This report describes the scientific, industrial, regulatory, and health care management system context in which biomarkers are being developed. It identifies some of the barriers which may impede biomarker research, discovery, development, commercialisation and, ultimately, uptake in clinics. It also focuses on the use of biomarkers in the health care system, as diagnostics and in medical tests, and explores the use of biomarkers for the development of improved medicines. It does not address the role of biomarkers upstream, as tools for basic research.
Biomarkers allow new ways of understanding disease processes and the ways in which medicines work to counteract disease. Within the practice of evidence-based medicine this knowledge can be used to improve disease diagnosis, to improve the safety and efficacy of existing medicines and to develop new medicines and targeted therapies. Novel molecular biomarkers have the potential to transform much of the current health care model, shifting the focus from a reactive ‘one-size-fits-all’ system to one that is more proactive and precise. In this new, proactive approach, disease or disease susceptibility may be diagnosed earlier, and disease may be controlled or possibly prevented before it starts; and when disease is detected, new biomarker-based diagnostics may be used to develop treatment strategies which are tailored to the characteristics of individual patients. Over the long term, the use of biomarkers may improve patient welfare by delivering better health outcomes.