[Federal Register Volume 79, Number 80 (Friday, April 25, 2014)]
[Notices]
[Pages 22994-22995]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-09468]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0396]


Public Meeting on Patient-Focused Drug Development for Neurologic 
Manifestations of Inborn Errors of Metabolism

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 Agency) is announcing 
a public meeting and an opportunity for public comment on Patient-
Focused Drug Development for neurologic manifestations of inborn errors 
of metabolism. Patient-Focused Drug Development is part of FDA's 
performance commitments made as part of the fifth authorization of the 
Prescription Drug User Fee Act (PDUFA V). The public meeting is 
intended to allow FDA to obtain patient perspectives on the impact of 
neurologic manifestations of inborn errors of metabolism on daily life 
as well as patient views on treatment approaches for neurologic 
manifestations of inborn errors of metabolism.

DATES: The public meeting will be held on June 10, 2014, from 9 a.m. to 
1 p.m. Registration to attend the meeting must be received by May 27, 
2014 (see the SUPPLEMENTARY INFORMATION section for instructions).

ADDRESSES: The 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. Entrance for the public meeting 
participants (non-FDA employees) is through Building 1, where routine 
security checks will be performed. For more information on parking and 
security procedures, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Submit either electronic or written comments by August 11, 2014. 
Submit electronic comments to www.regulations.gov. Submit written 
comments to the Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. All 
comments should be identified with the docket number found in brackets 
in the heading of this document.
    FDA will post the agenda approximately 5 days before the meeting 
at:http://www.fda.gov/Drugs/NewsEvents/ucm387057.htm.

FOR FURTHER INFORMATION CONTACT: Pujita Vaidya, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1170, Silver Spring, MD 20993, 301-796-
0684, FAX: 301-796-0684, email: [email protected].

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

    FDA has selected neurologic manifestations of inborn errors of 
metabolism as the focus of a public meeting under Patient-Focused Drug 
Development, an initiative that involves obtaining a better 
understanding of patients' perspectives on the severity of the disease 
and the available therapies for the condition. Patient-Focused Drug 
Development is being conducted to fulfill FDA performance commitments 
that are part of the authorization of PDUFA V under Title I of the Food 
and Drug Safety and Innovation Act (FDASIA) (Pub. L. 112-144). The full 
set of performance commitments is available on the FDA Web site at 
http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.
    FDA committed to obtain the patient perspective on 20 disease areas 
during the course of PDUFA V. For each disease area, the Agency will 
conduct a public meeting to discuss the disease and its impact on 
patients' daily lives, the types of treatment benefit that matter most 
to patients, and patients' perspectives on the adequacy of the 
available therapies. These meetings will include participation of FDA 
review divisions, the relevant patient communities, and other 
interested stakeholders.
    On April 11, 2013, FDA published a notice (78 FR 08441) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years (FYs) 2013-15, the first 3 years of the 5-year PDUFA V timeframe. 
The Agency used several criteria outlined in the April 11, 2013, notice 
to develop the list of disease areas. FDA obtained public comment on 
the Agency's proposed criteria and potential disease areas through a 
public docket and a public meeting that was convened on October 25, 
2012. In selecting the set of disease areas, FDA carefully considered 
the public comments received and the perspectives of review divisions 
at FDA. By the end of FY 2015, FDA will initiate a second public 
process for determining the disease areas for FY 2016-17. More 
information, including the list of disease areas and a general schedule 
of meetings, is posted on FDA's Web site at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm.

II. Public Meeting Information

A. Purpose and Scope of the Meeting

    The purpose of this Patient-Focused Drug Development meeting is to 
obtain input on the symptoms and other impacts of neurologic 
manifestations of inborn errors of metabolism that matter most to 
patients, as well as perspectives on current approaches to treating 
neurologic manifestations of inborn errors of metabolism. FDA expects 
that this information will come directly from patients, caregivers, and 
patient advocates. Inborn errors of metabolism include a range of 
genetic disorders in which the body has an enzyme deficiency, which 
causes buildup of harmful metabolites. Examples of inborn errors of 
metabolism include phenylketonuria, lysosomal storage disorders, Wilson 
disease, and many others. Symptoms vary depending on the condition and 
can be acute or chronic. Neurologic symptoms are common. For most 
inborn errors of

[[Page 22995]]

metabolism, a cure does not exist, and treatment focuses on prevention 
and management of signs and symptoms.
    The questions that will be asked of patients and patient 
stakeholders at the meeting are listed in this section, organized by 
topic. For each topic, a brief initial patient panel discussion will 
begin the dialogue and will be followed by a facilitated discussion 
inviting comments from other patient and patient stakeholder 
participants. In addition to input generated through this public 
meeting, FDA is interested in receiving patient input addressing these 
questions through written comments, which can be submitted to the 
public docket (see ADDRESSES).
    For the purposes of this Patient-Focused Drug Development Meeting, 
FDA is interested in hearing from patients specifically about the 
neurologic/neuropsychological aspects of inborn errors of metabolism.
    For context, please indicate if you are commenting on behalf of a 
child or loved one who has an inborn error of metabolism. If you are 
commenting on behalf of a child or loved one, please answer the 
following questions as much as possible from the patient's perspective.
Topic 1: Disease Signs, Symptoms, and Daily Impacts That Matter Most to 
Patients
    1. Of all the signs or symptoms that you/your child experiences 
because of the condition, which 1-3 neurologic/neuropsychological signs 
and/or symptoms have the most significant impact on your/your child's 
life? (Examples may include seizures, decreased muscle tone, sensory 
issues, etc.)
    2. Are there specific activities that are important to you/your 
child but that you/your child cannot do because of these neurologic/
neuropsychological signs or symptoms? (Examples of activities may 
include sleeping through the night, daily hygiene, going up the stairs, 
etc.)
    3. How have your/your child's neurologic/neuropsychological signs 
or symptoms changed over time?
Topic 2: Patient Perspectives on Current Approaches to Treating 
Neurologic Manifestations of Inborn Errors of Metabolism
    1. What are you/your child currently doing to help treat the 
condition or its signs/symptoms? (Examples may include prescription 
medicines, herbal therapies, acupuncture, over-the-counter products, 
and other therapies including nondrug therapies such as diet 
modification.)
    How well does this current treatment regimen treat the neurological 
symptoms of your/your child's disease? For example, how well do the 
treatments improve your/your child's ability to do specific activities?
    2. Assuming there is no complete cure for your/your child's 
condition, what specific attributes would you look for in an ideal 
treatment for the condition?
    3. The process of informed consent is an important way for 
researchers to communicate the purpose of a clinical trial and the 
potential benefits and risks of the trial so that people can make an 
informed decision about whether to participate. Informed consent also 
ensures that parents are fully informed and are given opportunities to 
ask questions about the clinical trial. In addition to informed consent 
from parents, assent from children may also be needed. Assent is the 
term used to describe when a child agrees to be in a clinical trial. 
Among other considerations, children should be old enough to understand 
basic facts about the clinical trial in order to provide assent to 
participate.
    In the informed consent process, what are important considerations 
to take into account in cases when the potential participant is a 
child? For example, how should the informed consent clearly communicate 
to the patient the potential benefits and risks of a study?

B. Meeting Attendance and Participation

    If you wish to attend this meeting, visit http://iempatientfocused.eventbrite.com. Please register by May 27, 2014. 
Those who are unable to attend the meeting in person can register to 
view a live Webcast of the meeting. You will be asked to indicate in 
your registration if you plan to attend in person or via the Webcast. 
Your registration will also contain your complete contact information, 
including name, title, affiliation, address, email address, and phone 
number. Seating will be limited, so early registration is recommended. 
Registration is free and will be on a first-come, first-served basis. 
However, FDA may limit the number of participants from each 
organization based on space limitations. Registrants will receive 
confirmation once they have been accepted. Onsite registration on the 
day of the meeting will be based on space availability. If you need 
special accommodations because of disability, please contact Pujita 
Vaidya (see FOR FURTHER INFORMATION CONTACT) at least 7 days before the 
meeting.
    Patients 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. They will be asked to 
send a brief summary of responses to the topic questions to 
[email protected]. Panelists will be notified of their 
selection a few days after the close of registration on May 27, 2014. 
FDA will try to accommodate all patients and patient advocate 
participants who wish to speak, either through the panel discussion or 
audience participation; however, the duration of comments may be 
limited by time constraints.
    Comments: Regardless of attendance at the public meeting, you can 
submit electronic or written responses to the questions pertaining to 
topics 1 and 2 to the public docket (see ADDRESSES) by August 11, 2014. 
Received comments may be seen in the Division of Dockets Management 
between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to 
the docket at http://www.regulations.gov.
    Transcripts: As soon as a transcript is available, FDA will post it 
at http://www.fda.gov/Drugs/NewsEvents/ucm387057.htm.

    Dated: April 22, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-09468 Filed 4-24-14; 8:45 am]
BILLING CODE 4160-01-P