Edinburgh Trials Methodology Hub

When a new drug is being developed it passes through several stages of development. After the initial preclinical development, first in man studies evaluate the safety and potential side effects. Then explanatory studies optimise the treatment, for example in terms of which patients have the greatest potential to benefit and what dose to use. Such studies look at underlying disease mechanisms, and test whether an intervention has the potential to be useful in practice. Then pragmatic studies test whether any potential benefit seen in the explanatory trials can be realised in clinical practice. For non-drug treatments the terminology is different, but the development path is similar.

There are many reasons why an intervention can fail as it progresses through the stages of development, and the aim of the Edinburgh Trials Methodology Hub is to undertake a programme of research which will help to increase the chances of success at each step of the development process. We shall attempt to learn lessons both from successful development programmes and also from others which have failed.

This will be achieved by encouraging interdisciplinary cllaboration between the scientists who are working on understanding underlying disease mechanisms and those with experience in clinical trials methodology. The end results will be to improve the design and analysis of future clinical trials, and to provide researchers with trials support and access to genetic and imaging technologies to identify patient subgroups with the greatest potential to benefit within ongoing trials.

Our vision is to deliver more effective translation of novel scientific ideas and discoveries (both drug and non-drug) into innovative clinical practice. A major challenge in clinical medicine is to identify therapeutic interventions that can be targeted to the correct patient at the right time to substantially improve disease outcomes. Traditionally, clinical trial design has focussed on delivering a standardised intervention on a large scale to patients with a specific clinical presentation which may actually have several different underlying causes, in terms of pathophysiology. This approach has been successful to an extent, but often these interventions have modest effects for the individual patient. Ideally therefore, one would wish to develop biomarkers which can be used in clinical practice to predict which patients will have the greatest response to an intervention, whilst excluding those who are likely to respond poorly or not at all. The vision of the Edinburgh Clinical Trials Methodology Hub will be to develop technologies to achieve this aim, by exploiting knowledge gained from animal models; by developing grid based networks for image analysis and processing; by exploiting access to genomic technologies and by developing a facility for secondary analysis of existing data to develop and validate prognostic models. The end result will be to improve the design of future clinical trials and to provide researchers access to imaging and genetic technologies to identify patient subgroups who respond best within ongoing trials.

In parallel with the planned methodological developments, the Edinburgh Hub will develop a training programme which combines elements of existing programmes in translational medicine, molecular medicine and public health sciences to develop the next generation of trialists and methodologists.