Lesko Lawrence J, Woodcock Janet
Office of Clinical Pharmacology and Biopharmaceutics, Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, Maryland 20857, USA.
Nat Rev Drug Discov. 2004 Sep;3(9):763-9. doi: 10.1038/nrd1499.
Pharmacogenomics and pharmacogenetics provide methodologies that can lead to DNA-based tests to improve drug selection, identify optimal dosing, maximize drug efficacy or minimize the risk of toxicity. Rapid advances in basic research have identified many opportunities for the development of 'personalized' treatments for individuals and/or subsets of patients defined by genetic and/or genomic tests. However, the integration of these tests into routine clinical practice remains a major multidisciplinary challenge, and even for well-established biomarkers there has been little progress. Here, we consider this challenge from a regulatory perspective, highlighting recent initiatives from the FDA that aim to facilitate the integration of pharmacogenetics and pharmacogenomics into drug development and clinical practice.
药物基因组学和药物遗传学提供了一些方法，这些方法可带来基于DNA的检测，以改善药物选择、确定最佳剂量、使药物疗效最大化或使毒性风险最小化。基础研究的迅速发展为针对由基因和/或基因组检测定义的个体和/或患者亚群开发“个性化”治疗创造了许多机会。然而，将这些检测整合到常规临床实践中仍然是一项重大的多学科挑战，即使对于已确立的生物标志物，进展也甚微。在此，我们从监管角度审视这一挑战，重点介绍美国食品药品监督管理局（FDA）近期旨在促进药物遗传学和药物基因组学融入药物研发及临床实践的举措。
