The advent of the genomic era generated great expectations for drug discovery and development, said Geoffrey Ginsburg of Duke University. Genomic information was expected to provide insights into the underlying biological mechanisms of disease and to highlight biological targets and pathways that would be amenable to new drug discovery. It indicated an approaching ability to stratify populations based on genomic-based biomarkers, leading to better clinical development programs. Genomic data would reveal how individuals might respond to, be resistant to, or have adverse effects from a drug, creating the potential for personalized medicines. As a result of these and other changes, genomic data would increase the efficiency of drug discovery and development, increase the success rate of new drugs, enhance safety, and decrease costs.

The genomic era has made major strides toward delivering on these promises, Ginsburg said. Several genomics-enabled products have been approved in recent years or are in development for use, including three that are described in Chapter 3 of this report: crizotinib for the treatment of non-small-cell lung cancer, pomaglumetad methionil for schizophrenia, and ivacaftor for cystic fibrosis. In addition, academic–industry partnerships have formed to leverage a deep understanding of disease biology from the academic realm and to meld that to product development and commercialization in industry. Precompetitive collaborations, such as the European Innovative Medicines Initiative and programs sponsored by the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH), have sought to lay the groundwork for new therapeutics.


Nicholas Davies from PricewaterhouseCoopers (PwC) elaborated on the potential and current use of genomic-based drug discovery and development during his presentation in the workshop’s initial session. The efficiency and quality of research inputs have undergone huge improvements. The cost of DNA sequencing has dropped by many orders of magnitude and continues to drop. The ability to find targets, screen compounds, and generate chemical libraries is immense. The development of companion diagnostics has made it possible to target patient subpopulations that would be expected to benefit from a specific treatment. As Mark Trusheim from the Sloan School of Management at the Massachusetts Institute of Technology added, in this way patients and providers have more and better treatment options, regulators gain a better sense of risk-benefit comparisons, drug and diagnostic innovators generate more products and profits, and

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