[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]
BILLING CODE 4160-01-P