Personalized medicine and rescuing "unsafe" drugs with pharmacogenomics: a regulatory perspective

The sequencing of the human genome and the revolution it has caused in biomedical science created hope for a new era in the prevention and treatment of serious illnesses. In the area of drug development, much of this hope is focused in the field of pharmacogenomics (PGx), which is the study of how individual genetic differences affect drug response. Many people expected advances in pharmacogenomics to lead to the rapid development of new "personalized medicines," where drugs and dosages could be tailored specifically to a patient's genotype. However, pharmacogenomics has largely failed to meet these expectations and the Food and Drug Administration has only approved a handful of drugs that rely on PGx data. This article evaluates how FDA regulates the use of pharmacogenomics and discusses how the current regulatory scheme fails to provide an adequate route for developing personalized medicine. The article then proposes modifying the current regulatory regime to encourage development of PGx-based drugs by either allowing PGx-based drugs to be approved with unvalidated biomarkers if the sponsor commits to Phase IV studies or using the Orphan Drug Act to provide economic incentives.