667FDA and Pharmacogenomics
Pharmacogenomics is an area of development the FDA views very positively. The
FDA has received numerous drug submissions that included pharmacogenomic data
since issuing guidance on how to do so in 2005. The most recent is 2013 guidance
document “Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase
Clinical Studies and Recommendations for Labeling”. This guidance is intended to
assist the pharmaceutical industry and other investigators engaged in new drug
development in evaluating how variations in the human genome, specifi cally DNA
sequence variants, could affect a drug’s pharmacokinetics, pharmacodynamics, effi -
cacy, or safety. It provides recommendations on when and how genomic information
should be considered to address questions arising during drug development and reg-
ulatory review, focusing on general principles of study design, data collection, and
data analysis in early-phase trials. It also provides recommendations for labeling.
Several cancer drugs have been approved that include pharmacogenomic data
for the guidance of physicians prescribing these drugs. The FDA also is exploring
similar guidelines for pharmacoproteomic data.
FDA Guidance for Pharmacogenomic Data Submissions
The updated guide to pharmacogenomic data submission was issued by the FDA in
March 2005. Current information relevant to pharmacogenomics is available on
FDA’s website ( http://www.fda.gov/cber/gdlns/pharmdtasub.htm ). In a press in
2005, the FDA recognized that pharmacogenomics allows health care providers to
identify sources of an individual’s profi le of drug response and predict the best pos-
sible treatment option for this individual. FDA’s efforts in this direction will facili-
tate the development of personalized medicine. FDA’s guidance “Pharmacogenomic
Data Submissions,” clarifi es how pharmacogenomic data will be evaluated. The
fi nal guidance describes what data will be needed during the marketing application
review process, the format for submissions, and the data that will be used during
regulatory decision making. The guidance also explains a new mechanism for
industry to voluntarily submit research data to further the scientifi c exchange of
information as we move into more advanced areas of pharmacogenomic research.
The voluntary data, which will be reviewed by an internal, agency-wide group and
will not be used for regulatory decision making, will help FDA and industry gain
valuable experience as this new fi eld continues to evolve.
FDA believes this approach will save time and resources and eliminate possible
delays in the application review process because parties will be able to familiarize
themselves with novel pharmacogenomic approaches as they evolve.
FDA has already received several pharmacogenomic data submissions through
both the regulatory and voluntary processes and will continue to work closely with
industry and the healthcare community on this exciting emerging technology.
FDA and Personalized Medicine