[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]


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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, 
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  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