[Federal Register Volume 68, Number 213 (Tuesday, November 4, 2003)]
[Notices]
[Pages 62461-62463]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 03-27646]


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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Draft Guidance for Industry on Pharmacogenomic Data Submissions; 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availability of a draft guidance for industry entitled 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 The draft guidance provides 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 The draft guidance is intended to facilitate scientific 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DATES:  Submit written or electronic comments on the draft guidance by 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ADDRESSES:  Submit written requests for single copies of the draft 
guidance to the Division of Drug Information (HFD-240), Center for Drug 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section for electronic access to the draft guidance document.
    Submit written comments on the draft guidance and on the collection 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 Submit electronic comments on the draft guidance and the 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://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.

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                                 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://www.fda.gov/cder/guidance/index.htm, http://www.fda.gov/cber/guidelines.htm, or http://www.fda.gov/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