[Federal Register Volume 78, Number 184 (Monday, September 23, 2013)]
[Notices]
[Pages 58316-58318]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-22960]
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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.
ACTION: Notice of public workshop; request for comments.
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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 http://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,
[email protected].
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: [email protected] 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 http://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
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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 http://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 http://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 http://www.regulations.gov.
Transcripts: Please be advised that as soon as a transcript is
available, it will be accessible at http://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
http://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\
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\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.
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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
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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