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Figure 1 Looking at a very crude model of the drug discovery and development process,we can map on where omic technologies are currently having an impact.Creation of registries and with biobanks provide access to large,more standardised data sets on participants and help identify relevant participants for both research and clinical trials. With the growth of clinical trials collecting omic data,we now also have the possibility of stratifying patients with biomarkers (and ultimately CDx) todefine those most likely to respond to particular therapeutic agents

and also embedded within health services. This research is focused on a better understanding of disease mechanisms, the identification of biomark- ers* of particular diseases or conditions and a characterisation of the patients’ drug responses (both good and bad). Such work, and the increas- ing affordability of genome sequencing, provides an opportunity to develop these biomarkers into CDx to enable drugs to be targeted to the specific patient populations most likely to respond posi- tively to treatment with a specific therapeutic agent (Figure 1) Figure 2 is a schematic that shows how retro-

spective analysis of clinical trial results might strat- ify the participants into different cohorts allowing insight into the genomic profile of those patients who might well respond to a drug therapy and those patients who are unlikely to respond. It should be noted that if such biomarkers had

been identified in the comparatively unregulated research environment, then if these biomarkers were to be used in a clinically applicable CDx, the research work that had been carried out to identify the biomarkers could well need to be repeated and validated under clinical regulatory conditions, in accordance with an appropriate quality system, in order to be eligible for registration as a CDx. An objective of the Pistoia Alliance ‘CDx/NGS

Drug DiscoveryWorld Summer 2019

and Regulation’ CoI was to bring together protag- onists in the research, clinical and regulatory domains relevant to CDx to consider whether stan- dards could be identified and agreed such that if the research analyses were aligned to them, then the data first used to identify the biomarker could form the basis of the regulatory filing of the CDx. Such an approach would have the benefit of min- imising the duplication of effort, saving time and cost in development, and as such getting more effective therapeutics to the marketplace faster, benefitting patients, healthcare providers and the companies providing the therapeutics and CDx. The inaugural workshop in April 2018 brought

together representatives from pharma and biotech, technology companies, clinical scientists and regu- lators to identify the key issues that would need to be addressed. One of the key learnings was that CDx business embraced a broad range of disci- plines such as genomics,NGS, bioinformatics, clin- ical and regulatory affairs and the workshop dele- gates were in large part familiar with some of those disciplines, but by no means all. As such, a first step was to help address these needs and the Pistoia Alliance CoI organised a symposium on the ‘Application of NGS Technologies in Precision Medicine’ in September 2018, and this symposium on the ‘Application of Bioinformatics in support of


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