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)
Food and Drug Administration
Federal Register Volume 80, Issue 137 (July 17, 2015)
| Page Range | 42503-42505 | |
| FR Document | 2015-17556 |
[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] ----------------------------------------------------------------------- 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. ----------------------------------------------------------------------- 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, graham.thompson@fda.hhs.gov. 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 PatientFocused@fda.hhs.gov 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
![Federal Register / Vol. 80, No. 137 / Friday, July 17, 2015 / Notices 42503
information via the internet at http:// for approval. To comply with this Protection Act (Pub. L. 109–462),
www.regulations.gov. Submit written requirement, FDA is publishing notice requires the label of a nonprescription
comments on the collection of of the proposed collection of drug product marketed without an
information to the Dockets Management information set forth in this document. approved application in the United
Branch (HFA–305), Food and Drug With respect to the following States to include a domestic address or
Administration, 5630 Fishers Lane, Rm. collection of information, FDA invites domestic telephone number through
1061, Rockville, MD 20852. All comments on: (1) Whether the proposed which a manufacturer, packer, and
comments should be identified with the collection of information is necessary distributor may receive a report of a
docket number found in brackets in the for the proper performance of FDA’s serious adverse event associated with
heading of this document. functions, including whether the the product. The guidance document
information will have practical utility; contains questions and answers relating
FOR FURTHER INFORMATION CONTACT: FDA (2) the accuracy of FDA’s estimate of the
PRA Staff, Office of Operations, Food to this labeling requirement and
burden of the proposed collection of provides guidance to industry on the
and Drug Administration, 8455 information, including the validity of
Colesville Rd., COLE–14526, Silver following topics: (1) The meaning of
the methodology and assumptions used; ‘‘domestic address’’ for purposes of the
Spring, MD 20993–0002, PRAStaff@ (3) ways to enhance the quality, utility,
fda.hhs.gov. labeling requirements of section 502(x)
and clarity of the information to be of the FD&C Act; (2) FDA’s
SUPPLEMENTARY INFORMATION: Under the collected; and (4) ways to minimize the recommendation for the use of an
PRA (44 U.S.C. 3501–3520), Federal burden of the collection of information introductory statement before the
Agencies must obtain approval from the on respondents, including through the domestic address or phone number that
Office of Management and Budget use of automated collection techniques,
is required to appear on the product
(OMB) for each collection of when appropriate, and other forms of
label under section 502(x) of the FD&C
information they conduct or sponsor. information technology.
Act; and (3) FDA’s intent regarding
‘‘Collection of information’’ is defined Guidance for Industry on Questions enforcing the labeling requirements of
in 44 U.S.C. 3502(3) and 5 CFR and Answers Regarding the Labeling of section 502(x) of the FD&C Act.
1320.3(c) and includes Agency requests Nonprescription Human Drug Products
or requirements that members of the Description of Respondents:
Marketed Without an Approved Respondents to this collection of
public submit reports, keep records, or Application as Required by the Dietary
provide information to a third party. information are manufacturers, packers,
Supplement and Nonprescription Drug and distributors whose name (pursuant
Section 3506(c)(2)(A) of the PRA (44 Consumer Protection Act
U.S.C. 3506(c)(2)(A)) requires Federal to section 502(b)(1) of the FD&C Act)
Agencies to provide a 60-day notice in OMB Control Number 0910–0641— appears on the label of a
the Federal Register concerning each Extension nonprescription drug product marketed
proposed collection of information, Section 502(x) of the FD&C Act (21 in the United States without an
including each proposed extension of an U.S.C. 352(x)), which was added by the approved application.
existing collection of information, Dietary Supplement and FDA estimates the burden of this
before submitting the collection to OMB Nonprescription Drug Consumer collection of information as follows:
TABLE 1—ESTIMATED ANNUAL THIRD-PARTY DISCLOSURE BURDEN 1
Number of Average
Number disclosures Total annual
Activity burden per Total hours
of respondents per disclosures disclosure
respondent
Including a domestic address or phone number and a
statement of its purpose on OTC drug labeling (21
U.S.C. 502(x)) .................................................................. 300 3 900 4 3,600
1 There are no capital costs or operating and maintenance costs associated with this collection of information.
Dated: July 10, 2015. DEPARTMENT OF HEALTH AND Patient-Focused Drug Development for
Leslie Kux, HUMAN SERVICES Huntington’s disease and Parkinson’s
Associate Commissioner for Policy. disease. Patient-Focused Drug
Food and Drug Administration Development is part of FDA’s
[FR Doc. 2015–17558 Filed 7–16–15; 8:45 am]
BILLING CODE 4164–01–P [Docket No. FDA–2012–N–0967] performance commitments made as part
of the fifth authorization of the
Public Meeting on Patient-Focused Prescription Drug User Fee Act (PDUFA
Drug Development for Huntington’s V). The public meeting is intended to
and Parkinson’s Diseases allow FDA to obtain patient
AGENCY: Food and Drug Administration, perspectives on the impact of
Huntington’s disease and Parkinson’s
srobinson on DSK5SPTVN1PROD with NOTICES
HHS.
disease on daily life and patient views
ACTION: Notice of public meeting;
on treatment approaches. Although
request for comments.
these are both neurological diseases,
SUMMARY: The Food and Drug since they are quite distinct, FDA will
Administration (FDA or Agency) is structure this public meeting into two
announcing a public meeting and an distinct sessions. The morning session,
opportunity for public comment on scheduled from 9 a.m. to 1 p.m., will be
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![Federal Register / Vol. 80, No. 137 / Friday, July 17, 2015 / Notices 42505
• Do your symptoms come and go? If 3. How has your ability to cope with accommodations because of a disability,
so, do you know of anything that makes symptoms changed over time? please contact Graham Thompson (see
your symptoms better? Worse? • Do your symptoms come and go? If FOR FURTHER INFORMATION CONTACT) at
4. How has your condition affected so, do you know of anything that makes least 7 days before the meeting.
your social interactions, including your symptoms better? Worse? Patients who are interested in
relationships with family and friends? 4. What worries you most about your presenting comments as part of the
5. How has your condition affected condition? initial panel discussions will be asked
your mood (for example: depression, 5. How has your condition affected to indicate in their registration which
apathy, patience/tolerance for your social interactions, including topic(s) they wish to address. These
frustration)? relationships with family and friends? patients also must send to
Topic 2: Patients’ Perspectives on Topic 2: Patients’ Perspectives on PatientFocused@fda.hhs.gov a brief
Current Approaches To Treating HD Current Approaches To Treating PD summary of responses to the topic
questions by September 8, 2015.
1. What are you currently doing to 1. What are you currently doing to Panelists will be notified of their
help treat your condition or its help treat your condition or its selection approximately 7 days before
symptoms? (Examples may include symptoms? (Examples may include the public meeting. We will try to
prescription medicines, over-the- prescription medicines, over-the- accommodate all patients and patient
counter products, and other therapies counter products, and other therapies stakeholders who wish to speak, either
including non-drug therapies such as including non-drug therapies such as through the panel discussion or
diet modification and exercise.) diet modification and exercise.) audience participation; however, the
(a) What specific symptoms do your • What specific symptoms do your
duration of comments may be limited by
treatments address? treatments address (for example:
time constraints.
(b) How has your treatment regimen depression, constipation, memory
changed over time, and why? difficulty, sleepiness, ability to move)? IV. Comments
2. How well does your current 2. How well does your current
Regardless if you attend the public
treatment regimen treat the most treatment regimen treat the most
meeting, you can submit electronic or
significant symptoms of your disease? significant symptoms of your disease?
• How well do these treatments written responses to the questions
(a) How well do these treatments pertaining to HD Topics 1 and 2 and PD
improve your ability to do specific improve your ability to do specific
activities that are important to you in Topics 1 and 2 to the public docket (see
activities that are important to you in ADDRESSES) by November 23, 2015.
your daily life? your daily life?
3. What are the most significant Received comments may be seen in the
(b) How well have these treatments Division of Dockets Management
worked for you as your condition has downsides to your current treatments,
and how do they affect your daily life? between 9 a.m. and 4 p.m., Monday
changed over time? through Friday, and will be posted to
3. What are the most significant (Examples of downsides may include
bothersome side effects, need to visit the docket at http://
downsides to your current treatments, www.regulations.gov.
and how do they affect your daily life? your doctor or take medications
(Examples of downsides may include frequently, cause sleepiness, etc.) V. Transcripts
4. Assuming there is no complete cure
bothersome side effects, interacts with As soon as a transcript is available,
for your condition, what would you
other medications, need to visit your FDA will post it at http://www.fda.gov/
look for in an ideal treatment for your
doctor more frequently, etc.) Drugs/NewsEvents/ucm451807.htm.
4. Assuming there is no complete cure condition or a specific aspect of your
for your condition, what would you condition? Dated: July 13, 2015.
look for in an ideal treatment for your III. Meeting Attendance and Leslie Kux,
condition or a specific aspect of your Participation Associate Commissioner for Policy.
condition? If you wish to attend this meeting,
[FR Doc. 2015–17556 Filed 7–16–15; 8:45 am]
BILLING CODE 4164–01–P
C. Parkinson’s Disease Discussion visit https://pfddhuntingtonparkinson.
Questions eventbrite.com. Please register by
September 14, 2015. If you are unable to DEPARTMENT OF HEALTH AND
Topic 1: Disease Symptoms and Daily attend the meeting in person, you can
Impacts That Matter Most to Patients HUMAN SERVICES
register to view a live webcast of the
1. Of all the symptoms that you meeting. When you register, you can Food and Drug Administration
experience because of your condition, indicate whether you plan to attend the
which one to three symptoms have the morning session on HD, the afternoon [Docket No. FDA–2015–N–0001]
most significant impact on your life? session on PD, or both. You will also be
(Examples may include difficulty asked to indicate in your registration if Vaccines and Related Biological
moving, pain, constipation, difficulty you plan to attend in person or via the Products Advisory Committee; Notice
concentrating or remembering, daytime webcast. Seating will be limited, so of Meeting
sleepiness, etc.) early registration is recommended.
2. Are there specific activities that are Registration is free and will be on a first- AGENCY: Food and Drug Administration,
important to you but that you cannot do come, first-served basis. However, FDA HHS.
srobinson on DSK5SPTVN1PROD with NOTICES
at all or as fully as you would like may limit the number of participants ACTION: Notice.
because of your condition? (Examples of from each organization based on space
activities may include daily hygiene, limitations. Registrants will receive This notice announces a forthcoming
feeding, dressing, etc.) confirmation once they have been meeting of a public advisory committee
• How do your symptoms affect your accepted. Onsite registration on the day of the Food and Drug Administration
daily life on the best days? On the worst of the meeting will be based on space (FDA). The meeting will be open to the
days? availability. If you need special public.
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Document Created: 2018-02-23 09:21:33
Document Modified: 2018-02-23 09:21:33
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice of public meeting; request for comments. | |
| 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. | |
| 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] | |
| FR Citation | 80 FR 42503 |
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