[Federal Register Volume 79, Number 130 (Tuesday, July 8, 2014)]
[Notices]
[Pages 38539-38541]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-15871]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-N-2014-0865]


Patient-Focused Drug Development for Idiopathic Pulmonary 
Fibrosis; Public Meeting; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
a public meeting and an opportunity for public comment on Patient-
Focused Drug Development for idiopathic pulmonary fibrosis. Patient-
Focused Drug Development is part of FDA's performance commitments made 
as part of the fifth authorization of the Prescription Drug User Fee 
Act (PDUFA V). The public meeting is intended to allow FDA to obtain 
patient perspectives on the impact of idiopathic pulmonary fibrosis on 
daily life as well as patient views on treatment approaches for 
idiopathic pulmonary fibrosis.

DATES: The public meeting will be held on September 26, 2014, from 1 
p.m. to 5 p.m. Registration to attend the meeting

[[Page 38540]]

must be received by September 10, 2014 (see SUPPLEMENTARY INFORMATION 
for instructions). Submit electronic or written comments by November 
26, 2014.

ADDRESSES: The public meeting will be held at the FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room 
(Rm. 1503), Silver Spring, MD 20993-0002. Participants must enter 
through Building 1 and undergo security screening. 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/ucm395774.htm.

FOR FURTHER INFORMATION CONTACT: Pujita Vaidya, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1144, Silver Spring, MD 20993, 301-796-
0684, FAX: 301-847-8443, email: [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA has selected idiopathic pulmonary fibrosis as the focus of a 
public meeting under Patient-Focused Drug Development, an initiative 
that involves obtaining a better understanding of patient perspectives 
on the severity of a disease and the available therapies for that 
condition. Patient-Focused Drug Development is being conducted to 
fulfill FDA performance commitments that are part of the 
reauthorization of PDUFA under Title I of the Food and Drug 
Administration 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 committed to obtain 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 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 communities, and other 
interested stakeholders.
    On April 11, 2013, FDA published a notice (78 FR 08441) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years (FY) 2013-2015, the first 3 years of the 5-year PDUFA V time 
frame. The Agency used several criteria outlined in the April 11 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 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. Purpose and Scope of Meeting

    The purpose of this Patient-Focused Drug Development meeting is to 
obtain input on the symptoms and other impacts of idiopathic pulmonary 
fibrosis that matter most to patients, as well as perspectives on 
current approaches to treating idiopathic pulmonary fibrosis. FDA 
expects that this information will come directly from patients, 
caregivers, and patient advocates. Idiopathic pulmonary fibrosis is a 
rare and life-threatening disease in which lung tissue become scarred 
over time. Many people with idiopathic pulmonary fibrosis survive only 
3 to 5 years from the time of diagnosis. Symptoms of idiopathic 
pulmonary fibrosis can include shortness of breath, dry cough, fatigue, 
and chest pain. There is no cure for idiopathic pulmonary fibrosis; 
symptomatic treatment options include corticosteroids, oxygen therapy, 
pulmonary rehabilitation, 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 initial patient panel discussion will 
begin the dialogue. This will be 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, which can be submitted to the 
public docket (see ADDRESSES).

Topic 1: Symptoms and Daily Impacts That Matter Most to Patients

     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 shortness of breath, cough, 
fatigue, etc.)
     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 household chores, 
walking up the stairs, etc.)
    [cir] How do your symptoms and their negative impacts affect your 
daily life on the best days?
    [cir] How do your symptoms and their negative impacts affect your 
daily life on the worst days?
     How has your condition and its symptoms changed over time?

Topic 2: Patient Perspectives on Treatment Approaches

     What are you currently doing to help treat your condition 
or its symptoms? (Examples may include prescription medicines, over-
the-counter products, and other therapies including non-drug therapies 
such as diet modification.) How well does your current treatment 
regimen treat the most significant symptoms of your disease?
     What are the most significant downsides to your current 
treatments and how do they affect your daily life? (Examples of 
downsides may include bothersome side effects, going to the hospital 
for treatment, etc.)
     Because there is no complete cure for your condition, what 
specific things would you look for in an ideal treatment for your 
condition?

III. Attendance and Participation

    If you wish to attend this meeting, visit http://patientfocusedIPF.eventbrite.com. Please register by September 10, 2014. If you are 
unable to attend the meeting in person, you can register to view a live 
Web cast of the meeting. You will be asked to indicate in your 
registration if you plan to attend in person or via the Web cast. 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

[[Page 38541]]

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 a 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 be asked to 
send a brief summary of responses to the topic questions to 
[email protected]. Panelists will be notified of their 
selection a few days after the close of registration on September 10, 
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.

IV. Comments

    Submit electronic or written responses to the questions pertaining 
to Topics 1 and 2 to the public docket (see ADDRESSES) by November 26, 
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.

V. Transcripts

    As soon as a transcript is available, FDA will post it at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm395774.htm.

    Dated: July 2, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-15871 Filed 7-7-14; 8:45 am]
BILLING CODE 4164-01-P