80 FR 42503 - Public Meeting on Patient-Focused Drug Development for Huntington's and Parkinson's Diseases

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 137 (July 17, 2015)

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 Huntington's disease and Parkinson's disease. 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 Huntington's disease and Parkinson's disease on daily life and patient views on treatment approaches. Although these are both neurological diseases, since they are quite distinct, FDA will structure this public meeting into two distinct sessions. The morning session, scheduled from 9 a.m. to 1 p.m., will be devoted to hearing patient perspectives on the impact of Huntington's disease on daily life and their views on currently available treatment approaches. The afternoon session, scheduled from 1 p.m. to 5 p.m., will be devoted to obtaining patient perspectives on the impact of Parkinson's disease on daily life and patient views on currently available treatment approaches.

[Federal Register Volume 80, Number 137 (Friday, July 17, 2015)]
[Notices]
[Pages 42503-42505]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-17556]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0967]


Public Meeting on Patient-Focused Drug Development for 
Huntington's and Parkinson's Diseases

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 Huntington's disease and Parkinson's 
disease. 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 Huntington's 
disease and Parkinson's disease on daily life and patient views on 
treatment approaches. Although these are both neurological diseases, 
since they are quite distinct, FDA will structure this public meeting 
into two distinct sessions. The morning session, scheduled from 9 a.m. 
to 1 p.m., will be

[[Page 42504]]

devoted to hearing patient perspectives on the impact of Huntington's 
disease on daily life and their views on currently available treatment 
approaches. The afternoon session, scheduled from 1 p.m. to 5 p.m., 
will be devoted to obtaining patient perspectives on the impact of 
Parkinson's disease on daily life and patient views on currently 
available treatment approaches.

DATES: The public meeting will be held on September 22, 2015, from 9 
a.m. to 5 p.m. Registration to attend the meeting must be received by 
September 14, 2015 (see SUPPLEMENTARY INFORMATION for instructions). 
Register here to attend the meeting: https://pfddhuntingtonparkinson.eventbrite.com. Submit electronic or written 
comments to the public docket by November 23, 2015.

ADDRESSES: The 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. 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.
    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, 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/ucm451807.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 Huntington's disease (HD) and Parkinson's disease 
(PD) 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 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 April 11, 2013, FDA published a notice (78 FR 21613) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years (FY) 2013 to 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. By 
the end of FY 2015, FDA will initiate a second public process for 
determining the disease areas for FYs 2016 to 2017. 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

    The purpose of this Patient-Focused Drug Development meeting is to 
obtain input on the symptoms and other impacts of HD and PD that matter 
most to patients, as well as perspectives on current approaches to 
treating these conditions. HD is a fatal genetic disorder that causes 
the progressive degeneration of nerve cells in the brain, resulting in 
uncontrolled movements, loss of intellectual faculties, and emotional 
disturbance. Each child of an HD parent has a 50-50 chance of 
inheriting the HD gene, and a person who inherits the HD gene will 
eventually develop the disease. Physicians may prescribe a number of 
medications to help control emotional and movement problems associated 
with HD. While medicines may help keep these clinical symptoms under 
control, there is no current treatment to stop or reverse the course of 
the disease.
    PD belongs to a group of conditions called motor system disorders, 
which are the result of the loss of dopamine-producing brain cells. As 
nerve cells become impaired or die, individuals begin to experience 
tremor, muscle rigidity or stiffness, slowing of movement, and impaired 
balance and coordination. The cause of PD is unknown, but factors such 
as genetics and environmental triggers may play a role. Although there 
is no cure for PD, medications can help manage the levels of dopamine 
and other neurotransmitters in the brain to improve symptoms. Deep 
brain stimulation is a surgery that may also be used to manage symptoms 
if medications are not effective.
    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).

B. Huntington's Disease Discussion Questions

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: ability to control movements, 
balance/coordination, difficulty concentrating, sleeping, mood/
behavior, 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 sleeping through the 
night, daily bathing/showering, cooking, eating, dressing, shopping, 
etc.)
     How do your symptoms affect your daily life on the best 
days? On the worst days?
    3. How has your condition and its symptoms changed over time?

[[Page 42505]]

     Do your symptoms come and go? If so, do you know of 
anything that makes your symptoms better? Worse?
    4. How has your condition affected your social interactions, 
including relationships with family and friends?
    5. How has your condition affected your mood (for example: 
depression, apathy, patience/tolerance for frustration)?
Topic 2: Patients' Perspectives on Current Approaches To Treating HD
    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 and exercise.)
    (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 treat the most 
significant symptoms of your disease?
    (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 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 bothersome side effects, interacts with other 
medications, need to visit your doctor more frequently, etc.)
    4. Assuming there is no complete cure for your condition, what 
would you look for in an ideal treatment for your condition or a 
specific aspect of your condition?

C. Parkinson's Disease Discussion Questions

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 moving, pain, 
constipation, difficulty concentrating or remembering, daytime 
sleepiness, 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 daily hygiene, feeding, 
dressing, etc.)
     How do your symptoms affect your daily life on the best 
days? On the worst days?
    3. How has your ability to cope with symptoms changed over time?
     Do your symptoms come and go? If so, do you know of 
anything that makes your symptoms better? Worse?
    4. What worries you most about your condition?
    5. How has your condition affected your social interactions, 
including relationships with family and friends?
Topic 2: Patients' Perspectives on Current Approaches To Treating PD
    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 and exercise.)
     What specific symptoms do your treatments address (for 
example: depression, constipation, memory difficulty, sleepiness, 
ability to move)?
    2. How well does your current treatment regimen treat the most 
significant symptoms of your disease?
     How well do these treatments improve your ability to do 
specific activities that are important to you in your daily life?
    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 to visit your 
doctor or take medications frequently, cause sleepiness, etc.)
    4. Assuming there is no complete cure for your condition, what 
would you look for in an ideal treatment for your condition or a 
specific aspect of your condition?

III. Meeting Attendance and Participation

    If you wish to attend this meeting, visit https://pfddhuntingtonparkinson.eventbrite.com. Please register by September 
14, 2015. If you are unable to attend the meeting in person, you can 
register to view a live webcast of the meeting. When you register, you 
can indicate whether you plan to attend the morning session on HD, the 
afternoon session on PD, or both. You will also 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 8, 2015. 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.

IV. Comments

    Regardless if you attend the public meeting, you can submit 
electronic or written responses to the questions pertaining to HD 
Topics 1 and 2 and PD Topics 1 and 2 to the public docket (see 
ADDRESSES) by November 23, 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.

V. Transcripts

    As soon as a transcript is available, FDA will post it at http://www.fda.gov/Drugs/NewsEvents/ucm451807.htm.

    Dated: July 13, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-17556 Filed 7-16-15; 8:45 am]
 BILLING CODE 4164-01-P