[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