Patient-Focused Drug Development for Hereditary Angioedema; Public Meeting; Request for Comments, 33503-33505 [2017-15202]
Download as PDF
33503
Federal Register / Vol. 82, No. 138 / Thursday, July 20, 2017 / Notices
TABLE 3—RECORDKEEPING BURDEN 1—Continued
Number of
recordkeepers
Number of
records per
recordkeeper
Total records
Hours per record
..............................
........................
........................
..............................
DSCSA Pilot project program
Total .................................................................
1 There
Total hours
29
are no capital costs or operating and maintenance costs associated with this collection of information.
TABLE 4—THIRD-PARTY DISCLOSURE BURDEN 1
Coordination with partnering entities related to requests to participate ...................................................
Coordination with partnering entities related to
progress reports .........................................................
Coordination with partnering entities related to final reports ............................................................................
Total ........................................................................
1 There
[FR Doc. 2017–15203 Filed 7–19–17; 8:45 am]
BILLING CODE 4164–01–P
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
Food and Drug Administration,
HHS.
Notice of public meeting;
request for comments.
ACTION:
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.’’ PatientFocused 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
SUMMARY:
asabaliauskas on DSKBBXCHB2PROD with NOTICES
Total
disclosures
Hours per
disclosure
Total hours
8
2
16
8
128
7
10
70
4
280
7
2
14
20
280
..............................
........................
........................
........................
688
are no capital costs or operating and maintenance costs associated with this collection of information.
Dated: July 14, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning,
Legislation, and Analysis.
AGENCY:
Number of
disclosures
per
respondent
Number of
respondents
DSCSA pilot project program
VerDate Sep<11>2014
18:50 Jul 19, 2017
Jkt 241001
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,
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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
E:\FR\FM\20JYN1.SGM
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asabaliauskas on DSKBBXCHB2PROD with NOTICES
33504
Federal Register / Vol. 82, No. 138 / Thursday, July 20, 2017 / Notices
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/201523389.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 https://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,
VerDate Sep<11>2014
18:50 Jul 19, 2017
Jkt 241001
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 PatientFocused 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 https://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
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Sfmt 4703
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 selflimited; 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,
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Federal Register / Vol. 82, No. 138 / Thursday, July 20, 2017 / Notices
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?
asabaliauskas on DSKBBXCHB2PROD with NOTICES
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-thecounter products; and other therapies,
including non-drug therapies.
• What, if anything, do you selfadminister 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-thecounter 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
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18:50 Jul 19, 2017
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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-focuseddrug-development-for-hereditaryangioedema-public-meeting-tickets32300298061. 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 https://www.fda.gov/
BiologicsBloodVaccines/NewsEvents/
WorkshopsMeetingsConferences/
ucm542320.htm.
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33505
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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2017–N–3857]
Enhanced Drug Distribution Security
Under the Drug Supply Chain Security
Act; Public Meetings; Request for
Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice of public meetings;
request for comments.
ACTION:
The Food and Drug
Administration (FDA, the Agency, or
we) is announcing three public meetings
entitled ‘‘Enhanced Drug Distribution
Security Under the Drug Supply Chain
Security Act (DSCSA).’’ These public
meetings are intended to provide
members of the pharmaceutical
distribution supply chain and other
interested stakeholders an opportunity
to discuss with FDA, and provide input
on, strategies and issues related to the
enhanced drug distribution security
provisions of the DSCSA.
DATES: The public meetings will be held
on: August 23, 2017, from 9 a.m. to 4
p.m.; December 5 and 6, 2017, from 9
a.m. to 4 p.m.; and February 28, 2018,
from 9 a.m. to 4 p.m.
ADDRESSES: The public meetings will be
held at FDA’s White Oak Campus,
10903 New Hampshire Ave., Bldg. 31,
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 parking and security
information, please refer to https://
www.fda.gov/AboutFDA/
WorkingatFDA/BuildingsandFacilities/
WhiteOakCampusInformation/
ucm241740.htm.
Comments: To permit the widest
possible opportunity to obtain public
comment, FDA is soliciting either
electronic or written comments on all
aspects of the public meeting topics.
You may submit comments as follows.
Please note that the deadlines for
submitting either electronic or written
comments are 30 days after the meeting
to which the comments relate.
Comments received by mail/hand
delivery/courier (for written/paper
SUMMARY:
E:\FR\FM\20JYN1.SGM
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Agencies
[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
https://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 https://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 https://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