Abstract
The use of pharmacogenomic information has opened new opportunities in drug discovery and development. The FDA has undertaken several initiatives to promote and exchange ideas in the field of pharmacogenomics as a key opportunity for the critical path. In this chapter, a regulatory science perspective of pharmacoge-nomics is discussed addressing the critical aspects of pharmacogenomics in drug development, drug therapy, regulatory decision making, and labeling implications.
The mission of the U.S. Food and Drug Administration (FDA) is to protect and advance the public health, and encourage scientific innovations to develop safe and effective drugs (1). However, the FDA's efforts to achieve this critical mission can be challenging because of rapidly emerging science and technology. To bridge the potential gap between scientific innovations and translating them into clinical use, the FDA has undertaken several initiatives to promote and exchange ideas and information in the field of pharmacogenomics. The FDA's “personalized medicine” initiatives make use of pharmacogenomics—the science that predicts a response to drugs based on a patient's genetic makeup.
Individualized therapy based on pharmacogenomics could be important in drug development. Pharmacogenomics has the potential to identify sources of interindividual variability in drug responses that affect the efficacy and safety of drugs. In addition, pharmacogenomics/pharmacogenetics can provide a key opportunity to improve the safety outcomes of existing therapies. For example, drugs with a narrow therapeutic range, such as warfarin and irinotecan, can benefit from this emerging science. By integrating the use of pharmacogenomics into routine clinical practice, pharmacogenomics can help to individualize therapy with the intent of maximizing effectiveness and minimizing risk (i.e., improve the benefit/risk ratio) (2).
In this chapter, a regulatory science perspective on pharmacogenomics will be discussed to address the critical aspects of pharmacogenomics in drug development, drug therapy, and regulatory decision making.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
U.S. FDA's Mission Statement. http://www.fda.gov/opacom/morechoices/mission.html. Accessed on July 25, 2007
U.S. FDA. Guidance for industry: pharmacogenomic data submissions. http://www.fda.gov/cder/guidance/6400fnl.pdf. Accessed on July 25, 2007
Woodcock J (2007) The prospects for “personalized medicine” in drug development and drug therapy. Clin Pharmacol Ther 81:164–169
FDA. Innovation or stagnation: challenge and opportunity on the critical path to new medical products. http://www.fda.gov/oc/initiatives/criticalpath/whitepaper.html. Accessed on July 25, 2007.
Lesko LJ (2007) Personalized medicine: elusive dream or imminent reality? Clin Pharmacol Ther 81:807–816
Lesko LJ, Woodcock J (2004) Translation of pharmacogenomics and pharmacogenetics: a regulatory perspective. Nat Rev Drug Discov 3:763–769
Lesko LJ, Woodcock J (2002) Pharmacogenomic-guided drug development: regulatory perspective. Pharmacogenomics J 2:20–24
Lesko LJ, Salerno RA, Spear BB, Anderson DC, Anderson T, Brazell C, Collins J, Dorner A, Essayan D, Gomez-Mancilla B, Hackett J, Huang SM, Ide S, Killinger J, Leighton J, Mansfield E, Meyer R, Ryan SG, Schmith V, Shaw P, Sistare F, Watson M, Worobec A (2003) Pharmacogenetics and pharmacogenomics in drug development and regulatory decision making: report of the first FDA-PWG-PhRMA-DruSafe workshop. J Clin Pharmacol 43: 342–358.
Ruaño G, Collins JM, Dorner AJ, Wang SJ, Guerciolini R, Huang SM (2004) Pharmaco-genomic data submissions to FDA: clinical pharmacology case studies. Pharmacogenomics 5:513–517.
Salerno RA, Lesko LJ (2006) Three years of promise, proposals, and progress on optimizing the benefit/risk of medicines: a commentary on the 3rd FDA-DIA-PWG-PhRMA-BIO pharmacogenomics workshop. Pharmacogenomics J 6:78–81
Huang SM, Goodsaid F, Rahman A, Frueh F, Lesko LJ (2006) Application of pharmacogenomics in clinical pharmacology. Toxicol Mech Methods 16:89–99
U.S. FDA. Physician labeling, new content and format requirements: FR notice 2006. http://www.fda.gov/cder/regulatory/physLabel/default.htm. Accessed on July 25, 2007
U.S. FDA. Genomics at FDA, table of valid genomic biomarkers in the context of approved drug labels. http://www.fda.gov/cder/genomics/genomic_biomarkers_table.htm. Accessed on July 25, 2007
Imuran product label. http://www.fda.gov/cder/foi/label/2005/016324s030,017391s013lbl.pdf. Accessed on July 25, 2007
Purinethol product label. http://www.fda.gov/cder/foi/label/2004/09053s024lbl.pdf. Accessed on July 25, 2007
U.S. FDA. Pediatric Subcommittee of the Oncology Drug Advisory Committee, 6-MP. http://www.fda.gov/OHRMS/DOCKETS/AC/03/transcripts/3971T1.pdf. Accessed on July 25, 2007
Camptosar product label. http://www.fda.gov/cder/foi/label/2006/020571s030lbl.pdf. Accessed on July 25, 2007
U.S. FDA. Clinical Pharmacology Subcomitte of the Advisory Committe for Pharmaceutical Science. Camptosar. 2004. http://www.fda.gov/ohrms/dockets/ac/04/briefing/2004-4079b1.htm. Accessed on July 25, 2007
Stratteraproductlabel.http://www.fda.gov/cder/foi/label/2007/021411s004s012s013s015s021lbl.pdf. Accessed on July 25, 2007
Coumadinproductlabel.http://www.fda.gov/cder/foi/label/2006/009218s102lbl.pdf. Accessed on July 25, 2007
Wysowski DK, Nourjah P, Swartz L (2007) Bleeding complications with warfarin use: a prevalent adverse effect resulting in regulatory action. Arch Intern Med 167:1414–1419
U.S. FDA. Clinical Pharmacology Subcommittee of the Advisory Committee for Pharmaceutical Science. Warfarin. 2005 http://www.fda.gov/ohrms/dockets/ac/cder05.html#Pharm Science. Accessed on July 25, 2007
Herceptin product label. http://www.fda.gov/cder/biologics/products/trasgen092598.htm. Accessed on July 25, 2007
Tarceva product label. http://www.fda.gov/cder/foi/label/2004/021743lbl.pdf. Accessed on July 25, 2007
Frueh FW, Goodsaid F, Rudman A, Huang SM, Lesko LJ (2005) The need for education in pharmacogenomics: a regulatory perspective. Pharmacogenomics J 5:218–220
Andersson T, Flockhart DA, Goldstein DB, Huang SM, Kroetz DL, Milos PM, Ratalin MJ, Thummel K (2005) Drug-metabolizing enzymes: evidence for clinical utility of pharmacogenomic test. Clin Pharmacol Ther 78:559–581
Huang SM, Lesko LJ (2005) Application of pharmacogenomics in clinical pharmacology. In: Knablein J and Muller RH, ed. Molecular Medicine, Correlation between genes, diseases and biopharmaceuticals, in ”Modern Biopharmaceuticals-Design, Development and Optimization, vol. 1. VCH: Wiley, 49–70
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2008 Humana Press, a part of Springer Science+Business Media, LLC
About this protocol
Cite this protocol
Kim, MJ., Huang, SM., Rahman, A., Frueh, F.W., Lesko, L.J. (2008). Pharmacogenomics. In: Cohen, N. (eds) Pharmacogenomics and Personalized Medicine. Methods in Pharmacology and Toxicology. Humana Press. https://doi.org/10.1007/978-1-59745-439-1_3
Download citation
DOI: https://doi.org/10.1007/978-1-59745-439-1_3
Publisher Name: Humana Press
Print ISBN: 978-1-934115-04-6
Online ISBN: 978-1-59745-439-1
eBook Packages: Springer Protocols