[Federal Register Volume 79, Number 131 (Wednesday, July 9, 2014)]
[Notices]
[Pages 38909-38910]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-15990]



[[Page 38909]]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0851]


Public Meeting on Patient-Focused Drug Development for Hemophilia 
A, Hemophilia B, von Willebrand Disease, and Other Heritable Bleeding 
Disorders

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

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    The Food and Drug Administration (FDA) is announcing a public 
meeting and an opportunity for public comment on Patient-Focused Drug 
Development for Hemophilia A, Hemophilia B, von Willebrand Disease, and 
other heritable bleeding disorders such as other factor deficiencies 
(including I, V, VII, X, XI) and platelet disorders. Patient-Focused 
Drug Development is an FDA performance commitment under the fifth 
authorization of the Prescription Drug User Fee Act (PDUFA V). The 
public meeting is intended to provide FDA with patients' perspectives 
on the impact on daily life of Hemophilia A, Hemophilia B, von 
Willebrand Disease, and other heritable bleeding disorders. FDA also is 
seeking patients' perspectives on the available therapies for these 
disorders.
    Dates and Time: The public meeting will be held on September 22, 
2014, from 9 a.m. to 5 p.m. Registration to attend the meeting must be 
received by September 12, 2014. Registration from those individuals 
interested in presenting comments as part of the panel discussions must 
be received by August 22, 2014 (see the SUPPLEMENTARY INFORMATION for 
instructions).
    Location: The meeting will be held at the FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room 
(Rm. 1503 B and C), Silver Spring, MD 20993. Entrance for public 
meeting participants (non-FDA employees) is through Building 1, where 
routine security checks will be performed. For more information on 
parking and security procedures, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Submit either electronic or written comments by November 28, 2014. 
Submit electronic comments to http://www.regulations.gov. Submit 
written comments to the Division of Dockets Management (HFA-305), Food 
and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 
20852. All comments should be identified with the docket number found 
in brackets in the heading of this document.
    FDA will post the agenda approximately 5 days before the workshop 
at: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm401758.htm.
    Contact Person: Henry Allen, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
71, Rm. 1125, Silver Spring, MD 20993, 240-402-8001, FAX: 301-595-1243, 
email: [email protected].

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

    FDA has selected Hemophilia A, Hemophilia B, von Willebrand 
Disease, and other heritable bleeding disorders as the focus of a 
public meeting under the Patient-Focused Drug Development initiative. 
This initiative involves obtaining a better understanding of patients' 
perspectives on the challenges posed by these disorders, and the impact 
of therapies for these disorders. The Patient-Focused Drug Development 
initiative is being conducted to fulfill FDA performance commitments 
that are part of the PDUFA reauthorization under Title I of the Food 
and Drug Safety and Innovation Act (Pub. L. 112-144). The full set of 
performance commitments is available on the FDA Web site at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.
    FDA has committed to obtaining the patient perspective on 20 
disease areas during the course of PDUFA V. For each disease area, the 
Agency will conduct a public meeting to discuss the disease and its 
impact on patients' daily lives, the types of treatment benefits that 
matter most to patients, and patients' perspectives on the adequacy of 
the available therapies. These meetings will include participation of 
FDA review divisions, the relevant patient communities, and other 
interested stakeholders.
    On April 11, 2013, FDA published a notice in the Federal Register 
(78 FR 21613) that announced the disease areas for meetings in fiscal 
years (FY) 2013-2015, the first 3 years of the 5-year PDUFA V 
timeframe. The Agency used several criteria outlined in the April 11, 
2013, notice to develop the list of disease areas. FDA obtained public 
comment on the Agency's proposed criteria and potential disease areas 
through a public docket and a public meeting that was convened on 
October 25, 2012. In selecting the set of disease areas, FDA carefully 
considered the public comments received and the perspectives of review 
divisions at FDA. By the end of FY 2015, FDA will initiate a second 
public process for determining the disease areas for meetings in FY 
2016-2017. More information, including the list of disease areas and a 
general schedule of meetings, is posted on FDA's Web site at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm.

II. Public Meeting Information

A. Purpose and Scope of the Meeting

    The purpose of this Patient-Focused Drug Development meeting is to 
obtain input on the symptoms and other impacts that matter most to 
patients with Hemophilia A, Hemophilia B, von Willebrand Disease, and 
other heritable bleeding disorders. FDA also intends to seek patients' 
perspectives on current approaches to treating these disorders. FDA 
expects that this information will come directly from patients, 
caregivers, and patient advocates.
    Heritable bleeding disorders are a diverse group of diseases and 
some involve lifelong defects in the clotting mechanism of the blood. 
The most frequently occurring of these disorders include Hemophilia A, 
Hemophilia B, and von Willebrand Disease. Less frequent yet also 
serious heritable bleeding disorders include Factor VII deficiency, 
Factor XIII deficiency, [alpha]2-antiplasmin deficiency and platelet 
disorders such as Gray platelet syndrome. Symptoms of heritable 
bleeding disorders include frequent nose bleed; prolonged and heavy 
menstrual bleeding; prolonged bleeding from cuts, trauma, dental 
extractions, and surgical procedures as well as bleeding into internal 
organs, muscles, and joints. Intracranial hemorrhage is a particularly 
serious and life-threatening manifestation. Specific treatment 
recommendations are determined by the type and severity of the 
disorder; but in general, therapies such as factor replacement, 
platelet transfusion, fresh frozen plasma, and cryoprecipitate are 
utilized.
    The questions that will be asked of patients and patient 
stakeholders at the meeting are provided in this document. For each 
topic, a brief patient panel discussion will begin the dialogue. This 
will be followed by a facilitated discussion inviting comments from 
other patient and patient stakeholder

[[Page 38910]]

participants. In addition to input generated through this public 
meeting, FDA is interested in receiving patient input addressing these 
questions through electronic or written comments, which can be 
submitted to the Division of Dockets Management (see Location). For 
context, please indicate if you are commenting as a patient with a 
heritable bleeding disorder or on behalf of a child or loved one.
Topic 1: The effects of your bleeding disorder that matter most to you
     Of all of the symptoms that you experience because of your 
condition, which one to three symptoms (bleeding or non-bleeding) have 
the most significant impact on your life? (Examples may include joint 
damage/pain, infections, prolonged and heavy bleeding with 
menstruation, fatigue, etc.)
     Are there specific activities that are important to you, 
but that you cannot do at all, or as well as you would like, because of 
your condition? Please describe, using specific examples. (Examples may 
include participating in physical activities, attending work/school, 
and family/social activities, etc.)
     How have your condition and its symptoms changed over 
time?
     What worries you most about your condition?
Topic 2: Perspectives on current approaches to treatment
     What are you currently doing to treat your condition or 
its symptoms? (Examples may include blood transfusions, replacement 
therapies, over-the-counter products, and/or other therapies).
    [cir] How well do these treatments work for you?
    [cir] What are the most significant disadvantages or complications 
of your current treatments, and how do they affect your daily life?
    [cir] How has your treatment changed over time and why?
    [cir] What aspects of your condition are not improved by your 
current treatment regimen?
    [cir] What treatment has had the most positive impact on your life?
     If you could create your ideal treatment, what would it do 
for you (i.e., what specific things would you look for in an ideal 
treatment)?
     If you had the opportunity to consider participating in a 
clinical trial studying experimental treatments, what things would you 
consider when deciding whether or not to participate?

B. Attendance and/or Participation in the Meeting

    If you wish to attend this meeting, visit https://www.eventbrite.com/e/patient-focused-public-meeting-on-heritable-bleeding-disorders-registration-11996980291. Please register by 
September 12, 2014. Those who are unable to attend the meeting in 
person can register to view a live Webcast of the meeting. You will be 
asked to indicate in your registration if you plan to attend in person 
or via the Webcast. Your registration will also contain your complete 
contact information, including name, title, affiliation, address, email 
address, and phone number. Seating will be limited, so early 
registration is recommended. Registration is free and will be on a 
first-come, first-served basis. However, FDA may limit the number of 
participants from each organization based on space limitations. 
Registrants will receive confirmation once they have been accepted. 
Onsite registration on the day of the meeting will be based on space 
availability. If you need special accommodations because of disability, 
please contact Henry Allen (see Contact Person) at least 7 days before 
the meeting.
    Patients and patient stakeholders who are interested in presenting 
comments as part of the initial panel discussions should register by 
August 22, 2014. You will be asked to indicate in your registration 
which topic(s) you wish to address. You will be asked to send a brief 
summary of responses to the topic questions to [email protected]. Panelists will be notified of their selection soon 
after August 22, 2014. FDA will try to accommodate all patients and 
patient advocate participants who wish to speak, either through the 
panel discussion or audience participation; however, the duration of 
comments may be limited by time constraints.
    Comments: Interested members of the public, including those who 
attend the meeting in person or via the Webcast, are invited to provide 
electronic or written responses to any or all of the questions 
pertaining to topics 1 and 2 to the Division of Dockets Management (see 
Location). Comments may be submitted until November 28, 2014. Received 
comments may be seen in the Division of Dockets Management between 9 
a.m. and 4 p.m., Monday through Friday, and will be posted to the 
docket at http://www.regulations.gov.
    Transcripts: Please be advised that as soon as a transcript is 
available, it will be accessible at http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm401761.htm and at http://www.regulations.gov. It may be viewed at 
the Division of Dockets Management (see Location). A transcript will 
also be available in either hardcopy or on CD-ROM, after submission of 
a Freedom of Information request. Written requests are to be sent to 
the Division of Freedom of Information (ELEM-1029), Food and Drug 
Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857.

    Dated: July 2, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-15990 Filed 7-8-14; 8:45 am]
BILLING CODE 4164-01-P