[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]



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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.

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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

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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 http://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-

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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