Anthony Akkari

The primary goal of the pharmaceutical industry is to develop safe and effective medications. As the industry matures and the existing arsenal of marketed therapeutics grows, novel drugs must exhibit greater efficacy and safety to achieve registration and favorable reimbursement. Furthermore, gaining market-share has become extremely competitive, in terms of both meaningful clinical effects and tolerated safety profiles. As a result, the pharmaceutical industry has experienced a steady decline in productivity in recent decades. However, the achievement of regulatory approvals for targeted therapeutics may reverse this drop in productivity. The convergence of high-throughput genetic analysis technologies and the exponentially expanding biological and genomic knowledgebase have provided many clear examples that genetic variation can affect both disease risk and drug response. Therefore, evaluation of genetic variation in clinical trial populations should be considered essential and routine from the earliest phases of drug development. Pharmacogenetics (PGx) in particular has gained considerable attention from drug developers, regulators and payers over the past decade as a means to achieving safer, efficacious and more cost-effective drugs. While PGx science has great potential to impact positively the success of developing a new medicine, the integration of PGx into the decision making processes of the drug development pipeline has been difficult. The goal of this chapter is to describe the principles and requirements of an efficient and valuable PGx strategy that makes use of every opportunity during the course of developing innovative medicines. This strategy combines a proven methodology with rigorous genetic science to create a "Pipeline Pharmacogenetic Program".