Document ID: FDA-2017-N-3068-0001
Agency: fda
Document Type: Notice
Title: Patient-Focused Drug Development for Hereditary Angioedema; Public
Meeting; Request for Comments
Posted Date: 2017-07-20T04:00Z

[Federal Register Volume 82, Number 138 (Thursday, July 20, 2017)]
[Notices]
[Pages 33503-33505]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-15202]

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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-3068]

Patient-Focused Drug Development for Hereditary Angioedema; 
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, the Agency, or we) is 
announcing a public meeting and an opportunity for public comment on 
``Patient-Focused Drug Development for Hereditary Angioedema.'' 
Patient-Focused Drug Development is part of FDA's performance 
commitment under 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 hereditary angioedema 
(HAE) on daily life. FDA also is seeking patients' views on treatment 
approaches for HAE.

DATES: The public meeting will be held on September 25, 2017, from 9 
a.m. to 3 p.m. Registration to attend must be received by August 10, 
2017. Submit either electronic or written comments on the public 
meeting by November 20, 2017. See the SUPPLEMENTARY INFORMATION section 
for registration date and information.

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. Entrance for the public meeting 
participants (non-FDA employees) is through Building 1, where routine 
security check procedures will be performed. For parking and security 
information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    You may submit comments as follows. Please note that late, untimely 
filed comments will not be considered. Electronic comments must be 
submitted on or before November 20, 2017. The https://www.regulations.gov electronic filing system will accept comments until 
midnight Eastern Time at the end of November 20, 2017. Comments 
received by mail/hand delivery/courier (for written/paper submissions) 
will be considered timely if they are postmarked or the delivery 
service acceptance receipt is on or before that date.

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Since your comment will be made 
public, you are solely responsible for ensuring that your comment does 
not include any confidential information that you or a third party may 
not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2017-N-3068 for ``Patient-Focused Drug

[[Page 33504]]

Development for Hereditary Angioedema.'' Received comments, those filed 
in a timely manner (see ADDRESSES), will be placed in the docket and, 
except for those submitted as ``Confidential Submissions,'' publicly 
viewable at https://www.regulations.gov or at the Dockets Management 
Staff between 9 a.m. and 4 p.m., Monday through Friday.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on https://www.regulations.gov. 
Submit both copies to the Dockets Management Staff. If you do not wish 
your name and contact information to be made publicly available, you 
can provide this information on the cover sheet and not in the body of 
your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR 56469, September 18, 2015, or 
access the information at: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.
    FDA will post the agenda approximately 5 days before the meeting at 
http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm542319.htm.

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; or Loni Warren Henderson, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
71, Rm. 1118, Silver Spring, MD 20993, 240-402-8180, 
PatientFocused_CBER@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: 

I. Background on Patient-Focused Drug Development

    FDA has selected HAE 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 HAE 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 committed to obtaining the patient perspective on 20 disease 
areas during the course of PDUFA V. For each disease area, the Agency 
is conducting 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), announcing the disease areas for meetings in fiscal 
years (FYs) 2013-2015, the first 3 years of the 5-year PDUFA V time 
frame. The Agency used several criteria outlined in that 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 
initiated a second public process for determining the disease areas for 
FY 2016-2017 and published a notice in the Federal Register on July 2, 
2015 (80 FR 38216), announcing the selection of eight disease areas. 
More information, including the list of disease areas and a general 
schedule of meetings, is posted at https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm.

II. Purpose and Scope of the Meeting

    As part of the Patient-Focused Drug Development, FDA will obtain 
input on the symptoms and other aspects of the disease that matter most 
to patients with HAE. FDA also intends to seek patients' perspectives 
on current approaches to treating HAE. FDA expects that this 
information will come directly from patients, caregivers, and patient 
advocates.
    HAE is a rare genetic disorder that affects less than 200,000 
individuals in the United States. It is associated with episodic 
recurrent attacks of swelling of the body caused by abnormalities in a 
protein called C1-Esterase Inhibitor. Most cases occur because there is 
either not enough of the protein or because the protein does not work 
normally to help prevent swelling of the body.
    In individuals with HAE, the swelling attacks may involve various 
areas of the body, including the gastrointestinal tract, arms, legs, 
face, or throat and larynx (voice box). Symptoms of this condition 
often begin during childhood but may also appear in adulthood. The 
swelling episodes are usually self-limited; may or may not be 
associated with any triggering factors; and in severe cases involving 
the larynx, may be life-threatening. If not recognized early and left 
untreated, swelling of the larynx, called laryngeal edema, may acutely 
restrict airflow to the lungs and could result in death. 
Gastrointestinal tract swellings are often associated with nausea, 
vomiting, and abdominal pain, which can be severe and require 
hospitalization. Several FDA-approved therapies affecting different 
biological mechanisms are available to treat or prevent acute attacks 
of HAE.
    The questions that will be asked of patients and patient 
representatives at the meeting are listed in this section and organized 
by topic. The two main topics for discussion are: (1) Symptoms and 
impact on activities of daily life that matter most to patients; and 
(2) perspectives on current approaches to treatment. For each topic, a 
brief patient/caregiver panel discussion will begin the dialogue. This 
will be followed by a facilitated discussion, inviting comments from 
other patient and 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,

[[Page 33505]]

which can be submitted to the Dockets Management Staff (see ADDRESSES). 
For context, please indicate if you are commenting as a patient with 
HAE or on behalf of a child or loved one.

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

    (1) Of all of the symptoms that you experience because of your 
condition, which one of these symptoms has the most significant impact 
on your life? Examples may include nausea, vomiting, abdominal pain, 
swelling of extremities, facial swelling, tongue swelling, hoarseness 
or loss of voice, shortness of breath, and difficulty urinating.
    (2) Are there specific activities that are important to you 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; and attending work or 
school and family or social activities, during or between attacks.
    (3) How have your condition and its symptoms changed over time?
    (4) What worries you most about your condition?

Topic 2: Patients' Perspectives on Current Approaches to Treatment

    (1) What are you currently doing to treat your condition and its 
symptoms?
     What, if anything, are you doing to prevent acute HAE 
attacks? Examples may include treatments with prescription medicines; 
over-the-counter products; and other therapies, including non-drug 
therapies.
     What, if anything, do you self-administer for acute HAE 
attacks?
     If you give yourself medication for acute HAE attacks, 
which types of attacks, with respect to body location(s), are you 
comfortable treating yourself?
     What treatment has your health professional used for your 
acute HAE attacks? Examples may include prescription medicines; over-
the-counter products; and other therapies, including non-drug 
therapies.
    (2) How well do these treatments work for you?
    (3) What are the most significant disadvantages or complications of 
your current treatments, and how do they affect your daily life?
    (4) How has your treatment regimen changed over time and why?
    (5) What aspects of your condition are not improved by your current 
treatment regimen?
    (6) What treatment has had the most positive impact on your quality 
of life?
    (7) Short of a complete cure for your condition, what specific 
things would you look for in an ideal treatment for your condition?
    (8) 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. Meeting Attendance and Participation

    Registration: If you wish to attend this meeting, visit https://www.eventbrite.com/e/patient-focused-drug-development-for-hereditary-angioedema-public-meeting-tickets-32300298061. Persons interested in 
attending this public meeting must register by August 10, 2017. If you 
are unable to attend the meeting in person, you can register to view a 
live Webcast of the meeting. You will be asked to indicate in your 
registration if you plan to attend in person or via the Webcast. 
Registration is free and based on space availability, with priority 
given to early registrants. Early registration is recommended because 
seating is limited; therefore, FDA may limit the number of participants 
from each organization. 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 due 
to a disability, please contact Barbara Kass or Loni Warren Henderson 
(see FOR FURTHER INFORMATION CONTACT) no later than September 18, 2017.
    Requests for Oral Presentations: Patients and patient 
representatives 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 and 
patient representatives also must send to 
PatientFocused_CBER@fda.hhs.gov a brief summary of responses to the 
topic questions by August 3, 2017. Panelists will be notified of their 
selection approximately 7 days before the public meeting. We will try 
to accommodate all patients and patient representatives who wish to 
speak, either through the panel discussion or audience participation; 
however, the duration of comments may be limited by time constraints.
    Transcripts: Please be advised that, as soon as a transcript of the 
public meeting is available, it will be accessible at https://www.regulations.gov. It may be viewed at the Dockets Management Staff 
(see ADDRESSES). A link to the transcript will also be available on the 
Internet at http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm542320.htm.

    Dated: July 13, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning, Legislation, and Analysis.
[FR Doc. 2017-15202 Filed 7-19-17; 8:45 am]
 BILLING CODE 4164-01-P