The Methodological Innovation theme aims to expand and disseminate CTSU’s innovations in the efficient design and conduct and rigorous analysis and interpretation of large-scale randomised trials and observational studies. Furthermore, through broad engagement with those who design, fund, regulate and participate in clinical studies, this work seeks to reduce the bureaucratic regulatory obstacles to such research.

Previous work

CTSU has had an international impact on clinical trial strategies by pioneering, and continuing to conduct, large streamlined “mega-trials” and individual-patient-data “meta-analyses”, in collaboration with thousands of investigators worldwide, which have transformed patient care. Similarly our work on large-scale observational studies (including UK Biobank) and meta-analyses of such studies has demonstrated how recruitment, phenotyping and long-term follow-up of hundreds of thousands of individuals is necessary and can be achieved cost-effectively. CTSU’s work has produced profound effects on regulatory and governance activities related to research (e.g. it led to the complete revision of FDA and MHRA requirements for trial monitoring and to the introduction of the “quality by design” concept).

Current and future developments

Advances in translational research, and consequent improvements in health, are critically dependent on reliable evidence from high quality clinical studies. However, there is widespread concern that cumbersome study design and burdensome research regulation have made such studies substantially more difficult and expensive to conduct. For example, the emerging field of “Big Data” offers the potential for significant improvements in scale (breadth, depth and duration) and efficiency (data accumulation, storage, processing and dissemination) of large-scale clinical research. However, in order to maximise the returns from these new opportunities, it will be important to develop and evaluate methods focused on the key drivers of study quality in large-scale studies, and to ensure that their use is not encumbered by disproportionate regulatory or bureaucratic obstacles.

Our work on the use of health records systems for recruitment, phenotyping and outcome ascertainment in such studies, and tackling obstacles to research due to disproportionate regulation, plays a central role in Oxford’s Big Data Institute.

Organisation and Funding

The work conducted by CTSU is funded by Medical Research Council, British Heart Foundation, and Cancer Research UK. Further details are provided in the relevant project pages.