Product Development in Hemophilia; Public Workshop, 55188-55189 [2018-23947]
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Federal Register / Vol. 83, No. 213 / Friday, November 2, 2018 / Notices
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17:57 Nov 01, 2018
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Dated: October 29, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–23951 Filed 11–1–18; 8:45 am]
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Jkt 247001
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–N–3693]
Product Development in Hemophilia;
Public Workshop
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of public workshop.
The Food and Drug
Administration (FDA, the Agency, or
we) is announcing the following public
workshop entitled ‘‘Product
Development in Hemophilia.’’ The
purpose of the public workshop is to
discuss issues related to development
and regulation of novel hemophilia
products.
SUMMARY:
PO 00000
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The public workshop will be
held on December 6, 2018, from 8:30
a.m. to 4:30 p.m. See the
SUPPLEMENTARY INFORMATION section for
registration date and information.
ADDRESSES: The public workshop will
be held at FDA’s White Oak Campus,
10903 New Hampshire Ave., Bldg. 31
Conference Center, the Great Room (Rm.
1503), Silver Spring, MD 20993–0002.
Entrance for the public workshop
participants (non-FDA employees) is
through Building 1 where routine
security check procedures will be
performed. For parking and security
information, please refer to https://
www.fda.gov/AboutFDA/Workingat
FDA/BuildingsandFacilities/
WhiteOakCampusInformation/
ucm241740.htm.
Docket: For access to the docket to
read background documents 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.
FOR FURTHER INFORMATION CONTACT: Joan
Ferlo Todd, Food and Drug
Administration, Center for Drug
Evaluation and Research, Office of
Hematology and Oncology Products,
10903 New Hampshire Ave., Bldg. 22,
Rm. 2139, Silver Spring, MD 20993–
0002, 301–796–6079, Joan.Todd@
fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
DATES:
I. Background
Hemophilia is a bleeding disorder
caused by deficiency of coagulation
factor VIII (hemophilia A) or
coagulation factor IX (hemophilia B).
Hemophilia treatment strategies are
intended to prevent or control bleeding
and the attendant complications.
Recently, hemophilia treatment
strategies have led to the development
of factor concentrates, recombinant
DNA technology products, antibodies,
and potential curative strategies such as
gene therapy. These new emerging
technologies raise new considerations
about trial design, novel endpoints,
patient-reported outcomes, and longterm safety collection.
This public workshop is intended to
provide a platform for engaging in a
discussion with experts in hemophilia
treatment, patients, and caregivers. The
purpose of this workshop is to advance
further development of patientexperience and patient-reported
outcomes for use in clinical trials,
facilitate reliable and interpretable
measurements of factor VIII/IX activity
E:\FR\FM\02NON1.SGM
02NON1
Federal Register / Vol. 83, No. 213 / Friday, November 2, 2018 / Notices
levels for gene therapy products, discuss
the need for long-term safety
assessments in gene therapy clinical
trials, and discern when to enroll
pediatric patients in gene therapy trials.
II. Topics for Discussion at the Public
Workshop
The workshop will feature
presentations and panel discussions on
hemophilia product development. The
presentations will include an overview
of product development in hemophilia,
and the regulatory challenges in the
development of novel hemophilia
therapies. Five sessions include
presentations to frame panel discussions
to cover the following topics:
1. Overview of product development
in hemophilia;
2. Efficacy endpoints related to
bleeding outcomes and considerations
for factor activity as a surrogate
endpoint;
3. Patient and caregiver perspectives
on developing outcomes for clinical
trials;
4. Discrepancies in the factor activity
measurements by different assays
observed in gene therapy trials and root
causes for the discrepancies; and
5. Clinical trial design considerations
for follow up on safety, efficacy,
enrollment of pediatric patients in gene
therapy trials, and the applicability of
on-demand treatment as a control group
in the evolving landscape of treatment
options in hemophilia.
III. Participating in the Public
Workshop
Registration: Persons interested in
attending this public workshop must
register online at https://
fdaoce.formstack.com/forms/
pdh120618 before 5 p.m. on December
3, 2018. Please provide complete
contact information for each attendee,
including name, title, affiliation,
address, email, and telephone.
Registration is free and based on
space availability, with priority given to
early registrants. Early registration is
recommended because seating is
limited; therefore, FDA may limit the
number of participants from each
organization. Registrants will receive
confirmation when they have been
accepted.
If you need special accommodations
due to a disability, please contact Joan
Ferlo Todd at Joan.Todd@fda.hhs.gov
no later than 5 p.m., on November 21,
2018.
Streaming Webcast of the Public
Workshop: This public workshop will
also be web-streamed on the day of the
workshop.
VerDate Sep<11>2014
17:57 Nov 01, 2018
Jkt 247001
If you have never attended a webcast
event before, test your connection at
https://collaboration.fda.gov/common/
help/en/support/meeting_test.htm. To
get a quick overview of the Adobe
webcast program, visit https://
www.adobe.com/go/connectpro_
overview. FDA has verified the website
addresses in this document, as of the
date this document publishes in the
Federal Register, but websites are
subject to change over time.
Transcripts: Please be advised that as
soon as a transcript of the public
workshop is available, it will be
accessible at https://
www.regulations.gov. It may be viewed
at the Dockets Management Staff (see
ADDRESSES). A link to the transcript will
be available on the internet at https://
www.fda.gov/NewsEvents/Meetings
ConferencesWorkshops/
ucm620602.htm.
Dated: October 29, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–23947 Filed 11–1–18; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Health Resources and Services
Administration
55189
to provide T/TA addressing the critical
health needs of MSAW in alignment
with HRSA priorities. T/TA provided at
the Migrant Stream Forums is targeted
to a broad range of health center staff
positions, and covers diverse topics that
address the needs of migrant health
centers and the patients they serve.
Supplemental funds are necessary to
support their timely and successful
implementation.
This supplemental funding will
augment the current NCA investment
for these T/TA opportunities through
support of enhanced personnel
presence, the availability of continuing
education unit-bearing educational
sessions to meet the diverse needs of
multidisciplinary health center staff,
and speaker and participant stipends
that underscore the unique value these
in-person regional T/TA sessions
provide.
FOR FURTHER INFORMATION CONTACT:
Tracey Orloff, Strategic Partnerships
Division Director in the Bureau of
Primary Health Care, Office of Quality
Improvement, at TOrloff@hrsa.gov.
Dated: October 26, 2018.
George Sigounas,
Administrator.
[FR Doc. 2018–24008 Filed 11–1–18; 8:45 am]
BILLING CODE 4165–15–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Health Center Program
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services.
ACTION: Announcement of Supplemental
Award.
AGENCY:
HRSA provided supplemental
grant funds to a currently funded
National Training and Technical
Assistance Cooperative Agreement
(NCA) award recipient to coordinate
and provide training and technical
assistance (T/TA) to health centers that
serve migrant and seasonal agricultural
workers (MSAW) and their families
through three regional forums.
SUPPLEMENTARY INFORMATION:
Recipient of the Award: The National
Center for Farmworker Health, Inc.
Amount of Non-Competitive Awards:
$150,000.
Period of Supplemental Funding:
Fiscal years 2018 and 2019 (contingent
upon available funding and satisfactory
performance).
CFDA Number: 93.129.
Authority: Section 330(l) of the Public
Health Service Act, as amended.
JUSTIFICATION: The award recipient will
lead the coordination and management
of three regional Migrant Stream Forums
SUMMARY:
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Fmt 4703
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National Institutes of Health
Eunice Kennedy Shriver National
Institute of Child Health & Human
Development; Notice of Closed
Meeting
Pursuant to section 10(d) of the
Federal Advisory Committee Act, as
amended, notice is hereby given of the
following meeting.
The meeting will be closed to the
public in accordance with the
provisions set forth in sections
552b(c)(4) and 552b(c)(6), Title 5 U.S.C.,
as amended. The grant applications and
the discussions could disclose
confidential trade secrets or commercial
property such as patentable material,
and personal information concerning
individuals associated with the grant
applications, the disclosure of which
would constitute a clearly unwarranted
invasion of personal privacy.
Name of Committee: National Institute of
Child Health and Human Development
Special Emphasis Panel.
Date: November 20, 2018.
Time: 1:00 p.m. to 3:00 p.m.
Agenda: To review and evaluate grant
applications.
E:\FR\FM\02NON1.SGM
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]]>Agencies
[Federal Register Volume 83, Number 213 (Friday, November 2, 2018)]
[Notices]
[Pages 55188-55189]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-23947]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-N-3693]
Product Development in Hemophilia; Public Workshop
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is
announcing the following public workshop entitled ``Product Development
in Hemophilia.'' The purpose of the public workshop is to discuss
issues related to development and regulation of novel hemophilia
products.
DATES: The public workshop will be held on December 6, 2018, from 8:30
a.m. to 4:30 p.m. See the SUPPLEMENTARY INFORMATION section for
registration date and information.
ADDRESSES: The public workshop will be held at FDA's White Oak Campus,
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993-0002. Entrance for the public
workshop participants (non-FDA employees) is through Building 1 where
routine security check procedures will be performed. For parking and
security information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
Docket: For access to the docket to read background documents 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.
FOR FURTHER INFORMATION CONTACT: Joan Ferlo Todd, Food and Drug
Administration, Center for Drug Evaluation and Research, Office of
Hematology and Oncology Products, 10903 New Hampshire Ave., Bldg. 22,
Rm. 2139, Silver Spring, MD 20993-0002, 301-796-6079,
[email protected].
SUPPLEMENTARY INFORMATION:
I. Background
Hemophilia is a bleeding disorder caused by deficiency of
coagulation factor VIII (hemophilia A) or coagulation factor IX
(hemophilia B). Hemophilia treatment strategies are intended to prevent
or control bleeding and the attendant complications. Recently,
hemophilia treatment strategies have led to the development of factor
concentrates, recombinant DNA technology products, antibodies, and
potential curative strategies such as gene therapy. These new emerging
technologies raise new considerations about trial design, novel
endpoints, patient-reported outcomes, and long-term safety collection.
This public workshop is intended to provide a platform for engaging
in a discussion with experts in hemophilia treatment, patients, and
caregivers. The purpose of this workshop is to advance further
development of patient-experience and patient-reported outcomes for use
in clinical trials, facilitate reliable and interpretable measurements
of factor VIII/IX activity
[[Page 55189]]
levels for gene therapy products, discuss the need for long-term safety
assessments in gene therapy clinical trials, and discern when to enroll
pediatric patients in gene therapy trials.
II. Topics for Discussion at the Public Workshop
The workshop will feature presentations and panel discussions on
hemophilia product development. The presentations will include an
overview of product development in hemophilia, and the regulatory
challenges in the development of novel hemophilia therapies. Five
sessions include presentations to frame panel discussions to cover the
following topics:
1. Overview of product development in hemophilia;
2. Efficacy endpoints related to bleeding outcomes and
considerations for factor activity as a surrogate endpoint;
3. Patient and caregiver perspectives on developing outcomes for
clinical trials;
4. Discrepancies in the factor activity measurements by different
assays observed in gene therapy trials and root causes for the
discrepancies; and
5. Clinical trial design considerations for follow up on safety,
efficacy, enrollment of pediatric patients in gene therapy trials, and
the applicability of on-demand treatment as a control group in the
evolving landscape of treatment options in hemophilia.
III. Participating in the Public Workshop
Registration: Persons interested in attending this public workshop
must register online at https://fdaoce.formstack.com/forms/pdh120618
before 5 p.m. on December 3, 2018. Please provide complete contact
information for each attendee, including name, title, affiliation,
address, email, and telephone.
Registration is free and based on space availability, with priority
given to early registrants. Early registration is recommended because
seating is limited; therefore, FDA may limit the number of participants
from each organization. Registrants will receive confirmation when they
have been accepted.
If you need special accommodations due to a disability, please
contact Joan Ferlo Todd at [email protected] no later than 5 p.m.,
on November 21, 2018.
Streaming Webcast of the Public Workshop: This public workshop will
also be web-streamed on the day of the workshop.
If you have never attended a webcast event before, test your
connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a quick overview of the Adobe webcast program,
visit https://www.adobe.com/go/connectpro_overview. FDA has verified
the website addresses in this document, as of the date this document
publishes in the Federal Register, but websites are subject to change
over time.
Transcripts: Please be advised that as soon as a transcript of the
public workshop is available, it will be accessible at https://www.regulations.gov. It may be viewed at the Dockets Management Staff
(see ADDRESSES). A link to the transcript will be available on the
internet at https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm620602.htm.
Dated: October 29, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-23947 Filed 11-1-18; 8:45 am]
BILLING CODE 4164-01-P
