[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