[Federal Register Volume 88, Number 53 (Monday, March 20, 2023)]
[Notices]
[Pages 16640-16642]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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Pharmacogenomic Data Submissions; Draft Guidance for Industry; 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the availability of a draft guidance for industry entitled 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 This draft guidance is intended 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 The draft guidance is 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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by June 20, 2023.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:

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     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
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 https://www.regulations.gov.
     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:
     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.
     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 https://www.regulations.gov or at the Dockets Management Staff office between 9 
a.m. and 4 p.m., Monday through Friday, 240-402-7500.
     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 https://www.regulations.gov. 
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: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov 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

[[Page 16642]]

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:
     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.
     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 https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, https://www.regulations.gov, or https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics.

    Dated: March 14, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-05561 Filed 3-17-23; 8:45 am]
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