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]

Download as PDF 3384 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. jbell on DSKJLSW7X2PROD with NOTICES SUMMARY: VerDate Sep<11>2014 18:20 Jan 17, 2020 Jkt 250001 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 PO 00000 Frm 00050 Fmt 4703 Sfmt 4703 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 E:\FR\FM\21JAN1.SGM 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 VerDate Sep<11>2014 18:20 Jan 17, 2020 Jkt 250001 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 PO 00000 Frm 00051 Fmt 4703 Sfmt 4703 3385 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 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/newsevents/fda-meetings-conferences-andworkshops/fda-rare-disease-day-2020- E:\FR\FM\21JAN1.SGM 21JAN1 3386 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 http:// 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 jbell on DSKJLSW7X2PROD with NOTICES 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. VerDate Sep<11>2014 18:20 Jan 17, 2020 Jkt 250001 PO 00000 Frm 00052 Fmt 4703 Sfmt 4703 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 21JAN1

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]]

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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.

-----------------------------------------------------------------------

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 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