Modern advances in the basic biomedical sciences are creating opportunities to support personalized medicine through the development of targeted therapies tailored to the biological characteristics of individual patients. The evolution of personalized medicine is dependent upon the development and application of clinically valid biomarkers, as well as companion diagnostics capable of accurately identifying and measuring a particular biomarker of interest. However, ongoing challenges in the discovery, validation, and clinical application of biomarkers present significant barriers to progress. As part of its mandate under the Prescription Drug User Fee Act Reauthorization of 2012, the U.S. Food and Drug Administration (FDA) is committed to advancing the development and use of biomarkers in clinical trials by enhancing its regulatory review and communication processes.
On September 5, the Engelberg Center for Health Care Reform hosted a public workshop to help advance biomarker science in the context of therapeutic product development. Discussions identified challenges for biomarker applications in early and late phase clinical trials, as well as best practices for successful biomarker-based programs (including the co-development of in-vitro diagnostic devices and the use of biomarkers as outcome measures in clinical trials). This meeting was convened by Brookings under a cooperative agreement with FDA.