[Federal Register Volume 76, Number 34 (Friday, February 18, 2011)]
[Notices]
[Pages 9583-9584]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2011-3679]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2011-D-0082]
Draft Guidance for Industry on Clinical Pharmacogenomics:
Premarketing Evaluation in Early Phase Clinical Studies; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
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SUMMARY: The Food and Drug Administration (FDA) is announcing the
availability of a draft guidance for industry entitled ``Clinical
Pharmacogenomics: Premarketing Evaluation in Early Phase Clinical
Studies.'' The draft guidance is intended to assist the pharmaceutical
industry and other investigators engaged in new drug development in
evaluating how variations in the human genome could affect the clinical
pharmacology properties and clinical responses of drugs.
DATES: Although you can comment on any guidance at any time (see 21 CFR
10.115(g)(5)), to ensure that the Agency considers your comment on this
draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance by April 19, 2011.
ADDRESSES: Submit written requests for single copies of the draft
guidance to the Division of Drug Information, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, rm. 2201, Silver Spring, MD 20993-0002 or the
Office of Communication, Outreach and Development (HFM-40), Center for
Biologics Evaluation and Research (CBER), Food and Drug Administration,
1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448. Send one
self-addressed adhesive label to assist that office in processing your
requests. See the SUPPLEMENTARY INFORMATION section for electronic
access to the guidance document.
Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
rm. 1061, Rockville, MD 20852.
FOR FURTHER INFORMATION CONTACT:
Lawrence J. Lesko, Center for Drug Evaluation and Research, Food and
Drug Administration, 10903 New Hampshire Ave., Bldg. 51, rm. 3178,
Silver Spring, MD 20993-0002, 301-796-1565; or
Shiew-Mei Huang, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 51, rm. 3188, Silver
Spring, MD 20993-0002, 301-796-1541; or
Stephen Ripley, Center for Biologics Evaluation and Research, Food and
Drug Administration (HFM-17), 1401 Rockville Pike, suite 200N,
Rockville, MD 20852-1448, 301-827-6210.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance entitled
``Clinical Pharmacogenomics: Premarketing Evaluation in Early Phase
Clinical Studies.'' Pharmacogenomics (PGx) broadly refers to the study
of variations of DNA and RNA characteristics and their relation to drug
exposure and/or response. Drug exposure refers to either the
administered dose or levels in a body tissue or fluid (e.g., blood,
plasma, cerebrospinal fluid). Drug response results from the interplay
of pharmacokinetics (e.g., drug absorption, metabolism, and excretion),
and pharmacodynamics (i.e., all of the effects of the drug on various
physiologic and pathologic processes, including effectiveness and
adverse effects). Genetic variations can also influence the exposure-
response (E/R) relationship of drugs. PGx studies can enhance the
understanding of interindividual differences in the efficacy and safety
of investigational drugs.
Drug development is commonly described as going through ``phases''
(21 CFR 312.21). The first two phases collect information about safety
and dosing, so that the larger, later (phase 3) studies (the adequate
and well-controlled studies needed to support marketing approval) can
gather the additional information about effectiveness and safety that
is needed to evaluate the overall benefit-risk relationship of the drug
and to provide an adequate basis for physician labeling. Much of the
genomic information collected and assessed during the early phases is
often described as ``exploratory.'' Phase 2 studies that suggest
genomic influences can lead to phase 3 trials that incorporate findings
into prespecified hypotheses, such as enriching the study with
genomically defined individuals, determining dose based on demonstrated
variability in earlier studies, and defining a priori hypothesis
testing of a primary endpoint in a genomic subset.
PGx information obtained from genomic investigations during the
course of drug development (and from postmarketing studies) can improve
the effectiveness and safety of drugs by identifying patients at high
risk for a serious adverse event or absence of benefit; improving the
benefit/risk relationship of drugs by using genomic tests to identify
patients most likely to respond, or unable to respond to a drug; and by
helping to select optimal doses based on genotype-driven differences in
PK (pharmacodynamics) and/or PD (pharmacodynamics) of a drug. An
important prerequisite to successful use of genetic information in drug
development is appropriate collection and storage of DNA samples from
all clinical trials, both exploratory and the adequate and well-
controlled studies intended to support effectiveness and safety.
This draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance
represents the Agency's current thinking on conducting pharmacogenomic
studies in
[[Page 9584]]
early phase clinical studies. It does not create or confer any rights
for or on any person and does not operate to bind FDA or the public. An
alternative approach may be used if such approach satisfies the
requirement of the applicable statutes and regulations.
II. Paperwork Reduction Act of 1995
This draft guidance refers to previously approved collections of
information that are subject to review by the Office of Management and
Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-
3520). The collections of information in 21 CFR 201.57 have been
approved under OMB control number 0910-0572.
III. Comments
Interested persons may submit to the Division of Dockets Management
(see ADDRESSES) either electronic or written comments regarding this
document. It is only necessary to send one set of comments. It is no
longer necessary to send two copies of mailed comments. Identify
comments with the docket number found in brackets in the heading of
this document. Received comments may be seen in the Division of Dockets
Management between 9 a.m. and 4 p.m., Monday through Friday.
IV. Electronic Access
Persons with access to the Internet may obtain the document at
either http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances, or http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances, or http://www.regulations.gov.
Dated: February 14, 2011.
Leslie Kux,
Acting Assistant Commissioner for Policy.
[FR Doc. 2011-3679 Filed 2-17-11; 8:45 am]
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