Complex Issues in Developing Medical Devices for Pediatric Patients Affected by Rare Diseases; Public Workshop; Request for Comments, 58316-58318 [2013-22960]
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Dated: September 16, 2013.
Jill Hartzler Warner,
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Medical Programs.
[FR Doc. 2013–23022 Filed 9–20–13; 8:45 am]
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DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2013–N–1073]
Complex Issues in Developing Medical
Devices for Pediatric Patients Affected
by Rare Diseases; Public Workshop;
Request for Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice of public workshop;
request for comments.
ACTION:
The Food and Drug Administration
(FDA) is announcing the following
public workshop entitled ‘‘Complex
Issues in Developing Medical Devices
for Pediatric Patients Affected by Rare
Diseases.’’ This public workshop is
organized by the Center for Devices and
Radiological Health (CDRH) and the
Office of Orphan Products Development
(OOPD) and is being held in
conjunction with the Center for Drug
Evaluation and Research’s workshop
entitled ‘‘Complex Issues in Developing
Drug and Biological Products for Rare
Diseases.’’ The purpose of the public
workshop is to discuss issues related to
the following broad topics associated
with medical devices for the diagnosis
and treatment of pediatric patients
affected by rare diseases: Current
approaches toward use of medical
devices for pediatric clinical practice;
Humanitarian Device Exemption (HDE)
marketing pathway, including the
Humanitarian Use Device (HUD)
designation process; Pediatric SpecialtySpecific Practice Areas; Clinical Trials
and Registries; and Pediatric Needs
Assessment and Possible Approaches to
Advancing Pediatric Medical Device
Development. FDA is seeking input into
these topics from academicians, clinical
practitioners, patients and advocacy
groups, industry, and governmental
agencies. The input from this public
workshop will help in developing a
strategic plan to encourage and
accelerate the development of new
medical devices and therapies for
pediatric patients affected by rare
diseases. This is part of an ongoing
effort by FDA to address the needs of
pediatric patients affected by rare
diseases.
Date and Time: The workshop will be
held on January 8, 2014, from 8 a.m. to
5 p.m. This public workshop is being
held in conjunction with FDA’s public
workshop entitled ‘‘Complex Issues in
Developing Drug and Biological
Products for Rare Diseases’’ which will
be held on January 6, 2014, from 8 a.m.
PO 00000
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Fmt 4703
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to 5 p.m. and on January 7, 2014, from
8 a.m. to 4:45 p.m.
Location: The public workshop will
be held at FDA White Oak Campus,
10903 New Hampshire Ave., Bldg. 31
Conference Center, the Great Room
(section A of Rm. 1503), Silver Spring,
MD 20993–0002. Entrance for the public
workshop 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.
Contact Person: Carol Krueger, Center
for Devices and Radiological Health,
Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 66, Rm.
3663, Silver Spring, MD 20993–0002,
301–796–3241, Carol.Krueger@
fda.hhs.gov.
Registration: Registration is free and
available on a first-come, first-served
basis. Persons interested in attending
the Complex Issues in Developing
Medical Devices for Pediatric Patients
Affected by Rare Diseases public
workshop must register online by
December 6, 2013, 5 p.m. Early
registration is recommended because
facilities are limited and, therefore, FDA
may limit the number of participants
from each organization. If time and
space permit, onsite registration on the
day of the public workshop will be
provided beginning at 7:30 a.m.
If you need special accommodations
due to a disability, please contact Susan
Monahan (email: Susan.Monahan@
fda.hhs.gov or phone: 301–796–5661) no
later than December 27, 2013.
To register for the public workshop,
please visit FDA’s Medical Devices
News & Events—Workshops &
Conferences calendar at https://
www.fda.gov/MedicalDevices/
NewsEvents/WorkshopsConferences/
default.htm. (Select this public
workshop from the posted events list.)
Please provide complete contact
information for each attendee, including
name, title, affiliation, address, email,
and telephone number. Those without
Internet access should contact Carol
Krueger to register (see Contact Person).
Registrants will receive confirmation
after they have been accepted. You will
be notified if you are on a waiting list.
Streaming Webcast of the Public
Workshop: This public workshop will
also be Webcast. Persons interested in
viewing the Webcast must register
online by December 6, 2013, 5 p.m.
Early registration is recommended
because Webcast connections are
limited. Organizations are requested to
E:\FR\FM\23SEN1.SGM
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emcdonald on DSK67QTVN1PROD with NOTICES
Federal Register / Vol. 78, No. 184 / Monday, September 23, 2013 / Notices
register all participants, but to view
using one connection per location.
Webcast participants will be sent
technical system requirements after
registration and will be sent connection
access information after January 1, 2014.
If you have never attended a Connect
Pro event before, test your connection at
https://collaboration.fda.gov/common/
help/en/support/meeting_test.htm. To
get a quick overview of the Connect Pro
program, visit https://www.adobe.com/
go/connectpro_overview. (FDA has
verified the Web site addresses in this
document, but FDA is not responsible
for any subsequent changes to the Web
sites after this document publishes in
the Federal Register.)
Comments: FDA is holding this public
workshop in response to section 510 of
the Food and Drug Safety and
Innovation Act to discuss ways to
encourage and accelerate the
development of new medical devices
and therapies for pediatric rare diseases.
In order to permit the widest possible
opportunity to obtain public comment,
FDA is soliciting either electronic or
written comments on all aspects of the
public workshop topics. The deadline
for submitting comments regarding this
public workshop is February 5, 2014.
Regardless of attendance at the public
workshop, interested persons may
submit either electronic comments
regarding this document to https://
www.regulations.gov or written
comments to the Division of Dockets
Management (HFA–305), Food and Drug
Administration, 5630 Fishers Lane, Rm.
1061, Rockville MD 20852. 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. In addition,
when responding to specific topics as
outlined in section II of this document,
please identify the topic you are
addressing. 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 https://
www.regulations.gov.
Transcripts: Please be advised that as
soon as a transcript is available, it will
be accessible at https://
www.regulations.gov. It may be viewed
at the Division of Dockets Management
(see Comments). 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 to the Division
of Freedom of Information (ELEM–
1029), Food and Drug Administration,
12420 Parklawn Dr., Element Bldg.,
Rockville, MD 20857. A link to the
transcripts will also be available
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approximately 45 days after the public
workshop on the Internet at https://
www.fda.gov/MedicalDevices/
NewsEvents/WorkshopsConferences/
default.htm. (Select this public
workshop from the posted events list).
SUPPLEMENTARY INFORMATION:
I. Background
The demand by health care
professionals and consumers for safe
and effective medical devices for use for
pediatric patients affected by rare
diseases continues to steadily increase.
To meet that demand, clinicians and
organizations representing patients and
physicians have cited the widespread
practice of modifying adult devices for
pediatric use. Certain adult medical
devices may be inappropriate for
pediatric use due to a variety of factors,
including patient size, growth, and
development, or may require design
changes or special labeling for pediatric
use.1
OOPD was established to promote the
development of products (drugs,
biologics, medical devices, or medical
foods) that demonstrate promise for the
diagnosis, prevention, and/or treatment
of rare diseases or conditions. One of
OOPD’s functions is to designate
devices as HUDs, which allows them to
be eligible for marketing approval under
an HDE application. The HDE pathway,
authorized under section 520(m) of the
Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360j(m)), provides an
alternative pathway to market devices
intended to treat or diagnose a disease
or condition that affects fewer than
4,000 individuals in the United States
per year. Roughly a quarter of the
medical devices that have received HDE
marketing approval are available for
pediatric patients.
In 2007, Congress passed the Pediatric
Medical Device Safety and Improvement
Act (the Act). The Act addresses
pediatric device needs by allowing
sponsors of pediatric HUDs to make a
profit on sales of those devices;
explicitly permitting extrapolation of
adult effectiveness data to support a
pediatric indication or extrapolation of
pediatric subpopulation effectiveness
data to support an indication for another
pediatric subpopulation based on a
similar course of the disease or
condition or a similar effect of the
device; and providing grants to pediatric
device consortia that provide technical
1 ’’Premarket
Assessment of Pediatric Medical
Devices.’’ Issued May 14, 2004. This guidance may
be found on FDA’s Web site at www.fda.gov/
downloads/MedicalDevices/
DeviceRegulationandGuidance/
GuidanceDocuments/UCM089742.pdf.
PO 00000
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support and assistance to pediatric
device innovators.
FDA is committed to supporting the
development and availability of safe and
effective medical devices for pediatric
patients affected by rare diseases. The
Agency has sponsored a number of
workshops on issues relevant to
pediatric device development in recent
years.
II. Topics for Discussion at the Public
Workshop
FDA seeks to address and receive
comments on the following topics:
A. Current Clinical Practice
1. The current use and practice trends
of medical devices in rare disease
pediatric populations. For example,
how much off-label use occurs? How
much modification and adaptation of
existing adult devices occurs?
2. What risks or adverse outcomes
have been reported in association with
off-label use of medical devices in rare
disease pediatric populations?
B. HUD/HDE
1. Is there any confusion about the
designation process for HUDs or the
application process for HDE’s? Where
have barriers been encountered in the
HDE marketing pathway, and how can
they be mitigated? Please provide
examples of any specific issues, how
frequently they occur and suggestions to
constructively address these barriers.
2. Please comment on Institutional
Review Board issues that arise for HDEs
that are indicated for pediatric rare
diseases.
C. Specialty Practice Areas
1. For specialty practices areas (e.g.
cardiology, orthopedics, and neurology)
what existing medical devices appear to
have the best potential for modification
for rare diseases that affect the pediatric
population? If possible, please prioritize
existing medical devices in terms of
minimal change, moderate change, or
significant change required. Also state
whether no medical device is currently
available to address the need.
2. What are the best ways to foster
efficient networking across agencies,
academia, professional societies, and
patient groups to address the medical
device needs of pediatric patients with
rare diseases?
D. Clinical Trials
1. What are the most challenging
barriers in the process of designing
protocols for devices used to treat/
diagnose rare pediatric diseases?
2. What are unique challenges in
identifying appropriate endpoints for
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Federal Register / Vol. 78, No. 184 / Monday, September 23, 2013 / Notices
protocols for devices used to treat/
diagnose rare pediatric diseases?
3. What barriers related to statistical
analyses must be addressed in order to
promote device development for rare
pediatric diseases?
4. How can new registries be
developed or current registries be
leveraged to provide robust data on the
safety and effectiveness of pediatric
medical devices to support premarket
approval and clearance, and/or enhance
postmarket surveillance activities
related to pediatric medical devices?
E. Pediatric Needs Assessment
1. Describe the parameters that should
be used in determining priority areas of
development of devices, including both
therapeutic and diagnostic devices, in
pediatric rare diseases.
2. What is the best approach to
conduct needs assessment of medical
devices required for use with pediatric
rare diseases?
F. Device Related Issues for Diagnostic
Devices
1. What are medical device related
issues that need to be addressed for
development of diagnostic medical
devices?
emcdonald on DSK67QTVN1PROD with NOTICES
G. Advancing Development
1. What incentives could help
advance the development of diagnostic
and therapeutic medical devices to treat
pediatric rare diseases?
2. How can possible or probable use
in pediatric practice be considered early
in the development stages of all devices
designed to treat a rare disease or
condition?
3. What are potential private
resources (e.g., registries, industry, or
patient advocacy groups) that could be
tapped to advance the development of
medical devices for rare diseases in the
pediatric population?
4. What are potential improvements
or changes that can be made to FDA
guidance, regulations, or current science
in order to help develop and improve
medical devices to address the needs of
the pediatric population affected by rare
diseases?
Dated: September 17, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013–22960 Filed 9–20–13; 8:45 am]
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DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2013–N–0001]
Clinical Trial Design for Intravenous
Fat Emulsion Products; Public
Workshop
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of public workshop.
The Food and Drug Administration’s
(FDA) Center for Drug Evaluation and
Research, in cosponsorship with the
American Society for Parenteral and
Enteral Nutrition, is announcing a 1-day
public workshop entitled ‘‘Clinical Trial
Design for Intravenous Fat Emulsion
Products.’’ This workshop will provide
a forum to discuss trial design of
clinical trials intended to support
registration of intravenous fat emulsion
products.
Date and Time: The public workshop
will be held on October 29, 2013, from
8 a.m. to 5 p.m. (EST).
Location: The public workshop 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 20993–0002.
Contact Person: Wes Ishihara, Center
for Drug Evaluation and Research, Food
and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD
20993–0002, 301–796–0069, FAX: 301–
796–9904, email: richard.ishihara@
fda.hhs.gov.
Registration: There is no fee to attend
the public workshop, but attendees
must register in advance. Space is
limited, and registration will be on a
first-come, first-served basis. Persons
interested in attending this workshop
must register online at https://
netforum.avectra.com/eweb/
DynamicPage.aspx?Site=ASPEN&
WebCode=EventDetail&evt_key=
eb9c4068-8b66-4ac0-ae4f-ac266c08e33e
before October 22, 2013. For those
without Internet access, please contact
Wes Ishihara (see Contact Person) to
register. On-site registration will not be
available.
If you need special accommodations
because of disability, please contact Wes
Ishihara (see Contact Person) at least 7
days in advance.
SUPPLEMENTARY INFORMATION: This
workshop will provide a forum to
discuss the key issues in clinical trial
design for intravenous fat emulsions.
Stakeholders, including industry
sponsors, academia, patients receiving
parenteral nutrition, and FDA, will
discuss challenging issues related to
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selection of endpoints and assessment
methodologies in registration trials.
Trial design strategies and possible
candidates for endpoints will be
explored.
Transcripts: Transcripts of the
workshop will be available for review at
the Division of Dockets Management
(HFA–305), Food and Drug
Administration, 5630 Fishers Lane, Rm.
1061, Rockville, MD 20852, and on the
Internet at https://www.regulations.gov
approximately 30 days after the
workshop. A transcript will also be
available in either hard copy or on CD–
ROM, after submission of a Freedom of
Information request. Send written
requests to the Division of Freedom of
Information (ELEM–1029), Food and
Drug Administration, 12420 Parklawn
Dr., Rockville, MD 20857. Send faxed
requests to 301–827–9267.
Dated: September 17, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013–23020 Filed 9–20–13; 8:45 am]
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National Institutes of Health
Submission for OMB Review; 30-day
Comment Request: The Framingham
Heart Study (FHS)
Under the provisions of
Section 3507(a)(1)(D) of the Paperwork
Reduction Act of 1995, the National
Institutes of Health (NIH) has submitted
to the Office of Management and Budget
(OMB) a request for review and
approval of the information collection
listed below. This proposed information
collection was previously published in
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pages 26639–41 and allowed 60-days for
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Direct Comments to OMB: Written
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Attention: NIH Desk Officer.
SUMMARY:
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]]>Agencies
[Federal Register Volume 78, Number 184 (Monday, September 23, 2013)]
[Notices]
[Pages 58316-58318]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-22960]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2013-N-1073]
Complex Issues in Developing Medical Devices for Pediatric
Patients Affected by Rare Diseases; Public Workshop; Request for
Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
-----------------------------------------------------------------------
The Food and Drug Administration (FDA) is announcing the following
public workshop entitled ``Complex Issues in Developing Medical Devices
for Pediatric Patients Affected by Rare Diseases.'' This public
workshop is organized by the Center for Devices and Radiological Health
(CDRH) and the Office of Orphan Products Development (OOPD) and is
being held in conjunction with the Center for Drug Evaluation and
Research's workshop entitled ``Complex Issues in Developing Drug and
Biological Products for Rare Diseases.'' The purpose of the public
workshop is to discuss issues related to the following broad topics
associated with medical devices for the diagnosis and treatment of
pediatric patients affected by rare diseases: Current approaches toward
use of medical devices for pediatric clinical practice; Humanitarian
Device Exemption (HDE) marketing pathway, including the Humanitarian
Use Device (HUD) designation process; Pediatric Specialty-Specific
Practice Areas; Clinical Trials and Registries; and Pediatric Needs
Assessment and Possible Approaches to Advancing Pediatric Medical
Device Development. FDA is seeking input into these topics from
academicians, clinical practitioners, patients and advocacy groups,
industry, and governmental agencies. The input from this public
workshop will help in developing a strategic plan to encourage and
accelerate the development of new medical devices and therapies for
pediatric patients affected by rare diseases. This is part of an
ongoing effort by FDA to address the needs of pediatric patients
affected by rare diseases.
Date and Time: The workshop will be held on January 8, 2014, from 8
a.m. to 5 p.m. This public workshop is being held in conjunction with
FDA's public workshop entitled ``Complex Issues in Developing Drug and
Biological Products for Rare Diseases'' which will be held on January
6, 2014, from 8 a.m. to 5 p.m. and on January 7, 2014, from 8 a.m. to
4:45 p.m.
Location: The public workshop will be held at FDA White Oak Campus,
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room
(section A of Rm. 1503), Silver Spring, MD 20993-0002. Entrance for the
public workshop 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.
Contact Person: Carol Krueger, Center for Devices and Radiological
Health, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
66, Rm. 3663, Silver Spring, MD 20993-0002, 301-796-3241,
Carol.Krueger@fda.hhs.gov.
Registration: Registration is free and available on a first-come,
first-served basis. Persons interested in attending the Complex Issues
in Developing Medical Devices for Pediatric Patients Affected by Rare
Diseases public workshop must register online by December 6, 2013, 5
p.m. Early registration is recommended because facilities are limited
and, therefore, FDA may limit the number of participants from each
organization. If time and space permit, onsite registration on the day
of the public workshop will be provided beginning at 7:30 a.m.
If you need special accommodations due to a disability, please
contact Susan Monahan (email: Susan.Monahan@fda.hhs.gov or phone: 301-
796-5661) no later than December 27, 2013.
To register for the public workshop, please visit FDA's Medical
Devices News & Events--Workshops & Conferences calendar at https://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/default.htm.
(Select this public workshop from the posted events list.) Please
provide complete contact information for each attendee, including name,
title, affiliation, address, email, and telephone number. Those without
Internet access should contact Carol Krueger to register (see Contact
Person). Registrants will receive confirmation after they have been
accepted. You will be notified if you are on a waiting list.
Streaming Webcast of the Public Workshop: This public workshop will
also be Webcast. Persons interested in viewing the Webcast must
register online by December 6, 2013, 5 p.m. Early registration is
recommended because Webcast connections are limited. Organizations are
requested to
[[Page 58317]]
register all participants, but to view using one connection per
location. Webcast participants will be sent technical system
requirements after registration and will be sent connection access
information after January 1, 2014. If you have never attended a Connect
Pro event before, test your connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get
a quick overview of the Connect Pro program, visit https://www.adobe.com/go/connectpro_overview. (FDA has verified the Web site
addresses in this document, but FDA is not responsible for any
subsequent changes to the Web sites after this document publishes in
the Federal Register.)
Comments: FDA is holding this public workshop in response to
section 510 of the Food and Drug Safety and Innovation Act to discuss
ways to encourage and accelerate the development of new medical devices
and therapies for pediatric rare diseases. In order to permit the
widest possible opportunity to obtain public comment, FDA is soliciting
either electronic or written comments on all aspects of the public
workshop topics. The deadline for submitting comments regarding this
public workshop is February 5, 2014.
Regardless of attendance at the public workshop, interested persons
may submit either electronic comments regarding this document to https://www.regulations.gov or written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
Rm. 1061, Rockville MD 20852. 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. In addition, when responding to specific
topics as outlined in section II of this document, please identify the
topic you are addressing. 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 https://www.regulations.gov.
Transcripts: Please be advised that as soon as a transcript is
available, it will be accessible at https://www.regulations.gov. It may
be viewed at the Division of Dockets Management (see Comments). 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 to the Division of Freedom of Information (ELEM-1029),
Food and Drug Administration, 12420 Parklawn Dr., Element Bldg.,
Rockville, MD 20857. A link to the transcripts will also be available
approximately 45 days after the public workshop on the Internet at
https://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/default.htm. (Select this public workshop from the posted events list).
SUPPLEMENTARY INFORMATION:
I. Background
The demand by health care professionals and consumers for safe and
effective medical devices for use for pediatric patients affected by
rare diseases continues to steadily increase. To meet that demand,
clinicians and organizations representing patients and physicians have
cited the widespread practice of modifying adult devices for pediatric
use. Certain adult medical devices may be inappropriate for pediatric
use due to a variety of factors, including patient size, growth, and
development, or may require design changes or special labeling for
pediatric use.\1\
---------------------------------------------------------------------------
\1\ ''Premarket Assessment of Pediatric Medical Devices.''
Issued May 14, 2004. This guidance may be found on FDA's Web site at
www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM089742.pdf.
---------------------------------------------------------------------------
OOPD was established to promote the development of products (drugs,
biologics, medical devices, or medical foods) that demonstrate promise
for the diagnosis, prevention, and/or treatment of rare diseases or
conditions. One of OOPD's functions is to designate devices as HUDs,
which allows them to be eligible for marketing approval under an HDE
application. The HDE pathway, authorized under section 520(m) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360j(m)), provides an
alternative pathway to market devices intended to treat or diagnose a
disease or condition that affects fewer than 4,000 individuals in the
United States per year. Roughly a quarter of the medical devices that
have received HDE marketing approval are available for pediatric
patients.
In 2007, Congress passed the Pediatric Medical Device Safety and
Improvement Act (the Act). The Act addresses pediatric device needs by
allowing sponsors of pediatric HUDs to make a profit on sales of those
devices; explicitly permitting extrapolation of adult effectiveness
data to support a pediatric indication or extrapolation of pediatric
subpopulation effectiveness data to support an indication for another
pediatric subpopulation based on a similar course of the disease or
condition or a similar effect of the device; and providing grants to
pediatric device consortia that provide technical support and
assistance to pediatric device innovators.
FDA is committed to supporting the development and availability of
safe and effective medical devices for pediatric patients affected by
rare diseases. The Agency has sponsored a number of workshops on issues
relevant to pediatric device development in recent years.
II. Topics for Discussion at the Public Workshop
FDA seeks to address and receive comments on the following topics:
A. Current Clinical Practice
1. The current use and practice trends of medical devices in rare
disease pediatric populations. For example, how much off-label use
occurs? How much modification and adaptation of existing adult devices
occurs?
2. What risks or adverse outcomes have been reported in association
with off-label use of medical devices in rare disease pediatric
populations?
B. HUD/HDE
1. Is there any confusion about the designation process for HUDs or
the application process for HDE's? Where have barriers been encountered
in the HDE marketing pathway, and how can they be mitigated? Please
provide examples of any specific issues, how frequently they occur and
suggestions to constructively address these barriers.
2. Please comment on Institutional Review Board issues that arise
for HDEs that are indicated for pediatric rare diseases.
C. Specialty Practice Areas
1. For specialty practices areas (e.g. cardiology, orthopedics, and
neurology) what existing medical devices appear to have the best
potential for modification for rare diseases that affect the pediatric
population? If possible, please prioritize existing medical devices in
terms of minimal change, moderate change, or significant change
required. Also state whether no medical device is currently available
to address the need.
2. What are the best ways to foster efficient networking across
agencies, academia, professional societies, and patient groups to
address the medical device needs of pediatric patients with rare
diseases?
D. Clinical Trials
1. What are the most challenging barriers in the process of
designing protocols for devices used to treat/diagnose rare pediatric
diseases?
2. What are unique challenges in identifying appropriate endpoints
for
[[Page 58318]]
protocols for devices used to treat/diagnose rare pediatric diseases?
3. What barriers related to statistical analyses must be addressed
in order to promote device development for rare pediatric diseases?
4. How can new registries be developed or current registries be
leveraged to provide robust data on the safety and effectiveness of
pediatric medical devices to support premarket approval and clearance,
and/or enhance postmarket surveillance activities related to pediatric
medical devices?
E. Pediatric Needs Assessment
1. Describe the parameters that should be used in determining
priority areas of development of devices, including both therapeutic
and diagnostic devices, in pediatric rare diseases.
2. What is the best approach to conduct needs assessment of medical
devices required for use with pediatric rare diseases?
F. Device Related Issues for Diagnostic Devices
1. What are medical device related issues that need to be addressed
for development of diagnostic medical devices?
G. Advancing Development
1. What incentives could help advance the development of diagnostic
and therapeutic medical devices to treat pediatric rare diseases?
2. How can possible or probable use in pediatric practice be
considered early in the development stages of all devices designed to
treat a rare disease or condition?
3. What are potential private resources (e.g., registries,
industry, or patient advocacy groups) that could be tapped to advance
the development of medical devices for rare diseases in the pediatric
population?
4. What are potential improvements or changes that can be made to
FDA guidance, regulations, or current science in order to help develop
and improve medical devices to address the needs of the pediatric
population affected by rare diseases?
Dated: September 17, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-22960 Filed 9-20-13; 8:45 am]
BILLING CODE 4160-01-P
