[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]
 BILLING CODE 4164-01-P


