Role of pharmacogenomics in drug development

Abstract

AbstractPharmacogenomics is charged with the task of uncovering genetic variations that underlie responses to drugs. While the results in the field have been slow in coming, more recent high‐throughput screening methods and data‐mining approaches are expected to effectively expedite the drug development process in the near future. While these new techniques are likely to reduce costs, the selection of the most pertinent study cohort with respect to the biological mechanism of the study compound is a critical element in risk management. Enrichment of the study cohort with carriers of “at‐risk” variants in genes that reside within and/or influence the biological pathway targeted by the drug candidate, would be expected to lower the risk of drug failure, while both delivering faster and better results at lower costs. In this context, at deCODE Genetics, Inc., the powerful coupling of family‐based linkage to ultra‐high throughput genotyping, gene array, and proteomics technology, together with innovative bioinformatic resources, provides a focused integrative strategy for pinpointing disease‐causing genes that may generate validated drug targets and genes that are responsible for differential response to drugs. deCODE has compiled one of the world's most comprehensive collection of population data on genealogy, genotypes, and phenotypes. This combination of resources provides an effective system for uncovering genes that predispose to disease or regulate drug response. In addition, this approach allows for the development of novel therapeutic strategies and diagnostic tests that, apart from being applicable to assessment of disease susceptibility and clinical response to drugs, provide for an effective clinical trial design, ultimately leading to safer and more efficacious drugs for patients. Drug Dev. Res. 62:86–96, 2004. © 2004 Wiley‐Liss, Inc.