[Federal Register Volume 78, Number 139 (Friday, July 19, 2013)]
[Notices]
[Pages 43209-43210]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-17327]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2013-N-0815]
Narcolepsy Public Meeting on Patient-Focused Drug Development
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public meeting; request for comments.
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SUMMARY: The Food and Drug Administration (FDA) is announcing a public
meeting and an opportunity for public comment on Patient-Focused Drug
Development for narcolepsy. Patient-Focused Drug Development is part of
FDA's performance commitments in the fifth authorization of the
Prescription Drug User Fee Act (PDUFA V). The public meeting is
intended to allow FDA to obtain patients' perspectives on the impact of
narcolepsy on daily life as well as the available therapies for
narcolepsy.
DATES: The public meeting will be held on September 24, 2013, from 1
p.m. to 5 p.m. Registration to attend the meeting must be received by
September 13, 2013. See the SUPPLEMENTARY INFORMATION section for
information on how to register for the meeting. Submit electronic or
written comments by November 25, 2013.
ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903
New Hampshire Ave., Building 31 Conference Center, Section A of the
Great Room (Rm. 1503), Silver Spring, MD 20993. Entrance for the public
meeting participants is through Building 1, where routine security
check procedures 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 electronic comments to 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 meeting
at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm359018.htm.
FOR FURTHER INFORMATION CONTACT: Pujita Vaidya, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 1170, Silver Spring, MD 20993, 301-796-
0684, FAX: 301-847-8443, email: [email protected].
SUPPLEMENTARY INFORMATION:
I. Background on Patient-Focused Drug Development
FDA has selected narcolepsy to be the focus of a meeting under
Patient-Focused Drug Development, an initiative that involves obtaining
a better understanding of patients' perspectives on the severity of the
disease and the available therapies for the condition. Patient-Focused
Drug Development is being conducted to fulfill FDA's performance
commitments made as part of the authorization of PDUFA under Title I of
the Food and Drug Safety and Innovation Act (FDASIA) (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 obtain the patient perspective in 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 benefit 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 community, and other interested
stakeholders.
On April 11, 2013, FDA published a notice in the Federal Register
(78 FR 22613) announcing the disease areas for meetings in fiscal years
(FY) 2013-2015, the first 3 years of the 5-year PDUFA V timeframe. To
develop the list of disease areas, the Agency used several criteria
that were outlined in the April 11 notice. The Agency gathered public
comment on these criteria and potential disease areas through a notice
for public comment published in the Federal Register on September 24,
2012 (77 FR 55849), and through a public meeting
[[Page 43210]]
held on October 25, 2012. In selecting the disease areas, FDA carefully
considered the public comments received and the perspectives of its
review divisions. By the end of FY 2015, FDA will initiate another
public process for determining the disease areas for 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
As part of Patient-Focused Drug Development, FDA will gather
patient and patient stakeholder input on symptoms of narcolepsy that
matter most to patients and on current approaches to treating
narcolepsy. Narcolepsy is a chronic disorder of the central nervous
system caused by the brain's inability to control sleep-wake cycles and
is characterized by excessive daytime sleepiness, cataplexy,
hallucination, and disturbed nocturnal sleep. Although there is no cure
for narcolepsy, medications and lifestyle modifications can help
patients manage their symptoms. FDA is interested in obtaining a better
understanding of patients' perspectives on the severity of the disease
and assessments of available therapies.
The questions that will be asked of patients and patient
stakeholders at the meeting are listed in this section, organized by
topic. For each topic, a brief patient panel discussion will begin the
dialogue, followed by a facilitated discussion inviting comments from
other patient and patient stakeholder participants. In addition to
input generated through this public meeting, FDA is interested in
receiving patient input addressing these questions through the public
docket (see ADDRESSES).
Topic 1: Disease symptoms and daily impacts that matter most to
patients:
1. Of all the symptoms that you experience because of your
condition, which one to three symptoms have the most significant impact
on your life? (Examples may include excessive daytime sleepiness,
cataplexy, etc.)
2. Are there specific activities that are important to you but that
you cannot do at all or as fully as you would like because of your
condition? (Examples of activities may include sleeping through the
night, work and school performance, etc.)
3. How have your symptoms changed over time?
3.1. Do your symptoms come and go? If so, do you know of anything
that makes your symptoms better? Worse?
Topic 2: Patients' perspectives on current approaches to treating
narcolepsy:
1. What are you currently doing to help treat your condition or its
symptoms? (Examples may include FDA-approved medicines, over-the-
counter products, and other therapies including non-drug therapies such
as lifestyle modifications.)
1.1. What specific symptoms do your therapies address?
1.2. How has your treatment regimen changed over time, and why?
2. How well does your current treatment regimen treat the most
significant symptoms of your disease?
2.1. How well do these therapies improve your ability to do
specific activities that are important to you in your daily life?
2.2. How well have these therapies worked for you as your condition
has changed over time?
3. What are the most significant downsides to your current
therapies, and how do they affect your daily life? (Examples of
downsides may include bothersome side effects, inconvenient dosing
schedules, access issues, etc.)
4. Assuming there is no complete cure for your condition, what
specific things would you look for in an ideal therapy for your
condition?
B. Meeting Attendance and/or Participation
If you wish to attend this meeting, visit http://patientfocusednarcolepsy.eventbrite.com. Please register by September
13, 2013. 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 whether you plan to attend in person or
via the webcast. Your registration should 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 Pujita
Vaidya (see FOR FURTHER INFORMATION CONTACT) at least 7 days before the
meeting.
Patients who are interested in presenting comments as part of the
initial panel discussions will be asked to indicate in their
registration which topic(s) they wish to address. They will also 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 the close of registration on September 13, 2013.
FDA will try to accommodate all patients and patient stakeholders who
wish to speak, either through the panel discussion or audience
participation; however, the duration of comments may be limited by time
constraints.
Interested members of the public, including those who attend the
meeting in person or through the webcast, are invited to provide
electronic or written responses to the questions pertaining to Topics 1
and 2 to the public docket (see ADDRESSES). Comments may be submitted
until November 25, 2013.
Dated: July 15, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-17327 Filed 7-18-13; 8:45 am]
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