IDEAS · Improving Design, Evaluation and Analysis of early drug development Studies

Improving Design, Evaluation and Analysis of early drug development Studies

Background Drug development is a long and costly process which suffers frequently failure that is often only determined during the final stage. It has been recognised that more care needs to be taken during the early stages of development to avoid lengthy and costly confirmatory studies with ineffective or harmful treatments. Furthermore, the number of new drugs approved has decreased drastically in the last 50 years. Early drug development is crucial to identify promising treatments that have the potential to transform patient's lives. To achieve this goal it is essential to implement efficient methods for the design and analysis of such early development studies. The expertise in this area is, however, limited and adequate methodology is only partially available. The main goals of the IDEAS network were to (a) train early-stage researchers in state of the art methods for designing, evaluating and analysing early phase studies; (b) develop novel methodology for early phase studies; (c) provide an international, collaborative environment; (d) raise awareness about cutting edge methods. Scientific achievements The network has successfully trained 14 early stage researchers in methods for early drug development with 11 having completed a doctoral degree and the remaining three on track to complete a doctorate in the next months. The research undertaken has led to novel methods for pre-clinical studies as well as early clinical trials. In the area of dose-finding, novel methods have been developed that allow pre-clinical data to be formally incorporated, methods for dose-schedule trials have been developed and novel approaches for dose-combination studies have been devised. Moreover, IDEAS has developed methodology to advance precision medicine. Novel designs accounting for subgroups in both Phase I and Phase II studies have been created, visualisation techniques explored and approaches to identify biomarkers developed. Additionally, novel statistical methods for biosimilar development have been proposed, advanced pharmacological models developed and estimation problems after adaptive designs have been explored. Dissemination The findings been published in high impact methods journals as well as applied journals and the novel methodology is already being started to be used in real studies which has been facilitated through the provision of open source software, training of end-users via bespoke publications and active engagement in studies. The network is now embarking on establishing a dedicated training route for quantitative drug developers. Outreach IDEAS has regularly engaged in outreach activities such as community days, a blog series and writing popular science articles. Additionally, the main research results were presented in short YouTube videos using animations and explaining the relevance of the methodological research in lay terms. To date, the videos have been viewed over 4,000 times. With over 200 different activities, we have reached over 66,000 people.

Data: CORDIS, © European Union