
[Federal Register Volume 84, Number 41 (Friday, March 1, 2019)]
[Notices]
[Pages 7082-7084]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-03675]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-N-0077]


Patient Perspectives on the Impact of Rare Diseases: Bridging the 
Commonalities; 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 
``Patient Perspectives on the Impact of Rare Diseases: Bridging the 
Commonalities.'' This public meeting is intended to obtain patients' 
and caregivers' perspectives on impacts of rare diseases on daily life 
and to assess commonalities that may help the Agency and medical 
product developers further understand and advance the development of 
treatments for rare diseases. Developing a treatment for a rare disease 
can present unique challenges, such as the small number of individuals 
affected and heterogenous etiologies and manifestations. While the 
differences between rare diseases are critically important, it is also 
important to assess commonalities to synergize product development in 
rare diseases. The goal of this meeting is to identify common issues 
and symptoms in rare diseases to help advance medical product 
development, potentially through the creation of novel endpoints or 
trial designs that focus on commonalities across a variety of rare 
diseases.

DATES: The public meeting will be held on April 29, 2019, from 1 p.m. 
to 5 p.m. The online registration to attend must be received by April 
15, 2019. Onsite registration on the day of the meeting will be based 
on space availability. Submit either electronic or written comments on 
the public meeting by May 30, 2019. 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/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    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 May 30, 2019. The https://www.regulations.gov 
electronic filing system will accept comments until midnight Eastern 
Time at the end of May 30, 2019. 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'').

[[Page 7083]]

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-0077 for ``Patient Perspectives on the Impact of Rare 
Diseases: Bridging the Commonalities.'' 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.gpo.gov/fdsys/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 20933, 301-796-7634, 
OOPDOrphanEvents@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

    The development of drugs, biologics, and devices for rare diseases 
involves unique challenges. The goal of this meeting is to identify 
common issues across rare diseases to help address some of these 
challenges. Rare diseases, often referred to as orphan diseases, are 
defined based on rarity of occurrence. Although these diseases are 
individually rare, collectively they are not. According to the National 
Institutes of Health, there are approximately 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 passed in 1983, drugs and biologics for over 750 rare 
disease indications have been developed and approved for marketing. In 
addition to drugs and biologics, there has been progress in the 
development of devices for rare diseases. Since 1990, the FDA has 
approved 74 medical devices for orphan indications under the Agency's 
Humanitarian Device Exemption program. Despite these successes, we 
recognize that thousands of rare diseases still have no 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 
collaboration between many stakeholders, including scientists, product 
developers, regulators, policy makers, and patients. FDA is committed 
to working with stakeholders to advance treatment options for patients 
with rare diseases.
    This public meeting will focus on the perspective of those affected 
by rare diseases. Patients, family members, and caregivers will provide 
important input on the impact of rare diseases on daily life. While the 
differences between rare diseases are critically important, this 
meeting will assess commonalities. The specific goal of this meeting is 
to identify common issues and symptoms in rare diseases to help advance 
medical product development, potentially through the generation of 
novel endpoints or trial designs that focus on commonalities across a 
variety of rare disease.
    FDA will provide a summary document from this public meeting. This 
meeting will include participants from FDA, the patient community, 
caregivers, and other interested stakeholders.

II. Topics for Discussion at the Public Meeting

    This public meeting will consist of panels of patients/caregivers 
and facilitated discussions. The aim of the meeting is to hear directly 
from patients with rare diseases and their caregivers and family 
members. The meeting will include patients with any rare disease and 
their caregivers and family members. It is not restricted to a specific 
rare disease or group of rare diseases.
    The meeting will focus on several related topics. Specifically, FDA 
would like to hear directly from patients with rare diseases and their 
caregivers and family members about disease symptoms, treatment 
considerations, and factors relevant to participating in a clinical 
study or registry. We invite the public to register and participate in 
our panel discussions. A detailed agenda and meeting topics will be 
posted on the following website in advance of the meeting: https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm628352.htm.

III. Participating in the Public Meeting

    Registration: To register for the public meeting, please visit the 
following website by April 15, 2019: https://patient-perspectives-rare-diseases.eventbrite.com. 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 April 15, 2019, 5 p.m. Eastern Time. Early 
registration is recommended because seating is

[[Page 7084]]

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 
OOPDOrphanEvents@fda.hhs.gov no later than April 15, 2019.
    Requests for Oral Presentations: Patients and patient 
representatives who are interested in presenting comments as part of 
the initial panel discussions will be asked to indicate in their 
registration which topic(s) they wish to address. These patients and 
patient representatives also must send to Eleanor Dixon-Terry 
(OOPDOrphanEvents@fda.hhs.gov or 301-796-7634) a brief summary of 
responses to the meeting topics by April 1, 2019. Details regarding the 
meeting agenda and topics will be available at https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm628352.htm.
    FDA will hold an open public comment period to give the public an 
opportunity to comment. 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.
    Panelists and open public comment period speakers will be notified 
of their selection approximately 7 days before the public meeting. We 
will try to accommodate all patients and patient representatives who 
wish to speak, either through the panel discussion, an open public 
comment period, or audience participation; 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 April 28, 2019: https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm628352.htm.
    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 https://www.adobe.com/go/connectpro_overview. 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/MeetingsConferencesWorkshops/ucm628352.htm.

    Dated: February 26, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-03675 Filed 2-28-19; 8:45 am]
 BILLING CODE 4164-01-P


