Chapter 19 - Genomics, New Drug Development, and Precision Medicines

Abstract

The last decade has seen a large increase in the amount of genomics data generated with increased expectations of how improved understanding of disease will lead to the development of more effective therapies and personalized medicines. Despite the regular reports of novel genes being identified in a range of disorders, the much-heralded promise of the genomic revolution has yet to be delivered. However, it is important to realize that this does not represent a failure of the science to deliver, because there are multiple clear examples of the predictability and clinical utility of the new genetics and genomics in developing new targeted drugs. To large extent it is a reflection of the length of time it takes to develop new drugs. The 1980s and 1990s saw a boom time for the pharmaceutical industry, producing many highly effective new classes of drugs, from statins to proton-pump inhibitors and quinolone antibiotics. All were novel therapeutic approaches offering significant benefit to individuals and society. The average 10- to 15-year time lag from gaining scientific knowledge to developing therapies is typical for the pharmaceutical industry, and reflects the complexity of drug discovery and the time required for preclinical and clinical testing to ensure safety and efficacy. This chapter will introduce the major concepts of new drug discovery and development, and give a broad overview of how genetics and genomics is used across the whole drug discovery and development pipeline, from pretarget identification to postmarketing surveillance to help discover and develop improved medicines.