Pharmacogenomic Data Submissions; Draft Guidance for Industry; Availability, 16640-16642 [2023-05561]
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Federal Register / Vol. 88, No. 53 / Monday, March 20, 2023 / Notices
I. Background
The Drug Price Competition and
Patent Term Restoration Act of 1984
(Pub. L. 98–417) and the Generic
Animal Drug and Patent Term
Restoration Act (Pub. L. 100–670)
generally provide that a patent may be
extended for a period of up to 5 years
so long as the patented item (human
drug product, animal drug product,
medical device, food additive, or color
additive) was subject to regulatory
review by FDA before the item was
marketed. Under these acts, a product’s
regulatory review period forms the basis
for determining the amount of extension
an applicant may receive.
A regulatory review period consists of
two periods of time: a testing phase and
an approval phase. For human
biological products, the testing phase
begins when the exemption to permit
the clinical investigations of the
biological product becomes effective
and runs until the approval phase
begins. The approval phase starts with
the initial submission of an application
to market the human biological product
and continues until FDA grants
permission to market the biological
product. Although only a portion of a
regulatory review period may count
toward the actual amount of extension
that the Director of USPTO may award
(for example, half the testing phase must
be subtracted as well as any time that
may have occurred before the patent
was issued), FDA’s determination of the
length of a regulatory review period for
a human biological product will include
all of the testing phase and approval
phase as specified in 35 U.S.C.
156(g)(1)(B).
FDA has approved for marketing the
human biologic product JIVI
(antihemophilic factor (recombinant),
PEGylated-aucl). JIVI is indicated for
use in previously treated adults and
adolescents (12 years of age and older)
with hemophilia A (congenital Factor
VIII deficiency) for: (1) on-demand
treatment and control of bleeding
episodes; (2) perioperative management
of bleeding; and (3) routine prophylaxis
to reduce the frequency of bleeding
episodes. Subsequent to this approval,
the USPTO received a patent term
restoration application for JIVI (U.S.
Patent No. 7,632,921) from Bayer
HealthCare LLC, and the USPTO
requested FDA’s assistance in
determining this patent’s eligibility for
patent term restoration. In a letter dated
September 12, 2019, FDA advised the
USPTO that this human biological
product had undergone a regulatory
review period and that the approval of
JIVI represented the first permitted
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17:19 Mar 17, 2023
Jkt 259001
commercial marketing or use of the
product. Thereafter, the USPTO
requested that FDA determine the
product’s regulatory review period.
II. Determination of Regulatory Review
Period
FDA has determined that the
applicable regulatory review period for
JIVI is 2,991 days. Of this time, 2,626
days occurred during the testing phase
of the regulatory review period, while
365 days occurred during the approval
phase. These periods of time were
derived from the following dates:
1. The date an exemption under
section 505(i) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 355(i))
became effective: June 23, 2010. FDA
has verified the applicant’s claim that
the date the investigational new drug
application became effective was on
June 23, 2010.
2. The date the application was
initially submitted with respect to the
human biological product under section
351 of the Public Health Service Act (42
U.S.C. 262): August 30, 2017. FDA has
verified the applicant’s claim that the
biologics license application (BLA) for
JIVI (BLA 125661) was initially
submitted on August 30, 2017.
3. The date the application was
approved: August 29, 2018. FDA has
verified the applicant’s claim that BLA
125661 was approved on August 29,
2018.
This determination of the regulatory
review period establishes the maximum
potential length of a patent extension.
However, the USPTO applies several
statutory limitations in its calculations
of the actual period for patent extension.
In its application for patent extension,
this applicant seeks 1,677 days of patent
term extension.
III. Petitions
Anyone with knowledge that any of
the dates as published are incorrect may
submit either electronic or written
comments and, under 21 CFR 60.24, ask
for a redetermination (see DATES).
Furthermore, as specified in section
60.30 (21 CFR 60.30), any interested
person may petition FDA for a
determination regarding whether the
applicant for extension acted with due
diligence during the regulatory review
period. To meet its burden, the petition
must comply with all the requirements
of section 60.30, including but not
limited to: must be timely (see DATES),
must be filed in accordance with section
10.20, must contain sufficient facts to
merit an FDA investigation, and must
certify that a true and complete copy of
the petition has been served upon the
patent applicant. (See H. Rept. 857, part
PO 00000
Frm 00062
Fmt 4703
Sfmt 4703
1, 98th Cong., 2d sess., pp. 41–42, 1984.)
Petitions should be in the format
specified in 21 CFR 10.30.
Submit petitions electronically to
https://www.regulations.gov at Docket
No. FDA–2013–S–0610. Submit written
petitions (two copies are required) to the
Dockets Management Staff (HFA–305),
Food and Drug Administration, 5630
Fishers Lane, Rm. 1061, Rockville, MD
20852.
Dated: March 13, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023–05570 Filed 3–17–23; 8:45 am]
BILLING CODE 4164–01–P
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.
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:
SUMMARY:
Electronic Submissions
Submit electronic comments in the
following way:
E:\FR\FM\20MRN1.SGM
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Federal Register / Vol. 88, No. 53 / Monday, March 20, 2023 / Notices
lotter on DSK11XQN23PROD with NOTICES1
• 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
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17:19 Mar 17, 2023
Jkt 259001
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-201509-18/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.
PO 00000
Frm 00063
Fmt 4703
Sfmt 4703
16641
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
E:\FR\FM\20MRN1.SGM
20MRN1
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Federal Register / Vol. 88, No. 53 / Monday, March 20, 2023 / Notices
lotter on DSK11XQN23PROD with NOTICES1
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
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17:19 Mar 17, 2023
Jkt 259001
(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/
GuidanceCompliance
RegulatoryInformation/Guidances/
default.htm, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, https://
www.regulations.gov, or https://
www.fda.gov/vaccines-blood-biologics/
guidance-compliance-regulatoryinformation-biologics.
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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2022–E–2805]
Determination of Regulatory Review
Period for Purposes of Patent
Extension; IC–8 APTHERA
INTRAOCULAR LENS
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA or the Agency) has
determined the regulatory review period
for IC–8 APTHERA INTRAOCULAR
LENS and is publishing this notice of
that determination as required by law.
FDA has made the determination
because of the submission of an
application to the Director of the U.S.
Patent and Trademark Office (USPTO),
Department of Commerce, for the
extension of a patent which claims that
medical device.
DATES: Anyone with knowledge that any
of the dates as published (see
SUPPLEMENTARY INFORMATION) are
incorrect may submit either electronic
SUMMARY:
PO 00000
Frm 00064
Fmt 4703
Sfmt 4703
or written comments and ask for a
redetermination by May 19, 2023.
Furthermore, any interested person may
petition FDA for a determination
regarding whether the applicant for
extension acted with due diligence
during the regulatory review period by
September 18, 2023. See ‘‘Petitions’’ in
the SUPPLEMENTARY INFORMATION section
for more information.
ADDRESSES: You may submit comments
as follows. Please note that late,
untimely filed comments will not be
considered. The https://
www.regulations.gov electronic filing
system will accept comments until
11:59 p.m. Eastern Time at the end of
May 19, 2023. Comments received by
mail/hand delivery/courier (for written/
paper submissions) will be considered
timely if they are received on or before
that date.
Electronic Submissions
Submit electronic comments in the
following way:
• 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
E:\FR\FM\20MRN1.SGM
20MRN1
Agencies
[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]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
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]]
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]
BILLING CODE 4164-01-P