive and other complex trials. We particularly focus on support of local CTUs, which has led to joint positions with both the Cambridge CTU and Papworth Hospital’s CTU. Additionally we have been successful in building links with industry with several ongoing collaborations.

Other Research Themes:

• SURPH: Statistical methods Using data Resources to improve Population Health
• SOMX: Statistical Omics
• PREM: Precision Medicine and Inference for Complex Outcomes