[Federal Register: November 4, 2003 (Volume 68, Number 213)]
[Notices]
[Page 62461-62463]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr04no03-60]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 2003D-0497]
Draft Guidance for Industry on Pharmacogenomic Data Submissions;
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availability of a draft guidance for industry entitled
``Pharmacogenomic Data Submissions.'' The draft guidance provides
recommendations to sponsors holding investigational new drug
applications (INDs), new drug applications (NDAs), and biologics
license applications (BLAs) on what pharmacogenomic data to submit to
the agency during the drug development process, the format of
submissions, and how the data will be used in regulatory
decisionmaking. The draft guidance is intended to facilitate scientific
progress in the area of pharmacogenomics, which should enable the FDA
to use pharmacogenomic data in regulatory policies and decision making.
DATES: Submit written or electronic comments on the draft guidance by
February 2, 2004. General comments on agency guidance documents are
welcome at any time. Submit written or electronic comments on the
collection of information by January 5, 2004.
ADDRESSES: Submit written requests for single copies of the draft
guidance to the Division of Drug Information (HFD-240), Center for Drug
Evaluation and Research, Food and Drug Administration, 5600 Fishers
Lane, Rockville, MD 20857; or the Office of Communication, Training and
Manufacturers Assistance (HFM-40), Center for Biologics Evaluation and
Research, Food and Drug Administration, 1401 Rockville Pike, 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section for electronic access to the draft guidance document.
Submit written comments on the draft guidance and on the collection
of information to the Division of Dockets Management (HFA-305), Food
and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD
20852. Submit electronic comments on the draft guidance and the
collection of information to http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/dockets/ecomments.
FOR FURTHER INFORMATION CONTACT: Lawrence Lesko, Center for Drug
Evaluation and Research (HFD-850), Food and Drug Administration, 5600
Fishers Lane, Rockville, MD 20857, 301-594-5690, or
Raj Puri, Center for Biologics Evaluation and Research (HFM-735),
[[Page 62462]]
Food and Drug Administration, 1401 Rockville Pike, Rockville, MD 20852-
1448, 301-827-0471.
SUPPLEMENTARY INFORMATION:
I. Background
Although the field of pharmacogenomics is in its infancy, the
promise of pharmacogenomics lies in its potential to predict sources of
interindividual variability in drug response (both efficacy and
toxicity), thus allowing individualization of therapy to maximize
effectiveness and minimize risk. Pharmaceutical sponsors have been
reluctant to embark on programs of pharmacogenomic testing during the
FDA-regulated phases of drug development, due to uncertainties in how
FDA will react to the data being generated.
To facilitate scientific progress in the area of pharmacogenomics,
FDA is announcing the availability of a draft guidance for industry
entitled ``Pharmacogenomic Data Submissions.'' The draft guidance
provides recommendations to sponsors holding INDs, NDAs, and BLAs on
what pharmacogenomic data to submit to the agency during the drug
development process, the format of submissions, and how the data will
be used in regulatory decisionmaking. The draft guidance is also
intended to faciliate the agency's use of such data during regulatory
decisionmaking.
Sponsors submitting or holding INDs, NDAs, or BLAs are subject to
FDA requirements for submitting to the agency data relevant to drug
safety and efficacy (Sec. Sec. 312.22, 312.23, 312.31, 312.33, 314.50,
314.81, 601.2, and 601.12 (21 CFR 312.22, 312.23, 312.31, 312.33,
314.50, 314.81, 601.2, and 601.12)). These regulations were developed
before the advent of widespread animal or human genetic or gene
expression testing. FDA has received numerous inquiries about how
sponsors who are conducting such testing can comply with the
regulations. From a public policy perspective, a number of factors
should be considered when interpreting how these regulations should
apply to the developing field of pharmacogenomics. This draft guidance
discusses these factors as well as the content and possible formats for
submitting pharmacogenomic data to the agency in INDs, NDAs, and BLAs
and how FDA expects to use the data in regulatory decisionmaking.
II. Comments
Interested persons may submit to the Division of Dockets Management
(see ADDRESSES) written or electronic comments on the draft guidance.
Two copies of mailed comments are to be submitted, except that
individuals may submit one copy. Comments are to be identified with the
docket number found in brackets in the heading of this document. The
draft guidance and received comments are available for public
examination in the Division of Dockets Management between 9 a.m. and 4
p.m., Monday through Friday.
III. The Paperwork Reduction Act of 1995
Under the Paperwork Reduction Act (44 U.S.C. 3501-3520) (the PRA),
Federal agencies must obtain approval from the Office of Management and
Budget (OMB) for each collection of information they conduct or
sponsor. ``Collection of information'' is defined in 44 U.S.C. 3502(3)
and 5 CFR 1320.3(c) and includes agency requests or requirements that
members of the public submit reports, keep records, or provide
information to a third party. Section 3506(c)(2)(A) of the PRA (44
U.S.C. 3506(c)(2)(A)) requires Federal agencies to provide a 60-day
notice in the Federal Register concerning each proposed collection of
information before submitting the collection to OMB for approval. To
comply with this requirement, FDA is publishing notice of the proposed
collection of information set forth in this document.
With respect to the following collection of information, FDA
invites comments on these topics: (1) Whether the proposed collection
of information is necessary for the proper performance of FDA's
functions, including whether the information will have practical
utility; (2) the accuracy of FDA's estimate of the burden of the
proposed collection of information, including the validity of the
methodology and assumptions used; (3) ways to enhance the quality,
utility, and clarity of the information to be collected; and (4) ways
to minimize the burden of the collection on respondents, including
through the use of automated collection techniques, when appropriate,
and other forms of information technology.
Title: Draft Guidance for Industry on Pharmacogenomic Data
Submissions.
Description: The draft guidance provides recommendations to
sponsors submitting or holding INDs, NDAs, or BLAs on what
pharmacogenomic data should be submitted to the agency during the drug
development process. Sponsors holding and applicants submitting INDs,
NDAs, or BLAs are subject to FDA requirements for submitting to the
agency data relevant to drug safety and efficacy (Sec. Sec. 312.22,
312.23, 312.31, 312.33, 314.50, 314.81, 601.2, and 601.12).
Description of Respondents: Sponsors submitting or holding INDs,
NDAs, and BLAs for human drugs and biologics.
Burden Estimate: The draft guidance interprets FDA regulations for
IND, NDA, or BLA submissions, clarifying when the regulations require
pharmacogenomics data to be submitted and when the submission of such
data is voluntary. The pharmacogenomic data submissions described in
the draft guidance that are required to be submitted to an IND, NDA,
BLA, or annual report are covered by the information collection
requirements under parts 312, 314, and 601 (21 CFR parts 312, 314, and
601) and are approved by OMB under control numbers 0910-0014 (part
312--INDs; approved until January 1, 2006); 0910-0001 (part 314--NDAs
and annual reports; approved until March 31, 2005); and 0910-0338
(approved until August 31, 2005).
The draft guidance distinguishes between pharmacogenomic tests that
may be considered valid biomarkers appropriate for regulatory
decisionmaking, and other, less well developed exploratory tests. The
submission of exploratory pharmacogenomic data is not required under
the regulations, although the agency encourages the voluntary
submission of such data.
The draft guidance describes the Voluntary Genomic Data Submission
(VGDS) that can be used for such a voluntary submission. The draft
guidance does not recommend a specific format for the VGDS, except that
such a voluntary submission be designated a VGDS. The data submitted in
a VGDS and the level of detail should be sufficient for FDA to be able
to interpret the information and independently analyze the data, verify
results, and explore possible genotype-phenotype correlations across
studies. FDA does not want the VGDS to be overly burdensome and time-
consuming for the sponsor.
FDA is requesting public comments on the following estimates of the
burden of preparing a voluntary submission described in the draft
guidance that should be designated as a VGDS. Based on FDA's
familiarity with sponsors' interest in submitting pharmacogenomic data
during the drug development process, FDA estimates that approximately
20 sponsors will submit approximately 80 VGDSs and that, on average,
each VGDS will take approximately 10 hours to prepare and submit to
FDA.
[[Page 62463]]
Table 1.--Estimated Annual Reporting Burden\1\
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No. of No. of Responses Total Annual Hours per
Respondents per Respondent Responses Response Total Hours
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Genomic Data 20 4 80 10 800
Submissions
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
information.
IV. Electronic Access
Persons with access to the Internet may obtain the draft guidance
document at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cder/guidance/index.htm, http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cber/guidelines.htm, or http://www.fda.gov/ohrms/dockets/
v/ohrms/dockets/
default.htm.
Dated: October 28, 2003.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 03-27646 Filed 11-3-03; 8:45 am]
BILLING CODE 4160-01-S