Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals; Guidance for Industry; Availability, 9533-9534 [2019-04816]
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Federal Register / Vol. 84, No. 51 / Friday, March 15, 2019 / Notices
guidance is not subject to Executive
Order 12866.
II. Paperwork Reduction Act of 1995
This draft guidance refers to
previously approved collections of
information found in FDA regulations.
These collections of information 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 part 50 have
been approved under OMB control
number 0910–0755; the collections of
information in 21 CFR part 312 have
been approved under OMB control
number 0910–0014; the collections of
information in 21 CFR part 812 have
been approved under OMB control
number 0910–0078; and the collections
of information in the guidance for
industry entitled ‘‘Oversight of Clinical
Investigations: A Risk-Based Approach
to Monitoring’’ have been approved
under OMB control number 0910–0733.
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may obtain the draft guidance at https://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/
BiologicsBloodVaccines/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm, https://
www.fda.gov/MedicalDevices/
DeviceRegulationandGuidance/
GuidanceDocuments/default.htm, or
https://www.regulations.gov.
Dated: March 11, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019–04814 Filed 3–14–19; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–D–1328]
Severely Debilitating or LifeThreatening Hematologic Disorders:
Nonclinical Development of
Pharmaceuticals; Guidance for
Industry; Availability
amozie on DSK9F9SC42PROD with NOTICES
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled ‘‘Severely
Debilitating or Life-Threatening
SUMMARY:
VerDate Sep<11>2014
16:53 Mar 14, 2019
Jkt 247001
Hematologic Disorders: Nonclinical
Development of Pharmaceuticals.’’ This
guidance outlines nonclinical studies
recommended for the development of
pharmaceuticals used to treat patients
with severely debilitating or lifethreatening hematologic disorders
(SDLTHDs) and addresses comments
received to the docket. This guidance is
intended to streamline the development
of pharmaceuticals used to treat patients
with SDLTHDs, other than cancer, while
protecting patients’ safety and avoiding
unnecessary use of animals, in
accordance with the 3R (reduce, refine,
replace) principles. This guidance
applies to pharmaceuticals used both to
treat the active disease and to prevent
the recurrence of a life-threatening or
debilitating event.
DATES: The announcement of the
guidance is published in the Federal
Register on March 15, 2019.
ADDRESSES: You may submit either
electronic or written comments on
Agency guidances 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.
PO 00000
Frm 00058
Fmt 4703
Sfmt 4703
9533
• 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–1328 for ‘‘Severely Debilitating
or Life-Threatening Hematologic
Disorders: Nonclinical Development of
Pharmaceuticals.’’ 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/
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.
E:\FR\FM\15MRN1.SGM
15MRN1
9534
Federal Register / Vol. 84, No. 51 / Friday, March 15, 2019 / Notices
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 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 guidance document.
FOR FURTHER INFORMATION CONTACT: John
Leighton, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 22, Rm. 2204, Silver Spring,
MD 20993–0002, 301–796–7550; or
Haleh Saber, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 22, Rm. 2117, Silver Spring,
MD 20993–0002, 301–796–7550.
SUPPLEMENTARY INFORMATION:
amozie on DSK9F9SC42PROD with NOTICES
I. Background
FDA is announcing the availability of
a guidance for industry entitled
‘‘Severely Debilitating or LifeThreatening Hematologic Disorders:
Nonclinical Development of
Pharmaceuticals.’’ The purpose of this
guidance is to assist sponsors in the
design of nonclinical studies for the
development of pharmaceuticals used to
treat patients with SDLTHDs. This
guidance is intended to streamline the
development of pharmaceuticals used to
treat patients with SDLTHDs, other than
cancer, while protecting patients’ safety
and avoiding unnecessary use of
animals, in accordance with the 3R
principles. This guidance applies to
pharmaceuticals used both to treat the
active disease and to prevent the
recurrence of a life-threatening or
debilitating event.
SDLTHDs include conditions in
which life expectancy is short or quality
of life is greatly diminished despite
available therapies. FDA has defined
life-threatening and severely debilitating
diseases in regulations (21 CFR 312.81).
A streamlined approach to drug
development is necessary to allow
patients with SDLTHDs earlier and
continued access to new and potentially
effective therapies. The draft guidance
for industry entitled ‘‘Rare Diseases:
Common Issues in Drug Development’’
(available at https://www.fda.gov/ucm/
groups/fdagov-public/@fdagov-drugsgen/documents/document/ucm4584
85.pdf) does not specifically facilitate
the nonclinical development of
VerDate Sep<11>2014
16:53 Mar 14, 2019
Jkt 247001
pharmaceuticals for treatment of
SDLTHDs. Because SDLTHDs are not all
rare diseases, they can fall outside the
scope of the draft guidance for rare
diseases. The present document
provides consistent guidance for all
nononcology SDLTHDs, independent of
disease incidence or prevalence.
This guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
The guidance represents the current
thinking of FDA on ‘‘Severely
Debilitating or Life-Threatening
Hematologic Disorders: Nonclinical
Development of Pharmaceuticals.’’ 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 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 collection of
information submitted under 21 CFR
part 312 has been approved under OMB
control number 0910–0014. The
collection of information submitted
under 21 CFR part 314 has been
approved under OMB control number
0910–0001.
III. Electronic Access
Persons with access to the internet
may obtain the guidance at either
https://www.fda.gov/Drugs/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm or https://
www.regulations.gov.
Dated: March 11, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019–04816 Filed 3–14–19; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2012–D–1145]
Enrichment Strategies for Clinical
Trials To Support Demonstration of
Effectiveness of Human Drugs and
Biological Products; Guidance for
Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
PO 00000
Notice of availability.
Frm 00059
Fmt 4703
Sfmt 4703
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled
‘‘Enrichment Strategies for Clinical
Trials to Support Demonstration of
Effectiveness of Human Drugs and
Biological Products.’’ The purpose of
this guidance is to assist industry in
developing enrichment strategies that
can be used in clinical investigations
intended to demonstrate effectiveness
(and in some cases safety) of human
drugs and biological products. This
guidance defines several types of
enrichment strategies, provides
examples of potential clinical trial
designs, and discusses potential
regulatory considerations when using
enrichment strategies in clinical trials.
This guidance finalizes the draft
guidance entitled ‘‘Enrichment
Strategies for Clinical Trials to Support
Approval of Human Drugs and
Biological Products’’ issued on
December 17, 2012.
DATES: The announcement of the
guidance is published in the Federal
Register on March 15, 2019.
ADDRESSES: You may submit either
electronic or written comments on
Agency guidances 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
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:
E:\FR\FM\15MRN1.SGM
15MRN1
]]>Agencies
[Federal Register Volume 84, Number 51 (Friday, March 15, 2019)]
[Notices]
[Pages 9533-9534]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-04816]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-D-1328]
Severely Debilitating or Life-Threatening Hematologic Disorders:
Nonclinical Development of Pharmaceuticals; 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 final guidance for industry entitled ``Severely
Debilitating or Life-Threatening Hematologic Disorders: Nonclinical
Development of Pharmaceuticals.'' This guidance outlines nonclinical
studies recommended for the development of pharmaceuticals used to
treat patients with severely debilitating or life-threatening
hematologic disorders (SDLTHDs) and addresses comments received to the
docket. This guidance is intended to streamline the development of
pharmaceuticals used to treat patients with SDLTHDs, other than cancer,
while protecting patients' safety and avoiding unnecessary use of
animals, in accordance with the 3R (reduce, refine, replace)
principles. This guidance applies to pharmaceuticals used both to treat
the active disease and to prevent the recurrence of a life-threatening
or debilitating event.
DATES: The announcement of the guidance is published in the Federal
Register on March 15, 2019.
ADDRESSES: You may submit either electronic or written comments on
Agency guidances 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-1328 for ``Severely Debilitating or Life-Threatening
Hematologic Disorders: Nonclinical Development of Pharmaceuticals.''
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/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.
[[Page 9534]]
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 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 guidance document.
FOR FURTHER INFORMATION CONTACT: John Leighton, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 2204, Silver Spring, MD 20993-0002, 301-
796-7550; or Haleh Saber, Center for Drug Evaluation and Research, Food
and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2117,
Silver Spring, MD 20993-0002, 301-796-7550.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry
entitled ``Severely Debilitating or Life-Threatening Hematologic
Disorders: Nonclinical Development of Pharmaceuticals.'' The purpose of
this guidance is to assist sponsors in the design of nonclinical
studies for the development of pharmaceuticals used to treat patients
with SDLTHDs. This guidance is intended to streamline the development
of pharmaceuticals used to treat patients with SDLTHDs, other than
cancer, while protecting patients' safety and avoiding unnecessary use
of animals, in accordance with the 3R principles. This guidance applies
to pharmaceuticals used both to treat the active disease and to prevent
the recurrence of a life-threatening or debilitating event.
SDLTHDs include conditions in which life expectancy is short or
quality of life is greatly diminished despite available therapies. FDA
has defined life-threatening and severely debilitating diseases in
regulations (21 CFR 312.81). A streamlined approach to drug development
is necessary to allow patients with SDLTHDs earlier and continued
access to new and potentially effective therapies. The draft guidance
for industry entitled ``Rare Diseases: Common Issues in Drug
Development'' (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm458485.pdf) does not
specifically facilitate the nonclinical development of pharmaceuticals
for treatment of SDLTHDs. Because SDLTHDs are not all rare diseases,
they can fall outside the scope of the draft guidance for rare
diseases. The present document provides consistent guidance for all
nononcology SDLTHDs, independent of disease incidence or prevalence.
This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on ``Severely Debilitating or Life-Threatening
Hematologic Disorders: Nonclinical Development of Pharmaceuticals.'' 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 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 collection of information submitted under 21 CFR part 312
has been approved under OMB control number 0910-0014. The collection of
information submitted under 21 CFR part 314 has been approved under OMB
control number 0910-0001.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or
https://www.regulations.gov.
Dated: March 11, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-04816 Filed 3-14-19; 8:45 am]
BILLING CODE 4164-01-P
