81 FR 90361 - Public Meeting on Patient-Focused Drug Development for Sarcopenia; Request for Comments

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 81, Issue 240 (December 14, 2016)

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 Sarcopenia.'' 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 sarcopenia on daily life as well as patient views on treatment approaches for sarcopenia.

[Federal Register Volume 81, Number 240 (Wednesday, December 14, 2016)]
[Notices]
[Pages 90361-90363]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2016-29998]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-N-2016-4198]


Public Meeting on Patient-Focused Drug Development for 
Sarcopenia; 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 Agency) is announcing 
a public meeting and an opportunity for public comment on ``Patient-
Focused Drug Development for Sarcopenia.'' 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 sarcopenia on daily life as well as 
patient views on treatment approaches for sarcopenia.

DATES: The public meeting will be held on April 6, 2017, from 1 p.m. to 
5 p.m. Registration to attend the meeting must be received by March 27, 
2017 (see SUPPLEMENTARY INFORMATION for instructions). Public comments 
will be accepted through June 6, 2017. See the ADDRESSES section for 
information about submitting comments to the public docket.

ADDRESSES: The public meeting will be held at the FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 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 more 
information on parking and security procedures, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    You may submit comments as follows:

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): Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Division of 
Dockets Management, 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-
2016-N-4198 for ``Public Meeting on Patient-Focused Drug Development 
for Sarcopenia; Request for Comments.'' Received comments will be 
placed in the docket and, except for those submitted as ``Confidential 
Submissions,'' publicly viewable at https://www.regulations.gov or at 
the Division of Dockets Management 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 Division of Dockets Management. 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: http://www.fda.gov/regulatoryinformation/dockets/default.htm.
    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 Division of Dockets Management, 5630 Fishers 
Lane, Rm. 1061, Rockville, MD 20852.
    FDA will post the agenda approximately 5 days before the meeting

[[Page 90362]]

at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm527587.htm.

FOR FURTHER INFORMATION CONTACT: Meghana Chalasani, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1146, Silver Spring, MD 20993-0002, 240-
402-6525, FAX: 301-847-8443, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

    FDA has selected sarcopenia as the focus of a public meeting under 
Patient-Focused Drug Development, an initiative that involves obtaining 
a better understanding of patient perspectives on the severity of a 
disease and the available therapies for that condition. Patient-Focused 
Drug Development is being conducted to fulfill FDA performance 
commitments that are part of the reauthorization of the PDUFA under 
Title I of the Food and Drug Administration Safety and Innovation Act 
(Pub. L. 112-144). The full set of performance commitments is available 
at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.
    FDA committed to obtain the patient perspective on at least 20 
disease areas during the course of PDUFA V. For each disease area, the 
Agency is conducting 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 in the Federal Register 
(78 FR 21613) announcing the disease areas for meetings in fiscal years 
(FYs) 2013-2015, the first 3 years of the 5-year PDUFA V time frame. 
The Agency used several criteria outlined in that 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. FDA 
initiated a second public process for determining the disease areas for 
FY 2016-2017, and published a notice in the Federal Register on July 2, 
2015 (80 FR 38216), announcing the selection of eight disease areas. 
More information, including the list of disease areas and a general 
schedule of meetings, is posted at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm.

II. Public Meeting Information

    As part of Patient-Focused Drug Development, FDA will obtain 
patient and patient stakeholder input on the symptoms of sarcopenia 
that matter most to patients and on current approaches to treating 
sarcopenia. Sarcopenia is a condition characterized by loss of muscle 
mass and loss of muscle function or strength that occurs with age. 
While there is currently no cure, treatments for sarcopenia are 
primarily non-drug therapies including exercise and nutrition. FDA is 
interested in the perspectives of patients with sarcopenia on (1) 
symptoms and the daily impacts of their condition, (2) current 
approaches to treatment, and (3) decision factors taken into account 
when selecting a treatment.
    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. This 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).
Topic 1: Disease Symptoms and Daily Impacts That Matter Most to 
Patients
    (1) Of all the symptoms that you experience because of your 
condition, which one to three symptoms have the most significant impact 
on your life? (Examples may include difficulty walking, feeling 
unsteady and falling frequently, having a decreased level of activity, 
etc.)
    (2) Are there specific activities that are important to you but 
that you cannot do at all or as fully as you would like because of your 
condition? (Examples of activities may include participation in social 
activities, household chores, daily hygiene, etc.)
    (3) How do your symptoms and their negative impacts affect your 
daily life on the best days? On the worst days?
    (4) How have your condition and its symptoms changed over time?
    (a) Would you define your condition today as being well managed?
    (5) What worries you most about your condition?
Topic 2: Patients' Perspectives on Current Approaches to Treatment
    (1) What are you currently doing to help treat your condition or 
its symptoms? (Examples may include prescription medicines, over-the-
counter products, and other therapies including non-drug therapies such 
as diet modification.)
    (a) What specific symptoms do your treatments address?
    (b) How has your treatment regimen changed over time, and why?
    (2) How well does your current treatment regimen control your 
condition?
    (a) How well do your treatments address specific activities that 
are important to you in your daily life?
    (b) How well have these treatments worked for you as your condition 
has changed over time?
    (3) What are the most significant downsides to your current 
treatments, and how do they affect your daily life? (Examples of 
downsides may include going to the hospital or clinic for treatment, 
time devoted to treatment, etc.)
    (4) What specific things would you look for in an ideal treatment 
for your condition?
    (a) What would you consider to be a meaningful improvement (for 
example, symptom improvements or functional improvements) in your 
condition that a treatment could provide?
III. Meeting Attendance and Participation
    If you wish to attend this meeting, visit https://sarcopeniapfdd.eventbrite.com. Please register by March 27, 2017. If 
you are unable to attend the meeting in person, you 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. 
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 a disability, please 
contact Meghana Chalasani (see FOR FURTHER INFORMATION CONTACT) at 
least 7 days before the meeting.

[[Page 90363]]

    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. These patients also 
must send to [email protected] a brief summary of responses to 
the topic questions by March 20, 2017. Panelists will be notified of 
their selection approximately 7 days before the public meeting. We will 
try to accommodate all patients and patient stakeholders who wish to 
speak, either through the panel discussion or audience participation; 
however, the duration of comments may be limited by time constraints.
    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 Division of Dockets 
Management (see ADDRESSES). A link to the transcript will also be 
available on the Internet at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm527587.htm.

    Dated: December 9, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-29998 Filed 12-13-16; 8:45 am]
BILLING CODE 4164-01-P