Notice2023-05561
Pharmacogenomic Data Submissions; Draft Guidance for Industry; Availability
Primary source
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Published
March 20, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Pharmacogenomic Data Submissions." This draft guidance is intended to facilitate progress in the field of pharmacogenomics and the use of pharmacogenomic data in drug development. The draft guidance is intended to clarify the contexts in which pharmacogenomic study findings and data must be included in submissions related to investigational new drug applications (INDs), new drug applications (NDAs), and biologics license applications (BLAs) based on the FDA's regulations. In addition, this document provides recommendations to sponsors and applicants on the format and content of the pharmacogenomic data submissions.
Full Text
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<title>Federal Register, Volume 88 Issue 53 (Monday, March 20, 2023)</title>
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[Federal Register Volume 88, Number 53 (Monday, March 20, 2023)]
[Notices]
[Pages 16640-16642]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-05561]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2022-D-2856]
Pharmacogenomic Data Submissions; Draft Guidance for Industry;
Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a draft guidance for industry entitled
``Pharmacogenomic Data Submissions.'' This draft guidance is intended
to facilitate progress in the field of pharmacogenomics and the use of
pharmacogenomic data in drug development. The draft guidance is
intended to clarify the contexts in which pharmacogenomic study
findings and data must be included in submissions related to
investigational new drug applications (INDs), new drug applications
(NDAs), and biologics license applications (BLAs) based on the FDA's
regulations. In addition, this document provides recommendations to
sponsors and applicants on the format and content of the
pharmacogenomic data submissions.
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 June 20, 2023.
ADDRESSES: You may submit comments as follows:
Electronic Submissions
Submit electronic comments in the following way:
[[Page 16641]]
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
will be posted to the docket unchanged. Because your comment will be
made public, you are solely responsible for ensuring that your comment
does not include any confidential information that you or a third party
may not wish to be posted, such as medical information, your or anyone
else's Social Security number, or confidential business information,
such as a manufacturing process. Please note that if you include your
name, contact information, or other information that identifies you in
the body of your comments, that information will be posted on <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> If you want to submit a comment with confidential
information that you do not wish to be made available to the public,
submit the comment as a written/paper submission and in the manner
detailed (see ``Written/Paper Submissions'' and ``Instructions'').
Written/Paper Submissions
Submit written/paper submissions as follows:
<bullet> Mail/Hand Delivery/Courier (for written/paper
submissions): Dockets Management Staff (HFA-305), Food and Drug
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
<bullet> For written/paper comments submitted to the Dockets
Management Staff, FDA will post your comment, as well as any
attachments, except for information submitted, marked and identified,
as confidential, if submitted as detailed in ``Instructions.''
Instructions: All submissions received must include the Docket No.
FDA-2022-D-2856 for ``Pharmacogenomic Data Submissions.'' Received
comments will be placed in the docket and, except for those submitted
as ``Confidential Submissions,'' publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets Management Staff office between 9
a.m. and 4 p.m., Monday through Friday, 240-402-7500.
<bullet> Confidential Submissions--To submit a comment with
confidential information that you do not wish to be made publicly
available, submit your comments only as a written/paper submission. You
should submit two copies total. One copy will include the information
you claim to be confidential with a heading or cover note that states
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will
review this copy, including the claimed confidential information, in
its consideration of comments. The second copy, which will have the
claimed confidential information redacted/blacked out, will be
available for public viewing and posted on <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Submit both copies to the Dockets Management Staff. If you do not wish
your name and contact information to be made publicly available, you
can provide this information on the cover sheet and not in the body of
your comments and you must identify this information as
``confidential.'' Any information marked as ``confidential'' will not
be disclosed except in accordance with 21 CFR 10.20 and other
applicable disclosure law. For more information about FDA's posting of
comments to public dockets, see 80 FR 56469, September 18, 2015, or
access the information at: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> and insert the docket number, found in brackets in
the heading of this document, into the ``Search'' box and follow the
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852, 240-402-7500.
You may submit comments on any guidance at any time (see 21 CFR
10.115(g)(5)).
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, 10001 New Hampshire Ave.,
Hillandale Building, 4th Floor, Silver Spring, MD 20993-0002 or the
Center for Biologics Evaluation and Research, Office of Communication,
Outreach, and Development, 10903 New Hampshire Ave., Bldg. 71, Rm.
3128, Silver Spring, MD 20903. 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.
FOR FURTHER INFORMATION CONTACT:
For Center of Drug Evaluation and Research: Michael Pacanowski,
Center for Drug Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 51, Rm. 2132, Silver Spring, MD 20993,
301-796-3919.
For Center of Biologics Evaluation and Research: Stephen Ripley,
Center for Biologics Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver
Spring, MD 20993, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Pharmacogenomic Data Submissions.'' The draft guidance
outlines FDA's expectations for the submission of data from
pharmacogenomic studies considering the advances in genomics research
that have occurred since FDA published final guidance on
``Pharmacogenomic Data Submissions'' in 2005. This guidance, when
finalized, will replace the 2005 final guidance.
Pharmacogenomic studies that evaluate the effect of variations in
DNA or RNA characteristics on drug concentrations or response have the
potential to help identify sources of inter-individual variability and
characterize the pharmacologic effects of a drug. In some cases,
pharmacogenomic studies can identify biomarkers that make it possible
to individualize therapy. In addition, pharmacogenomic biomarkers that
have well-accepted mechanistic and clinical significance are currently
being integrated into drug development (e.g., enriched clinical trial
designs) and clinical practice (e.g., clinical testing to determine
dose).
Sponsors submitting or holding INDs, NDAs, or BLAs are subject to
FDA requirements for submitting data to the Agency that are relevant to
drug safety and effectiveness (including 21 CFR 312, 314, and 601).
However, the regulations were developed before the advent of widespread
animal or human genetic testing (e.g., high-throughput DNA sequencing)
or gene expression testing and do not specifically address when such
data must be submitted. This document, when final, will constitute
FDA's current thinking about pharmacogenomic study results and the
associated data required to be submitted in an IND, NDA, or BLA, as
well as the FDA's recommendations as to the level of detail and format
for reporting.
In addition, this draft guidance has also removed references to the
Voluntary Genomic Data Submission program (VGDS; later referred to as
Voluntary eXploratory Data Submission program, or VXDS). The VGDS
program created a pathway for voluntary exchanges between FDA and the
pharmaceutical industry or other stakeholders regarding genomic and
other biomarker studies in the context of individual drug development
programs. The program helped the Agency gain knowledge regarding
genomics research in the context of drug development and
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practical experience with data submission and analysis. Since it was
first introduced in 2003, the VXDS program has received over 50
voluntary submissions. In recent years, FDA has established additional
pathways to interact with stakeholders on biomarker development, such
as the Biomarker Qualification Program and Critical Path Innovation
Meetings. Given the availability of these programs and decreasing use
of the program, FDA is considering ending the program, and references
to the VGDS program have been removed from this draft guidance.
However, FDA seeks public feedback on the following specific issues:
<bullet> The VGDS program created a pathway and infrastructure for
stakeholders to voluntarily submit genomic or other data to FDA, when
such data are not otherwise required to be submitted to FDA. Such a
submission pathway could support regulatory science initiatives (e.g.,
aggregating data from multiple programs to support endpoint
development). While it is FDA's plan to discontinue the VGDS program in
its current form, FDA requests feedback on the utility of maintaining a
voluntary submission pathway that is of value to both FDA and the
pharmaceutical industry.
<bullet> FDA requests public input on particular platforms or
technologies that would benefit most from standardization.
This draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance, when
finalized, will represent the current thinking of FDA on
pharmacogenomic data submissions to the Agency. It does not establish
any rights for any person and is not binding on FDA or the public. You
can use an alternative approach if it satisfies the requirements of the
applicable statutes and regulations.
II. Paperwork Reduction Act of 1995
While this guidance contains no collection of information, it does
refer to previously approved FDA collections of information. Therefore,
clearance by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not
required for this guidance. The previously approved collections of
information are subject to review by OMB under the PRA. The collections
of information in 21 CFR parts 50 and 56 pertaining to informed consent
have been approved under OMB control number 0910-0130. The collections
of information in 21 CFR part 312 pertaining to submissions of
investigational new drug applications (IND), including clinical trial
design and study protocols, IND Safety Reports, Annual Reports and
voluntary pharmacogenomic data have been approved under OMB control
number 0910-0014. The collections of information in 21 CFR part 314
pertaining to submissions of new drug applications have been approved
under OMB control number 0910-0001. The collections of information in
21 CFR part 601 pertaining to submissions of biologics license
applications have been approved under OMB control number 0910-0338.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at <a href="https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm">https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, <a href="https://www.regulations.gov">https://www.regulations.gov</a>, or <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics</a>.
Dated: March 14, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-05561 Filed 3-17-23; 8:45 am]
BILLING CODE 4164-01-P
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