Pharmacogenetics and pharmacogenomics in drug development and regulatory decision making: report of the first FDA-PWG-PhRMA-DruSafe Workshop.
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.
Food and Drug Administration, Center for Drug Evaluation and Research, Rockville, Maryland, USA.
Lawrence J
LJ
Lesko
Ronald A
RA
Salerno
Brian B
BB
Spear
Donald C
DC
Anderson
Timothy
T
Anderson
Celia
C
Brazell
Jerry
J
Collins
Andrew
A
Dorner
David
D
Essayan
Baltazar
B
Gomez-Mancilla
Joseph
J
Hackett
Shiew-Mei
SM
Huang
Susan
S
Ide
Joanne
J
Killinger
John
J
Leighton
Elizabeth
E
Mansfield
Robert
R
Meyer
Stephen G
SG
Ryan
Virginia
V
Schmith
Peter
P
Shaw
Frank
F
Sistare
Mark
M
Watson
Alexandra
A
Worobec
D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards; D003657:Decision Making; D015195:Drug Design; D023281:Genomics / Q000331:legislation & jurisprudence* / Q000379:methods* / Q000592:standards; D017408:Guidelines as Topic; D006801:Humans; D010597:Pharmacogenetics / Q000331:legislation & jurisprudence* / Q000379:methods* / Q000592:standards; D010600:Pharmacology; D011050:Policy Making; D012107:Research Design / Q000592:standards / Q000639:trends; D012449:Safety / Q000592:standards; D014481:United States; D014486:United States Food and Drug Administration / Q000592:standards