Pharmacogenetics and Pharmacogenomics in Drug Development and Regulatory Decision Making: Report of the First FDA-PWG-PhRMA-DruSafe Workshop

• Published in: Journal of clinical pharmacology Vol. 43; no. 4; pp. 342 - 358
• Main Authors: Lesko, Lawrence J., Salerno, Ronald A., Spear, Brian B., Anderson, Donald C., Anderson, Timothy, Brazell, Celia, Collins, Jerry, Dorner, Andrew, Essayan, David, Gomez-Mancilla, Baltazar, Hackett, Joseph, Huang, Shiew-Mei, Ide, Susan, Killinger, Joanne, Leighton, John, Mansfield, Elizabeth, Meyer, Robert, Ryan, Stephen G., Schmith, Virginia, Shaw, Peter, Sistare, Frank, Watson, Mark, Worobec, Alexandra
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01-04-2003; SAGE Publications; Sage Science; Wiley Subscription Services, Inc
• Subjects: Biological and medical sciences; Clinical trial. Drug monitoring; Clinical Trials as Topic - methods; Clinical Trials as Topic - standards; Decision Making; Drug Design; drug development process; General pharmacology; Genomics - legislation & jurisprudence; Genomics - methods; Genomics - standards; Guidelines as Topic; Humans; Medical sciences; Pharmacogenetics; Pharmacogenetics - legislation & jurisprudence; Pharmacogenetics - methods; Pharmacogenetics - standards; pharmacogenomics; Pharmacology; Pharmacology. Drug treatments; Policy Making; regulatory agencies; Research Design - standards; Research Design - trends; safe harbor; Safety - standards; United States; United States Food and Drug Administration - standards; United States; Drug; Evaluation; Pharmacogenetics; Toxicity; Legislation; Pharmacology; Review; Research and development; Recommendation; Genetics; Workshop; Safety; Pharmaceutical industry; Application; Food and Drug Administration
• ISSN: 0091-2700; 1552-4604
• DOI: 10.1177/0091270003252244

The use of pharmacogenetics and pharmacogenomics in the drug development process, and in the assessment of such data submitted to regulatory agencies by industry, has generated significant enthusiasm as well as important reservations within the scientific and medical communities. This situation has arisen because of the increasing number of exploratory and confirmatory investigations into variations in RNA expression patterns and DNA sequences being conducted in the preclinical and clinical phases of drug development, and the uncertainty surrounding the acceptance of these data by regulatory agencies. This report summarizes the outcome of a workshop cosponsored by the Food and Drug Administration (FDA), the Pharmacogenetics Working Group (PWG), the Pharmaceutical Research and Manufacturers of America (PhRMA), and the PhRMA Preclinical Safety Committee (DruSafe). The specific aim of the workshop was to identify key issues associated with the application of pharmacogenetics and pharmacogenomics, including the feasibility of a regulatory “safe harbor” for exploratory genome‐based data, and to provide a forum for industry‐regulatory agency dialogue on these important issues.