[Federal Register Volume 79, Number 237 (Wednesday, December 10, 2014)]
[Notices]
[Pages 73325-73326]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-28828]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-1998]


Patient-Focused Drug Development Public Meeting on Chagas Disease

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

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

SUMMARY: The Food and Drug Administration (FDA) is announcing a public 
meeting and an opportunity for public comment on Patient-Focused Drug 
Development for Chagas disease. Patient-Focused Drug Development is 
part of FDA's performance commitments in the fifth authorization of the 
Prescription Drug User Fee Act (PDUFA V). The meeting is intended to 
allow FDA to obtain patients' perspectives on the impact that Chagas 
disease has on their daily lives, as well as their perspectives on the 
available therapies for Chagas disease. FDA is also interested in 
discussing issues related to scientific challenges in developing drugs 
to treat Chagas disease. In the afternoon, FDA will 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 
treat Chagas disease. The input from this public meeting will help in 
developing topics for further discussion.

DATES: The meeting will be held on April 28, 2015, from 9 a.m. to 5 
p.m. Registration to attend the meeting must be received by April 20, 
2015. See the SUPPLEMENTARY INFORMATION section for information on how 
to register for the meeting. Submit electronic or written comments by 
June 29, 2015.

ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903 
New Hampshire Ave., Bldg. 31 Conference Center, Sections B and C of the 
Great Room (Rm. 1503), Silver Spring, MD 20993. Participants must enter 
through Building 1 and undergo security screening. For more information 
on parking and security procedures, please visit http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Submit electronic comments to http://www.regulations.gov. Submit 
written comments to the Division of Dockets Management (HFA-305), Food 
and Drug Administration, 5630 Fishers Lane, Room 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/ucm420130.htm.

FOR FURTHER INFORMATION CONTACT: Pujita Vaidya, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1144, Silver Spring, MD 20993, 301-796-
0684, FAX: 301-847-8443, [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background on Patient-Focused Drug Development

    FDA has selected Chagas disease as the focus of a 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's performance 
commitments made as part of the authorization of PDUFA V under Title I 
of the Food and Drug Safety and Innovation Act (Public Law 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 has committed to obtain the patient perspective in 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 community, and other interested 
stakeholders.
    On April 11, 2013, FDA published a notice (78 FR 21613) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years (FYs) 2013 through 2015, the first 3 years of the 5-year PDUFA V 
time frame. To develop the list of disease areas, the Agency used 
several criteria that were outlined in the April 11 notice. The Agency 
obtained public comment on these criteria and potential disease areas 
through a notice for public comment published in the Federal Register 
on September 24, 2012 (77 FR 58849), and through a public meeting held 
on October 25, 2012. In selecting the disease areas, FDA carefully 
considered the public comments received and the perspectives of its 
review divisions. On October 8, 2014, FDA published a notice in the 
Federal Register to initiate another public process to determine the 
disease areas for FYs 2016 through 2017 (79 FR 60857). 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

A. Purpose and Scope of the Meeting

    As part of Patient-Focused Drug Development, FDA will obtain 
patient and patient stakeholder input on symptoms of Chagas disease 
(American trypanosomiasis) that matter most to patients and on current 
approaches to treating Chagas disease. When left untreated, acute 
Chagas disease may progress to chronic Chagas disease. There are 
currently no FDA-approved drug therapies to treat acute or chronic 
Chagas disease. 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 and organized by 
topic. For each topic, a brief patient panel discussion will begin the 
dialogue, followed by a facilitated discussion inviting comments from 
other patients and patient stakeholders. In addition to input received 
through this public meeting, FDA is interested in receiving patient 
input addressing these

[[Page 73326]]

questions through written comments that can be submitted to the public 
docket (see ADDRESSES). When submitting comments, if you are commenting 
on behalf of a child, please indicate that and 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 worries you most about your condition?
    2. Do you experience symptoms because of your condition? If so, of 
all the symptoms that you experience, which one to three symptoms have 
the most significant impact on your life? (Examples may include 
irregular heartbeat, shortness of breath, difficulty swallowing, 
stomach pain, or constipation.)
    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 
condition? (Examples of activities may include sleeping through the 
night, daily hygiene, driving, being a blood or organ donor, or for 
women in reproductive age concern about getting pregnant and 
transmitting the infection to your children, etc.)
    4. How have your condition and its symptoms changed over time?
    5. Do your symptoms come and go? If so, do you know of anything 
that makes your symptoms better or worse?
Topic 2: Patient Perspectives on Current Approaches To Treat Chagas 
Disease
    1. What are you currently doing to help treat your condition? 
(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. 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, length of treatment, 
number of pills to take daily, going to the hospital for frequent 
checkups or treatment, restrictions on driving, potential consequences 
to your health and your child's health during pregnancy, etc.)
    3. What specific things would you look for in an ideal treatment 
for your condition?
    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 Chagas disease and identifying topics for 
future discussion. Discussion topics for the afternoon will include 
designs and endpoints for clinical trials as well as appropriate trial 
populations.

B. Meeting Attendance and Participation

    If you wish to attend the meeting, visit http://chagasdiseasepatientfocused.eventbrite.com. Please register for the 
meeting by April 20, 2015. If you are unable to attend the meeting in 
person, you can register to view a live Webcast. You will be asked to 
indicate in your registration whether 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 meetings will be based on space availability. If you need 
special accommodations because of a 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 must indicate in their registration which 
topic(s) they wish to address. These patients also must send a brief 
summary of responses to the topic questions by April 10, 2015, to 
[email protected]. 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 on a first-come, 
first-served basis.

III. Comments

    Regardless of attendance at the Patient-Focused Drug Development 
meeting, you can submit electronic or written comments, including 
responses to the questions pertaining to Topics 1 and 2, to the public 
docket (see ADDRESSES) by June 29, 2015. 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.

III. Transcripts

    As soon as a transcript is available, FDA will post it at http://www.fda.gov/Drugs/NewsEvents/ucm420130.htm.

    Dated: December 3, 2014.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2014-28828 Filed 12-9-14; 8:45 am]
BILLING CODE 4164-01-P