Food and Drug Administration Rare Disease Day 2020: Supporting the Future of Rare Disease Product Development; Public Meeting; Request for Comments, 3384-3386 [2020-00829]
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Federal Register / Vol. 85, No. 13 / Tuesday, January 21, 2020 / Notices
Electronic Submissions
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2019–N–5646]
Food and Drug Administration Rare
Disease Day 2020: Supporting the
Future of Rare Disease Product
Development; Public Meeting; Request
for Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice of public meeting;
request for comments.
ACTION:
The Food and Drug
Administration (FDA or the Agency) is
announcing a public meeting and an
opportunity for public comment on
‘‘FDA Rare Disease Day 2020:
Supporting the Future of Rare Disease
Product Development.’’ Developing a
treatment for a rare disease can present
unique challenges. The goal of this
meeting is to obtain stakeholders’
perspectives on challenges and
solutions in rare disease product
development and identify
commonalities that can support product
development across a variety of rare
diseases.
DATES: The public meeting will be held
on February 24, 2020, from 9 a.m. to 5
p.m. Submit either electronic or written
comments on the public meeting by
March 29, 2020. 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., Building
31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993–
0002. 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/about-fda/white-oakcampus-information/public-meetingsfda-white-oak-campus.
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 March 29, 2020. The
https://www.regulations.gov electronic
filing system will accept comments
until 11:59 p.m. Eastern Time at the end
of March 29, 2020. 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.
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SUMMARY:
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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. Because 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–
2019–N–5646 for ‘‘FDA Rare Disease
Day 2020: Supporting the Future of Rare
Disease Product Development.’’
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
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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.govinfo.gov/content/pkg/FR-201509-18/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.
FOR FURTHER INFORMATION, CONTACT:
Eleanor Dixon-Terry, Office of Orphan
Products Development, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 32, Rm. 5163, Silver Spring,
MD 20993, 301–796–7634,
OOPDOrphanEvents@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
Rare diseases, often referred to as
orphan diseases, are diseases that affect
less than 200,000 persons in the United
States. While these diseases are
individually rare, collectively they are
not rare. There are more than 7,000 rare
diseases affecting an estimated 30
million people in the United States.
Many of these rare diseases are serious
or life-threatening and many affect
children.
The combination of government
incentives and scientific advances has
fueled extraordinary development in
orphan drugs. Since the Orphan Drug
Act was first passed in 1983, drugs and
biologics for over 800 rare disease
indications have been developed and
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21JAN1
jbell on DSKJLSW7X2PROD with NOTICES
Federal Register / Vol. 85, No. 13 / Tuesday, January 21, 2020 / Notices
approved for marketing. In addition to
drugs and biologics, there has been
progress in the development of medical
devices for rare diseases. Since the
implementation of the Humanitarian
Use Device program in 1996, FDA has
approved 77 medical devices for an
orphan indication under the Agency’s
Humanitarian Device Exemption
program. Unfortunately, most rare
diseases still do not have approved
treatments.
Developing a treatment for a rare
disease can present unique challenges.
Potential challenges include the small
number of individuals affected, lack of
understanding of the natural history of
the disease, phenotypic heterogeneity,
and lack of validated endpoints for use
in clinical trials. Overcoming these
challenges requires the collaboration
between many stakeholders, including
scientists, product developers,
regulators, policy makers, and patients.
In addition, as scientific understanding
and technological development
advances, it is essential for these
stakeholders to stay abreast of the new
challenges and opportunities in rare
disease product development. Some
challenges that need to be addressed
include consideration of manufacturing
needs to support the development of
novel products, such as gene therapies,
and considerations related to products
developed for diseases or conditions
affecting one or a few individuals. FDA
is committed to working with
stakeholders to advance treatment
options for patients with rare diseases.
On April 29, 2019, FDA held a public
meeting focusing on the perspectives of
those affected by rare diseases. The FDA
Rare Disease Day 2020 meeting will
build on the previous meeting and
include perspectives from additional
stakeholders in rare disease product
development, such as academic
investigators and pharmaceutical
companies. While the differences
between rare diseases are critically
important, this meeting will look to find
commonalities that may help the
Agency and medical product developers
further understand and advance the
development of treatments for rare
diseases. The specific goal of this
upcoming meeting is to identify
challenges and solutions in rare disease
product development to optimize rare
disease medical product development.
Potential ways to accomplish this goal
may include identifying common
clinical trial designs and analytical
plans for natural history or registry
studies that would be applicable to
many rare diseases.
This meeting will include
participation of FDA, the patient
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community, patient advocacy groups,
academic investigators, medical product
developers, and other interested
stakeholders.
II. Topics for Discussion at the Public
Meeting
This public meeting will consist of
presentations and interactive panel
discussions. The presentations will
provide information to outline different
perspectives in rare disease product
development. The panel discussions
will be moderated and allow additional
panelists to provide individual
perspectives. There will be an
opportunity for discussion between the
panelists and the audience.
The meeting will focus on several
related topics. First, FDA would like to
hear from rare disease stakeholders on
strategies to optimize registry and
natural history data collection to
support rare disease product
development. Second, FDA would like
to hear from rare disease stakeholders
on new opportunities and challenges in
rare disease product development in the
setting of recent scientific advancements
that may enable the development of
medical products for diseases or
conditions affecting one or a few
individuals. FDA staff will also offer
their perspective on these topics. We
invite the public to register and
participate in the public meeting. A
detailed agenda will be posted on the
following website in advance of the
meeting: https://www.fda.gov/newsevents/fda-meetings-conferences-andworkshops/fda-rare-disease-day-2020supporting-future-rare-disease-productdevelopment-02242020-02242020.
III. Participating in the Public Meeting
Registration: To register for the public
meeting, please visit the following
website by February 17, 2020: https://
www.eventbrite.com/e/supporting-thefuture-of-rare-disease-productdevelopment-public-meetingregistration-77190744595. Please
provide complete contact information
for each attendee, including name, title,
affiliation, address, email, and
telephone.
Registration is free and based on
space availability, with priority given to
early registrants. Persons interested in
attending this public meeting must
register by February 17, 2020, 5 p.m.
Eastern Time. Early registration is
recommended because seating is
limited; therefore, FDA may limit the
number of participants from each
organization. Registrants will receive
confirmation when their registration has
been received. If time and space permit,
onsite registration on the day of the
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public meeting will be provided
beginning an hour prior to the start of
the meeting.
If you need special accommodations
due to a disability, please contact
Eleanor Dixon-Terry, at 301–796–7634,
or OOPDOrphanEvents@fda.hhs.gov no
later than February 17, 2020.
An agenda for the meeting and any
other background materials will be
made available 5 days before the
meeting at https://www.fda.gov/newsevents/fda-meetings-conferences-andworkshops/fda-rare-disease-day-2020supporting-future-rare-disease-productdevelopment-02242020-02242020.
Requests for Open Public Comment
Period Speakers: FDA will hold an open
public comment period to give the
public an opportunity to comment on
the meeting topics. Registration for open
public comment will occur in the
meeting registration and at the
registration desk on the day of the
meeting on a first-come, first-served
basis. The open public comment period
is for in-person attendees only.
Open public comment period
speakers will be notified of their
selection approximately 7 days before
the public meeting. We will try to
accommodate all who wish to speak,
either through the open public comment
period or audience participation during
the meeting; however, the duration of
comments may be limited by time
constraints.
Streaming Webcast of the Public
Meeting: For those unable to attend in
person, FDA will provide a live webcast
of the meeting. To register for the
streaming webcast of the public
meeting, please visit the following
website by February 23, 2020: https://
www.fda.gov/news-events/fda-meetingsconferences-and-workshops/fda-raredisease-day-2020-supporting-futurerare-disease-product-development02242020-02242020.
If you have never attended a FDA
York Media event before, test your
connection at https://
www.yorkmedia.com/webcast/
systemrequirements/. FDA has verified
the website addresses in this document,
as of the date this document publishes
in the Federal Register, but websites are
subject to change over time.
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/newsevents/fda-meetings-conferences-andworkshops/fda-rare-disease-day-2020-
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Federal Register / Vol. 85, No. 13 / Tuesday, January 21, 2020 / Notices
supporting-future-rare-disease-productdevelopment-02242020-02242020.
Dated: January 13, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–00829 Filed 1–17–20; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2020–N–0008]
Request for Nominations for
Individuals and Consumer
Organizations for Advisory
Committees
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA) is requesting that
any consumer organizations interested
in participating in the selection of
voting and/or nonvoting consumer
representatives to serve on its advisory
committees or panels notify FDA in
writing. FDA is also requesting
nominations for voting and/or
nonvoting consumer representatives to
serve on advisory committees and/or
SUMMARY:
panels for which vacancies currently
exist or are expected to occur in the near
future. Nominees recommended to serve
as a voting or nonvoting consumer
representative may be self-nominated or
may be nominated by a consumer
organization. FDA seeks to include the
views of women and men, members of
all racial and ethnic groups, and
individuals with and without
disabilities on its advisory committees
and, therefore, encourages nominations
of appropriately qualified candidates
from these groups.
DATES: Any consumer organization
interested in participating in the
selection of an appropriate voting or
nonvoting member to represent
consumer interests on an FDA advisory
committee or panel may send a letter or
email stating that interest to FDA (see
ADDRESSES) by February 20, 2020, for
vacancies listed in this notice.
Concurrently, nomination materials for
prospective candidates should be sent to
FDA (see ADDRESSES) by February 20,
2020. Nominations will be accepted for
current vacancies and for those that will
or may occur through December 31,
2020.
ADDRESSES: All statements of interest
from consumer organizations interested
in participating in the selection process
should be submitted electronically to
ACOMSSubmissions@fda.hhs.gov, by
mail to Advisory Committee Oversight
and Management Staff, 10903 New
Hampshire Ave., Bldg. 32, Rm. 5122,
Silver Spring, MD 20993–0002, or by
Fax: 301–847–8640.
Consumer representative nominations
should be submitted electronically by
logging into the FDA Advisory
Committee Membership Nomination
Portal: https://www.accessdata.fda.gov/
scripts/FACTRSPortal/FACTRS/
index.cfm, by mail to Advisory
Committee Oversight and Management
Staff, 10903 New Hampshire Ave., Bldg.
32, Rm. 5122, Silver Spring, MD 20993–
0002, or by Fax: 301–847–8640.
Additional information about becoming
a member of an FDA advisory
committee can also be obtained by
visiting FDA’s website at https://
www.fda.gov/AdvisoryCommittees/
default.htm.
For
questions relating to participation in the
selection process: Kimberly Hamilton,
Advisory Committee Oversight and
Management Staff, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 32, Rm. 5122, Silver Spring,
MD 20993–0002, 301–796–8220, email
kimberly.hamilton@fda.hhs.gov.
For questions relating to specific
advisory committees or panels, contact
the appropriate contact person listed in
table 1.
FOR FURTHER INFORMATION CONTACT:
TABLE 1—ADVISORY COMMITTEE CONTACTS
Contact person
Committee/panel
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Kathleen Hayes, Center for Biologics Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 71, Rm. 6307C, Silver Spring, MD 20993–0002, 301–
796–7864, email: Kathleen.Hayses@fda.hhs.gov.
Kalyani Bhatt, Center for Drugs Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 31, Rm. 2438, Silver Spring, MD 20993–0002, 301–796–
9005, email: Kalyani.Bhatt@fda.hhs.gov.
LaToya Bonner, Center for Drugs Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 31, Rm. 2428, Silver Spring, MD 20993–0002, 301–796–
2855, email: LaToya.Bonner@fda.hhs.gov.
Philip Bautista, Center for Drugs Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 31, Rm. 2430, Silver Spring, MD 20993–0002, 240–762–
8729, email: Philip.Bautista@fda.hhs.gov.
Patricio Garcia, Center for Devices and Radiological Health, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 66, Rm. G610, Silver Spring, MD 20993–0002, 301–796–
6875, email: Patricio.Garcia@fda.hhs.gov.
Sara Anderson, Center for Devices and Radiological Health, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 66, Rm. G616, Silver Spring, MD 20993–0002, 301–796–
7047, email: Sara.Anderson@fda.hhs.gov.
Evella Washington, Center for Devices and Radiological Health, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 66, Rm. G640, Silver Spring, MD 20993–0002, 301–796–
6683, email: Evella.Washington@fda.hhs.gov.
Joannie Adams-White, Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 5519, Silver Spring, MD 20993–0002, 301–
796–5421, email: Joannie.Adams-White@fda.hhs.gov.
Aden Asefa, Center for Devices and Radiological Health, Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 66, Rm. G642, Silver Spring, MD 20993–0002, 301–796–0400,
email: Aden.Asefa@fda.hhs.gov.
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Allergenic Products Advisory Committee.
Bone, Reproductive and Urological Drugs Advisory Committee, Psychopharmacologic
Drugs Advisory Committee.
Dermatologic and Ophthalmic Drugs Advisory
Committee.
Drug Safety and Risk Management Advisory
Committee.
Clinical Chemistry and Clinical Toxicology Devices Panel, Gastroenterology and Urology
Devices Panel, Obstetrics and Gynecology
Devices Panel.
Dental Products Devices Panel.
Circulatory Systems Devices Panel.
Medical Devices Dispute Resolution Panel.
Immunology Devices Panel; Microbiology Devices Panel.
E:\FR\FM\21JAN1.SGM
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]]>Agencies
[Federal Register Volume 85, Number 13 (Tuesday, January 21, 2020)]
[Notices]
[Pages 3384-3386]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-00829]
[[Page 3384]]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2019-N-5646]
Food and Drug Administration Rare Disease Day 2020: Supporting
the Future of Rare Disease Product Development; 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 or the Agency) is
announcing a public meeting and an opportunity for public comment on
``FDA Rare Disease Day 2020: Supporting the Future of Rare Disease
Product Development.'' Developing a treatment for a rare disease can
present unique challenges. The goal of this meeting is to obtain
stakeholders' perspectives on challenges and solutions in rare disease
product development and identify commonalities that can support product
development across a variety of rare diseases.
DATES: The public meeting will be held on February 24, 2020, from 9
a.m. to 5 p.m. Submit either electronic or written comments on the
public meeting by March 29, 2020. 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., Building 31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993-0002. 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/about-fda/white-oak-campus-information/public-meetings-fda-white-oak-campus.
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 March 29, 2020. The https://www.regulations.gov
electronic filing system will accept comments until 11:59 p.m. Eastern
Time at the end of March 29, 2020. 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. Because 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-2019-N-5646 for ``FDA Rare Disease Day 2020: Supporting the Future
of Rare Disease Product Development.'' 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.govinfo.gov/content/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.
FOR FURTHER INFORMATION, CONTACT: Eleanor Dixon-Terry, Office of Orphan
Products Development, Food and Drug Administration, 10903 New Hampshire
Ave., Bldg. 32, Rm. 5163, Silver Spring, MD 20993, 301-796-7634,
[email protected].
SUPPLEMENTARY INFORMATION:
I. Background
Rare diseases, often referred to as orphan diseases, are diseases
that affect less than 200,000 persons in the United States. While these
diseases are individually rare, collectively they are not rare. There
are more than 7,000 rare diseases affecting an estimated 30 million
people in the United States. Many of these rare diseases are serious or
life-threatening and many affect children.
The combination of government incentives and scientific advances
has fueled extraordinary development in orphan drugs. Since the Orphan
Drug Act was first passed in 1983, drugs and biologics for over 800
rare disease indications have been developed and
[[Page 3385]]
approved for marketing. In addition to drugs and biologics, there has
been progress in the development of medical devices for rare diseases.
Since the implementation of the Humanitarian Use Device program in
1996, FDA has approved 77 medical devices for an orphan indication
under the Agency's Humanitarian Device Exemption program.
Unfortunately, most rare diseases still do not have approved
treatments.
Developing a treatment for a rare disease can present unique
challenges. Potential challenges include the small number of
individuals affected, lack of understanding of the natural history of
the disease, phenotypic heterogeneity, and lack of validated endpoints
for use in clinical trials. Overcoming these challenges requires the
collaboration between many stakeholders, including scientists, product
developers, regulators, policy makers, and patients. In addition, as
scientific understanding and technological development advances, it is
essential for these stakeholders to stay abreast of the new challenges
and opportunities in rare disease product development. Some challenges
that need to be addressed include consideration of manufacturing needs
to support the development of novel products, such as gene therapies,
and considerations related to products developed for diseases or
conditions affecting one or a few individuals. FDA is committed to
working with stakeholders to advance treatment options for patients
with rare diseases.
On April 29, 2019, FDA held a public meeting focusing on the
perspectives of those affected by rare diseases. The FDA Rare Disease
Day 2020 meeting will build on the previous meeting and include
perspectives from additional stakeholders in rare disease product
development, such as academic investigators and pharmaceutical
companies. While the differences between rare diseases are critically
important, this meeting will look to find commonalities that may help
the Agency and medical product developers further understand and
advance the development of treatments for rare diseases. The specific
goal of this upcoming meeting is to identify challenges and solutions
in rare disease product development to optimize rare disease medical
product development. Potential ways to accomplish this goal may include
identifying common clinical trial designs and analytical plans for
natural history or registry studies that would be applicable to many
rare diseases.
This meeting will include participation of FDA, the patient
community, patient advocacy groups, academic investigators, medical
product developers, and other interested stakeholders.
II. Topics for Discussion at the Public Meeting
This public meeting will consist of presentations and interactive
panel discussions. The presentations will provide information to
outline different perspectives in rare disease product development. The
panel discussions will be moderated and allow additional panelists to
provide individual perspectives. There will be an opportunity for
discussion between the panelists and the audience.
The meeting will focus on several related topics. First, FDA would
like to hear from rare disease stakeholders on strategies to optimize
registry and natural history data collection to support rare disease
product development. Second, FDA would like to hear from rare disease
stakeholders on new opportunities and challenges in rare disease
product development in the setting of recent scientific advancements
that may enable the development of medical products for diseases or
conditions affecting one or a few individuals. FDA staff will also
offer their perspective on these topics. We invite the public to
register and participate in the public meeting. A detailed agenda will
be posted on the following website in advance of the meeting: https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-day-2020-supporting-future-rare-disease-product-development-02242020-02242020.
III. Participating in the Public Meeting
Registration: To register for the public meeting, please visit the
following website by February 17, 2020: https://www.eventbrite.com/e/supporting-the-future-of-rare-disease-product-development-public-meeting-registration-77190744595. Please provide complete contact
information for each attendee, including name, title, affiliation,
address, email, and telephone.
Registration is free and based on space availability, with priority
given to early registrants. Persons interested in attending this public
meeting must register by February 17, 2020, 5 p.m. Eastern Time. Early
registration is recommended because seating is limited; therefore, FDA
may limit the number of participants from each organization.
Registrants will receive confirmation when their registration has been
received. If time and space permit, onsite registration on the day of
the public meeting will be provided beginning an hour prior to the
start of the meeting.
If you need special accommodations due to a disability, please
contact Eleanor Dixon-Terry, at 301-796-7634, or
[email protected] no later than February 17, 2020.
An agenda for the meeting and any other background materials will
be made available 5 days before the meeting at https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-day-2020-supporting-future-rare-disease-product-development-02242020-02242020.
Requests for Open Public Comment Period Speakers: FDA will hold an
open public comment period to give the public an opportunity to comment
on the meeting topics. Registration for open public comment will occur
in the meeting registration and at the registration desk on the day of
the meeting on a first-come, first-served basis. The open public
comment period is for in-person attendees only.
Open public comment period speakers will be notified of their
selection approximately 7 days before the public meeting. We will try
to accommodate all who wish to speak, either through the open public
comment period or audience participation during the meeting; however,
the duration of comments may be limited by time constraints.
Streaming Webcast of the Public Meeting: For those unable to attend
in person, FDA will provide a live webcast of the meeting. To register
for the streaming webcast of the public meeting, please visit the
following website by February 23, 2020: https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-day-2020-supporting-future-rare-disease-product-development-02242020-02242020.
If you have never attended a FDA York Media event before, test your
connection at https://www.yorkmedia.com/webcast/systemrequirements/. FDA
has verified the website addresses in this document, as of the date
this document publishes in the Federal Register, but websites are
subject to change over time.
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/news-events/fda-meetings-conferences-
and-workshops/fda-rare-disease-day-2020-
[[Page 3386]]
supporting-future-rare-disease-product-development-02242020-02242020.
Dated: January 13, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-00829 Filed 1-17-20; 8:45 am]
BILLING CODE 4164-01-P
