[Federal Register Volume 79, Number 26 (Friday, February 7, 2014)]
[Notices]
[Pages 7464-7465]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-02629]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0967]


Pulmonary Arterial Hypertension 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 pulmonary arterial hypertension. 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 pulmonary arterial hypertension on daily 
life, as well as their perspectives on the available therapies for 
pulmonary arterial hypertension.

DATES: The public meeting will be held on May 13, 2014, from 1 p.m. to 
5 p.m. Registration to attend the meeting must be received by April 30, 
2014. See the SUPPLEMENTARY INFORMATION section for information on how 
to register for the meeting. Submit electronic or written comments by 
July 14, 2014.

ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903 
New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 
1503A), Silver Spring, MD 20993. Entrance for the public meeting 
participants (non-FDA employees) 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 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 meeting at 
http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm379694.htm.

FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1199, Silver Spring, MD 20993, 301-796-
5003, FAX: 301-847-8443, email: [email protected].

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

    FDA has selected pulmonary arterial hypertension as 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 V 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 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 (78 FR 21613) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years (FYs) 2013 through 2015, the first 3 years of the 5-year PDUFA V 
time frame. To develop the list of disease areas, the Agency used 
several criteria that were outlined in the April 11 notice. The Agency 
obtained 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 58849), and through a public meeting 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 FYs 2016 through 
2017. More information, including the list of disease areas and a 
general

[[Page 7465]]

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 obtain 
patient and patient stakeholder input on symptoms of pulmonary arterial 
hypertension that matter most to patients and on current approaches to 
treating pulmonary arterial hypertension. Pulmonary arterial 
hypertension is a rare, progressive condition that affects the heart 
and lungs. It is characterized by abnormally high blood pressure in the 
pulmonary artery and may be accompanied by shortness of breath, chest 
pain, fatigue, dizziness, fainting, lightheadedness, and swollen ankles 
and legs. There are several treatment options for pulmonary arterial 
hypertension, including medications, surgery, and lifestyle changes.
    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 written 
comments that can be submitted to 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 symptoms such as chest pain, 
shortness of breath, difficulty concentrating, and others.)
    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 may include activities such as household chores, 
walking up the stairs.)
     How do your symptoms and their negative impacts affect 
your daily life on the best days? On the worst days?
    3. How have your condition and its symptoms changed over time?
Topic 2: Patients' Perspectives on Current Approaches to Treating 
Pulmonary Arterial Hypertension
    1. What are you currently doing to help treat your condition or its 
symptoms? (Examples may include prescription medicines, over-the-
counter products, other therapies including non-drug therapies such as 
diet modification.)
     How has your treatment regimen changed over time, and why?
     How well does your current treatment regimen treat the 
most significant symptoms of your disease?
     Have the medications for pulmonary arterial hypertension 
made a difference to you? If so, in what ways?
    2. What are the most significant downsides to your current 
treatments, and how do they affect your daily life? (Examples may 
include downsides such as bothersome side effects, going to the 
hospital for treatment, and others.)
    3. Assuming there is no complete cure for your condition, what 
specific things would you look for in an ideal treatment for your 
condition?

B. Meeting Attendance and Participation

    If you wish to attend this meeting, visit https://patientfocusedpulmonaryarterialhypertension.eventbrite.com. Please 
register by April 30, 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 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 Graham 
Thompson (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. These patients will 
also be asked to send a brief summary of responses to the topic 
questions to [email protected]. We will notify panelists of 
their selection soon after the close of registration on April 30, 2014. 
We 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.
    Comments: Regardless of attendance at the public meeting, you can 
submit electronic or written responses to the questions pertaining to 
Topics 1 and 2 to the public docket (see ADDRESSES) by July 14, 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: As soon as a transcript is available, FDA will post it 
at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm379694.htm.

    Dated: January 30, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-02629 Filed 2-6-14; 8:45 am]
BILLING CODE 4160-01-P