[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]
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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.
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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]
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