[Federal Register Volume 81, Number 81 (Wednesday, April 27, 2016)]
[Notices]
[Pages 24818-24820]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-09785]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-N-2016-1134]


Public Meeting on Patient-Focused Drug Development for Patients 
Who Have Received an Organ Transplant

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

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

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 patients who have received an organ 
transplant. 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 
receiving an organ transplant on daily life and patient views on 
treatment approaches; the input from this public meeting will help in 
developing topics for further discussion. FDA is also interested in 
discussing issues related to scientific challenges in developing drugs 
to manage organ transplantation. In the afternoon, FDA will hold a 
workshop and provide information for and gain perspective from patients 
and patient advocacy organizations, health care providers, academic 
experts, and industry on various aspects of clinical development of 
drug products intended to manage organ transplantation.

DATES: The public meeting will be held on September 27, 2016, from 9 
a.m. to 5 p.m. Please register here for the meeting by September 20, 
2016: http://organtransplantpfdd.eventbrite.com. Submit electronic or 
written comments to the public docket by November 27, 2016.

ADDRESSES: The meeting and workshop 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. Participants must enter 
through Building 1 and undergo security screening. 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: http://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to http://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

[[Page 24819]]

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 http://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-1134 for ``Public Meeting on Patient-Focused Drug 
Development for Patients Who Have Received an Organ Transplant.'' 
Received comments will be placed in the docket and, except for those 
submitted as ``Confidential Submissions,'' publicly viewable at http://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 http://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 http://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 
at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm495933.htm.

FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1146, Silver Spring, MD 20993, 301-796-
5003, FAX: 301-847-8443, [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background on Patient-Focused Drug Development

    FDA has selected patients who have received an organ transplant 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 these conditions. 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 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 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 July 2, 2015, FDA published a notice (80 FR 32816) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years 2016-2017, final 2 years of 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. In selecting the 
set of disease areas, FDA carefully considered the public comments 
received and the perspectives of review divisions at FDA. 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 and Workshop Information

A. Purpose and Scope of the Meeting

    The purpose of this Patient-Focused Drug Development meeting is to 
obtain input on organ transplantation and current approaches to 
management of organ transplantation. In 2015, over 25,000 people in the 
United States received an organ transplant. Organ transplantation 
requires pharmacologic and non-pharmacologic management before and 
after receipt. There are FDA-approved therapies used to assist the 
immune system in responding properly to the transplanted organ. 
Treatment requires a combination of drugs given for the lifetime of a 
transplanted organ. FDA is committed to working with all stakeholders 
to develop safe and effective therapies for affected individuals.
    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). When submitting comments, if you are 
commenting on behalf of a child, please indicate that you are doing so 
and

[[Page 24820]]

answer the following questions as much as possible from the patient's 
perspective.
Topic 1: Disease Symptoms and Daily Impacts That Matter Most to 
Patients
    1. What have been the most significant changes in your overall 
health since you received your transplanted organ?
    (a) How long has it been since you received your transplant?
    2. Focusing on symptoms related to your organ transplant and post-
transplant effects, which 1-3 symptoms have the most significant impact 
on your life? (Examples may include pain, infection, anxiety, etc.)
    3. 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 
transplant? (Examples of activities may include sleeping through the 
night, driving, walking/running, exercising, etc.)
    (a) How do your symptoms and their negative impacts affect your 
daily life on the best days? On the worst days? (Examples may include 
limitations on the ability to undertake physically strenuous 
activities, restrictions on the ability to travel, lack of appetite, 
fatigue, etc.)
    4. How has your experience with your transplanted organ changed 
over time? Do particular symptoms come and go as your duration of time 
with a transplanted organ has increased? If so, do you know of anything 
that makes your symptoms better? Worse?
    5. What worries you most about your health post-transplant?
Topic 2: Patients' Perspectives on Transplant and Treatment Impacts
    1. What are you currently doing to maintain your transplanted organ 
or treat related health concerns following transplantation? (Examples 
may include immunosuppressants, antibiotics, antivirals, over-the-
counter products, and other therapies including non-drug therapies)
    (a) How has your post-transplant treatment regimen changed over 
time, and why?
    2. How well does your current treatment regimen manage the most 
significant symptoms you experience post-transplantation?
    (a) How well do these treatments improve your ability to do 
specific activities that are important to you in your daily life?
    (b) How well have these treatments worked for you as your 
experiences post-transplant have 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 bothersome side effects, need for multiple 
medications, risk of infection, need for hospitalization, etc.)
    (a) What are the biggest challenges you face in maintaining your 
post-transplant treatment regimen? (Examples of challenges may be 
bothersome side effects, need for multiple medications, etc.)
    4. What specific things would you look for in an ideal treatment 
for managing your transplanted organ?
    In the afternoon, discussion will be related to scientific topics, 
with the goal of understanding issues that may affect the development 
of drugs for the treatment of organ transplantation and identifying 
topics for future discussion. Discussion topics for the afternoon will 
include the following: Current treatment considerations, adherence, 
clinical trial designs, and clinical trial endpoints.

B. Meeting Attendance and Participation

    If you wish to attend this meeting, visit http://organtransplantpfdd.eventbrite.com. Please register by September 20, 
2016. 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 Graham 
Thompson (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. These patients also 
must send to [email protected] a brief summary of responses to 
the topic questions by September 12, 2016. 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.
    FDA will hold an open public comment period to give the public an 
opportunity to comment. Registration for open public comment will occur 
at the registration desk on the day of the meeting and workshop on a 
first-come, first-served basis.
    Docket Comments: Regardless of if you attend 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 
November 27, 2016. 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/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm495933.htm.

    Dated: April 21, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-09785 Filed 4-26-16; 8:45 am]
BILLING CODE 4164-01-P