Biomarker Qualification: Evidentiary Framework; Draft Guidance for Industry and Food and Drug Administration Staff; Availability, 63892-63894 [2018-26900]
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Federal Register / Vol. 83, No. 238 / Wednesday, December 12, 2018 / Notices
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amozie on DSK3GDR082PROD with NOTICES1
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Dated: December 6, 2018.
Julie Hocker,
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(AoD).
[FR Doc. 2018–26857 Filed 12–11–18; 8:45 am]
BILLING CODE 4154–01–P
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DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–D–4267]
Biomarker Qualification: Evidentiary
Framework; Draft Guidance for
Industry and Food and Drug
Administration Staff; 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announcing the availability of a draft
guidance for industry and FDA staff
entitled ‘‘Biomarker Qualification:
Evidentiary Framework.’’ This draft
guidance provides recommendations on
general considerations to address when
developing a biomarker for qualification
under the 21st Century Cures Act (Cures
Act), enacted on December 13, 2016,
that added a new section to the Federal
Food, Drug, and Cosmetic Act (FD&C
Act). Qualification of a biomarker is a
determination that within the stated
context of use, the biomarker can be
relied on to have a specific
interpretation and application in drug
development and regulatory review.
DATES: Submit either electronic or
written comments on the draft guidance
by February 11, 2019 to ensure that the
Agency considers your comment on this
draft guidance before it begins work on
the final version of the guidance.
ADDRESSES: You may submit comments
on any guidance at any time as follows:
SUMMARY:
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
PO 00000
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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–
2018–D–4267 for ‘‘Biomarker
Qualification: Evidentiary Framework;
Draft Guidance for Industry and FDA
Staff.’’ 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 between 9
a.m. and 4 p.m., Monday through
Friday.
• 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.gpo.gov/
E:\FR\FM\12DEN1.SGM
12DEN1
Federal Register / Vol. 83, No. 238 / Wednesday, December 12, 2018 / Notices
amozie on DSK3GDR082PROD with NOTICES1
fdsys/pkg/FR-2015-09-18/pdf/201523389.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.
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. 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:
Chris Leptak, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 22, Rm. 6461, Silver Spring,
MD 20993–0002, 301–796–0017.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a draft guidance for industry and FDA
staff entitled ‘‘Biomarker Qualification:
Evidentiary Framework.’’ This draft
guidance provides recommendations on
general considerations to address when
developing a biomarker for qualification
under the Cures Act, enacted on
December 13, 2016, that added a new
section 507, Qualification of Drug
Development Tools, to the FD&C Act (21
U.S.C. 357).1 This guidance discusses
the evidentiary framework that should
be used to support biomarker
qualification, as that term is now used
in section 507 of the FD&C Act, and it
was informed by public workshops that
predated the Cures Act. Specifically,
this guidance describes the needs
assessment, context of use, and benefitrisk considerations, and how these
considerations can relate to determining
the type and level of evidence to
support qualification of a biomarker.
This guidance also addresses general
statistical and clinical considerations
related to the correlation between the
biomarker and the outcome of interest,
as well as general analytical
1 Section 507 of the FD&C Act was added by
section 3011(a) of the Cures Act (Pub. L. 114–255).
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Jkt 247001
considerations related to the
performance characteristics of the
biomarker test.
Historically, biomarkers gained
acceptance for use in drug development
after evidence from scientific and
medical communities accumulated over
time, leading to the recognition of the
role and value of the biomarker in
decision making. This evidence was
considered as part of drug-specific
development efforts, and there was no
formal regulatory process to assess the
broader utility of the biomarker
independent from its use in a specific
drug program. Even after the Center for
Drug Evaluation and Research
established the legacy (pre-Cures Act)
Biomarker Qualification Program in
2007, progress in the development of
biomarkers and their application in drug
development has been hampered by the
lack of a clear, predictable, and specific
regulatory framework for the evidence
sufficient to support regulatory decision
making using biomarkers. This guidance
is an additional step towards informing
future guidances that will specifically
address this need, the Cures Act
requirements, and commitments from
the Prescription Drug User Fee
Reauthorization Performance Goals and
Procedures Fiscal Years 2018 through
2022 (PDUFA VI goals letter) 2.
This guidance was informed by
several public workshops 3 that
discussed the science to support
biomarker qualification; these
workshops convened before the
enactment of the Cures Act.
Development of this guidance was also
greatly facilitated by the efforts from the
biomarker development community—
including FDA, National Institutes of
Health (NIH), industry, academia,
patient groups, and the nonprofit
sector—that developed an October 2016
white paper describing a Framework for
Defining Evidentiary Criteria for
2 The
PDUFA VI goals letter is available at:
https://www.fda.gov/downloads/ForIndustry/
UserFees/PrescriptionDrugUserFee/
UCM511438.pdf.
3 Workshops convened to discuss the science to
support biomarker qualification included: Institute
of Medicine Workshop on Biomarker Qualification
(2009), FDA co-sponsored Biomarkers Workshop
with Howard Hughes Medical Institute (2013), FDA
co-sponsored Brookings meeting on Advancing the
Use of Biomarkers and Pharmacogenomics (2014),
FDA co-sponsored workshop with M–CERSI and
the Critical Path Institute on Evidentiary
Considerations for Integration of Biomarkers in
Drug Development (2015), NIH–FDA Workshop on
Biomarker Glossary of Terms (2015), the National
Biomarker Development Alliance’s Workshop on
Collaboratively Building a Foundation for FDA
Biomarker Qualification (2015), and Foundation for
the NIH–FDA Workshop on Developing an
Evidentiary Criteria Framework for Safety
Biomarkers Qualification (2016).
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63893
Biomarker Qualification.4 In addition to
considering public comments received
regarding this guidance, FDA
anticipates that the Agency will
incorporate additional information
required under the Cures Act and
discussed in the PDUFA VI goals letter
in a subsequent revised draft version of
this guidance. Ultimately, FDA
anticipates that a future revised draft
guidance on this topic will meet the
statutory requirement for guidance on a
conceptual framework describing
appropriate standards and scientific
approaches to support the development
of biomarkers as described in section
3011(b)(1)(A) of the Cures Act and meet
the commitment in section (1)(J)(6)(d) of
the PDUFA VI goals letter related to
publishing a draft guidance on general
evidentiary standards for biomarker
qualification. As part of FDA’s efforts to
delineate the conceptual framework to
support biomarker qualification and the
general evidentiary standards for
biomarker qualification, FDA also
anticipates that subsequent guidance on
biomarker qualification will address
specific aspects of evidentiary
considerations (e.g., statistical,
analytical) in greater detail. 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 ‘‘Biomarker Qualification:
Evidentiary Framework.’’ 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. This
guidance is not subject to Executive
Order 12866.
II. Paperwork Reduction Act of 1995
This draft guidance refers to
previously approved collections of
information that are subject to review by
the Office of Management and Budget
(OMB) under the Paperwork Reduction
Act of 1995 (44 U.S.C. 3501–3520). The
collections of information in 21 CFR
parts 312 and 314 have been approved
under OMB control numbers 0910–0014
and 0910–0001, respectively.
III. Electronic Access
4 Biomarkers Consortium Evidentiary Standards
Writing Group: Framework for Defining Evidentiary
Criteria for Biomarker Qualification. Final version
October 20, 2016. Available at: https://fnih.org/
sites/default/files/final/pdf/Evidentiary%20Criteria
%20Framework%20Final%20Version%20Oct
%2020%202016.pdf.
E:\FR\FM\12DEN1.SGM
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Federal Register / Vol. 83, No. 238 / Wednesday, December 12, 2018 / Notices
Persons with access to the internet
may obtain the draft guidance at either
https://www.fda.gov/Drugs/
GuidanceComplianceRegulatory
Information/Guidances/default.htm or
https://www.regulations.gov.
Dated: December 7, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–26900 Filed 12–11–18; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2015–D–4750]
The ‘‘Deemed to be a License’’
Provision of the BPCI Act: Questions
and Answers; Draft Guidance for
Industry; Availability; Request for
Comments on Preliminary List of
Affected Applications
AGENCY:
Food and Drug Administration,
HHS.
Notice of availability; request
for comments.
ACTION:
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled ‘‘The
‘Deemed to be a License’ Provision of
the BPCI Act: Questions and Answers.’’
This draft guidance is intended to
provide answers to common questions
about FDA’s interpretation of the
statutory provision under which an
application for a biological product
approved under the Federal Food, Drug,
and Cosmetic Act (FD&C Act) as of
March 23, 2020, will be deemed to be
a license for the biological product
under the Public Health Service Act
(PHS Act) on March 23, 2020. This
guidance also describes FDA’s
compliance policy for the labeling of
biological products that will be the
subject of deemed biologics license
applications (BLAs). This guidance is
intended to facilitate planning for the
March 23, 2020, transition date and
provide further clarity regarding the
Agency’s interpretation of this statutory
provision. FDA also invites comment on
the preliminary list of approved new
drug applications (NDAs) for biological
products under the FD&C Act that will
be deemed to be BLAs on the transition
date.
DATES: Submit either electronic or
written comments on the draft guidance
by February 11, 2019 to ensure that the
Agency considers your comment on this
amozie on DSK3GDR082PROD with NOTICES1
SUMMARY:
VerDate Sep<11>2014
18:39 Dec 11, 2018
Jkt 247001
draft guidance before it begins work on
the final version of the guidance.
ADDRESSES: You may submit comments
on any guidance at any time as follows:
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
identified, as confidential, if submitted
as detailed in ‘‘Instructions.’’
Instructions: All submissions received
must include the Docket No. FDA–
2015–D–4750 for ‘‘The ‘Deemed to be a
License’ Provision of the BPCI Act:
Questions and Answers; Draft Guidance
for Industry.’’ 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 between 9
a.m. and 4 p.m., Monday through
Friday.
• Confidential Submissions—To
submit a comment with confidential
information that you do not wish to be
made publicly available, submit your
PO 00000
Frm 00070
Fmt 4703
Sfmt 4703
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.gpo.gov/
fdsys/pkg/FR-2015-09-18/pdf/201523389.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.
You may submit comments on any
guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single
copies of this 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 to the Office of
Communication, Outreach and
Development, Center for Biologics
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 3128,
Silver Spring, MD 20993–0002. 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:
Janice Weiner, Center for Drug
Evaluation and Research, Food and
E:\FR\FM\12DEN1.SGM
12DEN1
Agencies
[Federal Register Volume 83, Number 238 (Wednesday, December 12, 2018)]
[Notices]
[Pages 63892-63894]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-26900]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-D-4267]
Biomarker Qualification: Evidentiary Framework; Draft Guidance
for Industry and Food and Drug Administration Staff; 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 and FDA staff
entitled ``Biomarker Qualification: Evidentiary Framework.'' This draft
guidance provides recommendations on general considerations to address
when developing a biomarker for qualification under the 21st Century
Cures Act (Cures Act), enacted on December 13, 2016, that added a new
section to the Federal Food, Drug, and Cosmetic Act (FD&C Act).
Qualification of a biomarker is a determination that within the stated
context of use, the biomarker can be relied on to have a specific
interpretation and application in drug development and regulatory
review.
DATES: Submit either electronic or written comments on the draft
guidance by February 11, 2019 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final
version of the guidance.
ADDRESSES: You may submit comments on any guidance at any time as
follows:
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 identified,
as confidential, if submitted as detailed in ``Instructions.''
Instructions: All submissions received must include the Docket No.
FDA-2018-D-4267 for ``Biomarker Qualification: Evidentiary Framework;
Draft Guidance for Industry and FDA Staff.'' 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 between 9 a.m. and 4 p.m., Monday through
Friday.
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.gpo.gov/
[[Page 63893]]
fdsys/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.
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. 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: Chris Leptak, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6461, Silver Spring, MD 20993-0002, 301-
796-0017.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
and FDA staff entitled ``Biomarker Qualification: Evidentiary
Framework.'' This draft guidance provides recommendations on general
considerations to address when developing a biomarker for qualification
under the Cures Act, enacted on December 13, 2016, that added a new
section 507, Qualification of Drug Development Tools, to the FD&C Act
(21 U.S.C. 357).\1\ This guidance discusses the evidentiary framework
that should be used to support biomarker qualification, as that term is
now used in section 507 of the FD&C Act, and it was informed by public
workshops that predated the Cures Act. Specifically, this guidance
describes the needs assessment, context of use, and benefit-risk
considerations, and how these considerations can relate to determining
the type and level of evidence to support qualification of a biomarker.
This guidance also addresses general statistical and clinical
considerations related to the correlation between the biomarker and the
outcome of interest, as well as general analytical considerations
related to the performance characteristics of the biomarker test.
---------------------------------------------------------------------------
\1\ Section 507 of the FD&C Act was added by section 3011(a) of
the Cures Act (Pub. L. 114-255).
---------------------------------------------------------------------------
Historically, biomarkers gained acceptance for use in drug
development after evidence from scientific and medical communities
accumulated over time, leading to the recognition of the role and value
of the biomarker in decision making. This evidence was considered as
part of drug-specific development efforts, and there was no formal
regulatory process to assess the broader utility of the biomarker
independent from its use in a specific drug program. Even after the
Center for Drug Evaluation and Research established the legacy (pre-
Cures Act) Biomarker Qualification Program in 2007, progress in the
development of biomarkers and their application in drug development has
been hampered by the lack of a clear, predictable, and specific
regulatory framework for the evidence sufficient to support regulatory
decision making using biomarkers. This guidance is an additional step
towards informing future guidances that will specifically address this
need, the Cures Act requirements, and commitments from the Prescription
Drug User Fee Reauthorization Performance Goals and Procedures Fiscal
Years 2018 through 2022 (PDUFA VI goals letter) \2\.
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\2\ The PDUFA VI goals letter is available at: https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf.
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This guidance was informed by several public workshops \3\ that
discussed the science to support biomarker qualification; these
workshops convened before the enactment of the Cures Act. Development
of this guidance was also greatly facilitated by the efforts from the
biomarker development community--including FDA, National Institutes of
Health (NIH), industry, academia, patient groups, and the nonprofit
sector--that developed an October 2016 white paper describing a
Framework for Defining Evidentiary Criteria for Biomarker
Qualification.\4\ In addition to considering public comments received
regarding this guidance, FDA anticipates that the Agency will
incorporate additional information required under the Cures Act and
discussed in the PDUFA VI goals letter in a subsequent revised draft
version of this guidance. Ultimately, FDA anticipates that a future
revised draft guidance on this topic will meet the statutory
requirement for guidance on a conceptual framework describing
appropriate standards and scientific approaches to support the
development of biomarkers as described in section 3011(b)(1)(A) of the
Cures Act and meet the commitment in section (1)(J)(6)(d) of the PDUFA
VI goals letter related to publishing a draft guidance on general
evidentiary standards for biomarker qualification. As part of FDA's
efforts to delineate the conceptual framework to support biomarker
qualification and the general evidentiary standards for biomarker
qualification, FDA also anticipates that subsequent guidance on
biomarker qualification will address specific aspects of evidentiary
considerations (e.g., statistical, analytical) in greater detail. This
draft guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115).
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\3\ Workshops convened to discuss the science to support
biomarker qualification included: Institute of Medicine Workshop on
Biomarker Qualification (2009), FDA co-sponsored Biomarkers Workshop
with Howard Hughes Medical Institute (2013), FDA co-sponsored
Brookings meeting on Advancing the Use of Biomarkers and
Pharmacogenomics (2014), FDA co-sponsored workshop with M-CERSI and
the Critical Path Institute on Evidentiary Considerations for
Integration of Biomarkers in Drug Development (2015), NIH-FDA
Workshop on Biomarker Glossary of Terms (2015), the National
Biomarker Development Alliance's Workshop on Collaboratively
Building a Foundation for FDA Biomarker Qualification (2015), and
Foundation for the NIH-FDA Workshop on Developing an Evidentiary
Criteria Framework for Safety Biomarkers Qualification (2016).
\4\ Biomarkers Consortium Evidentiary Standards Writing Group:
Framework for Defining Evidentiary Criteria for Biomarker
Qualification. Final version October 20, 2016. Available at: https://fnih.org/sites/default/files/final/pdf/Evidentiary%20Criteria%20Framework%20Final%20Version%20Oct%2020%202016.pdf.
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The draft guidance, when finalized, will represent the current
thinking of FDA on ``Biomarker Qualification: Evidentiary Framework.''
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. This
guidance is not subject to Executive Order 12866.
II. Paperwork Reduction Act of 1995
This draft guidance refers to previously approved collections of
information that are subject to review by the Office of Management and
Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-
3520). The collections of information in 21 CFR parts 312 and 314 have
been approved under OMB control numbers 0910-0014 and 0910-0001,
respectively.
III. Electronic Access
[[Page 63894]]
Persons with access to the internet may obtain the draft guidance
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or
https://www.regulations.gov.
Dated: December 7, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-26900 Filed 12-11-18; 8:45 am]
BILLING CODE 4164-01-P