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The FDA believes that pharmacogenomic testing can be smoothly integrated
into drug development processes. Currently, scientifi c understanding of pharma-
cogenomics is most advanced in the drug metabolism area, and early results are
expected in this fi eld. However, FDA anticipates rapid evolution of additional uses.
For example, it is hoped that pharmacogenomic testing will help identify cancers
that have a high probability of responding to a particular medication or regimen.
Pharmacogenomics may also be used to help track down the cause of certain rare,
serious drug side effects.
The guidance provides specifi c criteria and recommendations on submission of
pharmacogenomic data INDs and NDAs and Biological License Applications
(BLAs). This includes information on what data is needed, and how FDA will or
will not use such data in regulatory decisions. Because there is a need for scientifi c
exchange, the agency is asking for voluntary submissions of research information.
This data will help FDA gain experience as the fi eld evolves. In these cases, FDA
advises sponsors to clearly label voluntary submissions; and the agency advises that
it will not use information from voluntary reports for regulatory decisions. If a
sponsor subsequently develops additional data that meet the criteria for submission
for regulatory purposes, the Agency advises sponsors that such data should be sub-
mitted as explained in the guidance.
Joint Guidelines of the FDA and EU Regulators for Pharmacogenomics
In 2006, the FDA and European drug regulators agreed to a joint procedure that
pharmaceutical companies can follow to voluntarily submit pharmacogenomic data
to both agencies. The document, which can be assessed at the web site of EMEA
( http://www.emea.eu.int/ ), could benefi t pharmaceutical companies interested in
simultaneously selling products in Europe and the US that have pharmacogenomic
components. Specifi cally, the European Medicines Agency and the FDA released a
set of “guiding principles” describing how they will process drug developer’s
requests to jointly meet with both agencies about voluntary genomic data submis-
sion. The guiding principles have a list of defi nitions agreed to by the agencies, and
a fl owchart describing how voluntary submissions would be processed. The FDA’s
Interdisciplinary Pharmacogenomic Review Group and the EMEA’s
Pharmacogenetics Working Party will review the data submission packages.
Pharmacogenomic/Pharmacogenetic Information in Drug Labels
Currently, there are >50 drugs with pharmacogenetic discoveries on their labeling,
which can be accessed at: http://www.accessdata.fda.gov/scripts/cder/drugsatfda. The
FDA wants to see genomic information front and center in a drug’s label. The agency
has released guidelines for the “Clinical Pharmacology Section of Labeling for New
Prescription Drugs, Content and Format”. The format for new drug labels includes a
pharmacogenomics section, and will relocate pertinent genetic information to a box
22 Regulatory Aspects of Personalized Medicine