[Federal Register Volume 80, Number 104 (Monday, June 1, 2015)]
[Notices]
[Pages 31048-31050]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-13063]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-N-1798]


Patient-Focused Drug Development for Alpha-1 Antitrypsin 
Deficiency; Public Meeting

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 Alpha-1 Antitrypsin Deficiency (AATD). 
Patient-Focused Drug Development is an FDA performance commitment under 
the fifth authorization of the Prescription Drug User Fee Act (PDUFA 
V). The public meeting is intended to provide FDA with patients' 
perspectives on the impact on daily life of AATD. FDA also is seeking 
patients' perspectives on the available therapies for this disorder.

DATES: The public meeting will be held on September 29, 2015, from 9 
a.m. to 3:30 p.m. Registration to attend the meeting must be received 
by September 15, 2015. Registration from those individuals interested 
in presenting comments as part of the panel discussions should be 
received by July 31, 2015. See the SUPPLEMENTARY INFORMATION section 
for instructions on how to register for the meeting. Submit either 
electronic or written comments by November 30, 2015.

ADDRESSES: The public meeting will be held at the FDA White Oak Campus, 
10903 New Hampshire Ave., Building 31 Conference Center, the Great Room 
(rm. 1503), Silver Spring, MD 20993. Entrance for public meeting 
participants (non-FDA employees) is through Building 1, where routine 
security checks will be performed. For parking and security 
information, 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.

FOR FURTHER INFORMATION CONTACT: Barbara Kass, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, rm. 1125, Silver Spring, MD 20993, 240-402-
6887, FAX: 301-595-1243, email: [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background on Patient-Focused Drug Development

    FDA has selected AATD as the focus of a public meeting under the 
Patient-Focused Drug Development initiative. This initiative involves 
obtaining a better understanding of patients' perspectives on the 
challenges posed by AATD and the impact of current therapies for this 
condition. The Patient-Focused Drug Development initiative is being 
conducted to fulfill FDA performance commitments that are part of the 
PDUFA reauthorization 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 has committed to obtaining 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 benefits 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 in the Federal Register 
(78 FR 21613) that announced the disease areas for meetings in fiscal 
years (FY) 2013-2015, the first 3 years of the 5-year PDUFA V 
timeframe. The Agency used several criteria outlined in the April 11, 
2013, 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. FDA has initiated a second public process for 
determining the disease areas for meetings in FY 2016-2017 and 
published a notice in the Federal Register on October 8, 2014 (79 FR 
60857). 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 the Meeting

    The purpose of this Patient-Focused Drug Development meeting is to 
obtain input on the symptoms and other impacts that matter most to 
patients with AATD. FDA also intends to seek patients' perspectives on 
current approaches to treating this disorder. FDA expects that this 
information will come directly from patients, caregivers, and patient 
advocates.
    Individuals with AATD have low serum levels of Alpha-1-Antitrypsin 
(AAT, also known as Alpha-1 proteinase inhibitor (A1-PI)) and increased 
risks of developing a form of chronic obstructive lung disease called 
emphysema and, less frequently, liver disease. Some AATD patients with 
emphysema have symptoms of asthma. There are different genetic forms of 
the disease, but even among people with the same genetic form and 
similar levels of AAT in their blood, there is tremendous diversity in

[[Page 31049]]

clinical severity. A substantial percentage of individuals with severe 
AATD never develop symptomatic lung disease during their lifetimes. 
Others may develop the first signs and symptoms of lung disease between 
the ages of approximately 25 and 50 years, or older. Affected 
individuals often develop emphysema, which is a lung disease caused by 
damage to the small air sacs in the lung. Progression of emphysema in 
AATD may lead to respiratory failure, a need for lung transplantation, 
and eventually death. The only specific medication approved for raising 
the blood levels of AAT in severe AATD patients with emphysema is 
weekly intravenous treatment with A1-PI (Human) purified from human 
blood plasma.
    Severe AATD patients may also develop liver disease as infants, 
during childhood, or as adults. There is wide variation in the severity 
of liver disease among affected patients. Currently, no specific 
therapy for AATD-related liver disease is available other than liver 
transplantation, so the focus in these patients is on the prevention 
and management of other types of liver damage.
    The questions that will be asked of patients and patient caregivers 
at the meeting are provided in this document. The meeting will be 
divided into two main topics: (1) The effects of Alpha-1 Antitrypsin 
Deficiency that matter most to you, and (2) perspectives on current 
approaches to treatment. For each topic, a brief patient panel 
discussion will begin the dialogue. This will be followed by a 
facilitated discussion inviting comments from other patient and patient 
caregiver participants. In addition to input generated through this 
public meeting, FDA is interested in receiving patient input addressing 
these questions through electronic or written comments, which can be 
submitted to the Division of Dockets Management (see ADDRESSES). For 
context, please indicate if you are commenting as a patient with AATD 
or on behalf of a child or loved one.
    Topic 1: The effects of Alpha-1 Antitrypsin Deficiency that matter 
most to you.
     Of all of 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:
    (a) For lung disease: shortness of breath during specific 
activities or at rest, chronic cough, wheezing, weight loss, 
exacerbations of particular symptoms;
    (b) For liver disease: abdominal pain, loss of appetite, height and 
weight concerns.)
     Are there specific activities that are important to you, 
but that you cannot do at all, or as well as you would like, because of 
your condition? Please describe, using specific examples. (Examples may 
include: Participating in physical activities, attending work/school 
and family/social activities.)
     How have your condition and its symptoms changed over 
time?
     What worries you most about your condition?
    Topic 2: Perspectives on current approaches to treatment.
     What are you currently doing to treat your condition or 
its symptoms? (Examples may include:
    (a) For lung disease: Inhaled bronchodilators, inhaled 
corticosteroids, intravenous augmentation therapy with A1-PI (Human) on 
a regular or intermittent basis;
    (b) For liver disease: Ursodiol.)
    [cir] How well do these treatments work for you?
    [cir] What are the most significant disadvantages or complications 
of your current treatments, and how do they affect your daily life?
    [cir] How has your treatment changed over time and why?
    [cir] What aspects of your condition are not improved by your 
current treatment regimen?
    [cir] What treatment has had the most positive impact on your life?
     If you could create your ideal treatment, what would it do 
for you (i.e., what specific things would you look for in an ideal 
treatment)?
     If you had the opportunity to consider participating in a 
clinical trial studying experimental treatments, what things would you 
consider when deciding whether or not to participate?

III. Attendance and Registration

    If you wish to attend this meeting, visit https://www.eventbrite.com/e/public-meeting-on-patient-focused-drug-development-for-alpha-1-antitrypsin-deficiency-tickets-15617092143. If 
you do not have access to the Internet, you may mail or fax your 
registration information (including name, title, affiliation, address, 
email address, telephone, and fax numbers) to Barbara Kass (see FOR 
FURTHER INFORMATION CONTACT) by September 15, 2015. There is no 
registration fee for the public meeting. Early registration is 
recommended because seating is limited. FDA may limit the number of 
participants from each organization based on space limitations. 
Registrants will receive confirmation once they have been accepted. 
Registration on the day of the public meeting will be provided on a 
space available basis beginning at 8 a.m. Those who are unable to 
attend the meeting in person 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.
    If you need special accommodations because of disability, please 
contact Barbara Kass at least 7 days in advance. FDA will post the 
agenda approximately 5 days before the meeting at http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/MeetingsMeetingsConferences/ucm435242.htm.

IV. Comments

    Patients and patient caregivers who are interested in presenting 
comments as part of the panel discussions should register by July 31, 
2015. You will be asked to indicate in your registration which topic(s) 
you wish to address and to send a brief summary of responses to the 
topic questions to [email protected] by July 31, 2015. 
Panelists will be notified of their selection soon after August 28, 
2015. FDA will try to accommodate all patients and patient caregivers 
who wish to speak, either through the panel discussion or audience 
participation; however, the duration of comments may be limited by time 
constraints.
    Regardless of attendance at the public meeting, interested persons 
may submit either electronic or written responses to any or all of the 
questions pertaining to topics 1 and 2 to the Division of Dockets 
Management (see ADDRESSES). It is only necessary to send one set of 
comments. Identify comments with the docket number found in brackets in 
the heading of this document. To ensure consideration, submit comments 
by November 30, 2015. 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

    Please be advised that as soon as a transcript is available, it 
will be accessible at http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm435242.htm and at http://www.regulations.gov. It may also be viewed at the Division of Dockets 
Management (see ADDRESSES). A transcript will also be available in 
either hardcopy or on CD-ROM, after submission of a Freedom of 
Information request. Written requests are to be sent

[[Page 31050]]

to the Division of Freedom of Information (ELEM-1029), Food and Drug 
Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857.

    Dated: May 26, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-13063 Filed 5-29-15; 8:45 am]
 BILLING CODE 4164-01-P