80_FR_11496 80 FR 11455 - Pediatric Neurocognitive Workshop; Advancing the Development of Pediatric Therapeutics Public Workshop

80 FR 11455 - Pediatric Neurocognitive Workshop; Advancing the Development of Pediatric Therapeutics Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 41 (March 3, 2015)

Page Range11455-11456
FR Document2015-04376

The Food and Drug Administration's (FDA) Division of Gastroenterology and Inborn Errors Products Division and Division of Pediatric and Maternal Health in the Center for Drug Evaluation and Research, and the Office of Pediatric Therapeutics in the Office of the Commissioner are announcing a 2-day public workshop. Day 1 of the workshop is entitled ``Assessment of Neurocognitive Outcomes in the Inborn Errors of Metabolism''. Day 2 of the workshop is entitled, ``Advancing the Development of Pediatric Therapeutics: Assessment of Pediatric Neurocognitive Outcomes''. The purpose of this 2-day workshop is to provide a forum to consider issues related to advancing pediatric regulatory science in the evaluation of neurocognitive outcomes in pediatric patients.

Federal Register, Volume 80 Issue 41 (Tuesday, March 3, 2015) 
[Federal Register Volume 80, Number 41 (Tuesday, March 3, 2015)]
[Notices]
[Pages 11455-11456]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-04376]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-N-0001]


Pediatric Neurocognitive Workshop; Advancing the Development of 
Pediatric Therapeutics Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration's (FDA) Division of 
Gastroenterology and Inborn Errors Products Division and Division of 
Pediatric and Maternal Health in the Center for Drug Evaluation and 
Research, and the Office of Pediatric Therapeutics in the Office of the 
Commissioner are announcing a 2-day public workshop. Day 1 of the 
workshop is entitled ``Assessment of Neurocognitive Outcomes in the 
Inborn Errors of Metabolism''. Day 2 of the workshop is entitled, 
``Advancing the Development of Pediatric Therapeutics: Assessment of 
Pediatric Neurocognitive Outcomes''. The purpose of this 2-day workshop 
is to provide a forum to consider issues related to advancing pediatric 
regulatory science in the evaluation of neurocognitive outcomes in 
pediatric patients.

DATES: The public workshop will be held on April 16 and 17, 2015, from 
8 a.m. to 5 p.m.

ADDRESSES: The public workshop will be held in the FDA White Oak 
Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great 
Room (Rm. 1503A), Silver Spring, MD 20993-0002. Entrance for the public 
workshop participants (non-FDA employees) is through Building 1 where 
routine security check procedures will be performed. For parking and 
security information, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

FOR FURTHER INFORMATION CONTACT: For questions regarding Day 1 of the 
workshop, contact Richard (Wes) Ishihara, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Silver Spring, MD 20993-0002, 301-796-0069, 
[email protected].
    For questions regarding Day 2 of the workshop, contact Denise Pica-
Branco, Center for Drug Evaluation and Research, Food and Drug

[[Page 11456]]

Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-
0002,Telephone: 301-796-1732, FAX: 301-796-9858, 
[email protected].

SUPPLEMENTARY INFORMATION: The first day of the workshop will focus on 
approaches for assessing the efficacy of therapeutic products on 
neurocognitive outcomes in patients diagnosed with inborn errors of 
metabolism disorders. The session will address the role of natural 
history studies and methodological approaches for selecting appropriate 
assessment scales and standardizing neurocognitive assessments. The 
second day of the workshop will discuss identification of signals in 
animal studies and clinical trials that warrant further clinical 
investigation and testing that may be predictive of neurocognitive 
outcome in children. Additionally, strategies and methods to address 
the challenges of assessing long-term neurocognitive outcomes for 
products used to treat pediatric patients will be discussed.

Participation in the Public Workshop

    Registration: There is no fee to attend the public workshop, but 
attendees should register in advance. Space is limited, and 
registration will be on a first-come, first-served basis. Persons 
interested in attending this workshop must register online at 
[email protected] before March 31, 2015. For those 
without Internet access, please contact Denise Pica-Branco (see FOR 
FURTHER INFORMATION CONTACT) to register. Onsite registration will not 
be available.
    If you need special accommodations due to a disability, please 
contact Denise Pica-Branco (see FOR FURTHER INFORMATION CONTACT) at 
least 7 days in advance.
    Transcripts: Transcripts of the workshop will be available for 
review at the Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, and 
at http://www.regulations.gov approximately 30 days after the workshop. 
A transcript will also be available in either hard copy or on CD-ROM, 
after submission of a Freedom of Information request. Send written 
requests to the Division of Freedom of Information (ELEM-1029), Food 
and Drug Administration, 12420 Parklawn Dr., Element Bldg., Rockville, 
MD 20857. Send faxed requests to 301-827-9267.

    Dated: February 24, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-04376 Filed 3-2-15; 8:45 am]
BILLING CODE 4164-01-P

Federal Register / Vol. 80, No. 41 / Tuesday, March 3, 2015 / Notices 11455 DATES: Dates and Time: The scientific www.regulations.gov. You may submit a DEPARTMENT OF HEALTH AND workshop will be held on March 20, request to obtain a hard copy by sending HUMAN SERVICES 2015, from 8:30 a.m. to 5:30 p.m. a request to the Division of Freedom of ADDRESSES: The scientific workshop Information (ELEM–1029), Office of Food and Drug Administration will be held at FDA’s White Oak Management Programs, Food and Drug [Docket No. FDA–2015–N–0001] Campus, 10903 New Hampshire Ave., Administration, 12420 Parklawn Dr., Bldg. 31 Conference Center, the Great Element Bldg., Rockville, MD 20857. Pediatric Neurocognitive Workshop; Room (Rm. 1503A), Silver Spring, MD Advancing the Development of 20993–0002. Participants must enter SUPPLEMENTARY INFORMATION: FDA and Pediatric Therapeutics Public through Building 1 and undergo NIH are cosponsoring this scientific Workshop security screening. For parking and workshop to discuss current methodologies being used in drug AGENCY: Food and Drug Administration, security information, please refer to development and scientific research for HHS. http://www.fda.gov/AboutFDA/ WorkingatFDA/BuildingsandFacilities/ DMD. Recent scientific advances ACTION: Notice of public workshop. WhiteOakCampusInformation/ present an opportunity for the SUMMARY: The Food and Drug ucm241740.htm. development and validation of robust Administration’s (FDA) Division of Contact Persons: Mary Gross, Center methods for the objective, reliable, and Gastroenterology and Inborn Errors for Drug Evaluation and Research, Food quantitative measurement of DMD- Products Division and Division of and Drug Administration, 10903 New associated proteins. Pediatric and Maternal Health in the Hampshire Ave., Silver Spring, MD Center for Drug Evaluation and I. Background 20993–0002, 301–796–3519, Research, and the Office of Pediatric mary.gross@fda.hhs.gov; or Georgiann Dystrophinopathies result from Therapeutics in the Office of the Ienzi, Center for Drug Evaluation and genetic mutations in the dystrophin Commissioner are announcing a 2-day Research, Food and Drug gene that decrease dystrophin protein public workshop. Day 1 of the workshop Administration, 10903 New Hampshire expression levels and result in altered is entitled ‘‘Assessment of Ave., Silver Spring, MD 20993–0002, dystrophin function. These changes can Neurocognitive Outcomes in the Inborn 301–796–3515, georgiann.ienzi@ Errors of Metabolism’’. Day 2 of the lead to muscle degeneration and, in fda.hhs.gov. workshop is entitled, ‘‘Advancing the many patients, downstream pathologies If you need special accommodations Development of Pediatric Therapeutics: including inflammation and fibrosis that due to a disability, contact Mary Gross Assessment of Pediatric Neurocognitive or Georgiann Ienzi at least 7 days in interfere with muscle regeneration, loss of movement, orthopedic complications, Outcomes’’. The purpose of this 2-day advance. workshop is to provide a forum to Registration: The scientific workshop and ultimately respiratory and cardiac failure. consider issues related to advancing is free and seating will be on a first- pediatric regulatory science in the come, first-served basis. It may be II. Scope of the Scientific Workshop evaluation of neurocognitive outcomes necessary to limit both the number of in pediatric patients. attendees from individual organizations The workshop will include sessions DATES: The public workshop will be and the total number of attendees based which will focus on current held on April 16 and 17, 2015, from 8 on space limitations. Email registrations technologies used in the detection of a.m. to 5 p.m. should be sent to Dystrophin_ dystrophin. Presentations will provide Workshop@fda.hhs.gov by March 17, ADDRESSES: The public workshop will overviews of the technologies (including 2015. If you cannot attend in person, the be held in the FDA White Oak Campus, limitations, detection sensitivities, meeting will be Webcast live. 10903 New Hampshire Ave., Bldg. 31 linearity, and reproducibility). A panel Conference Center, the Great Room (Rm. Information about how to access the discussion will help identify Webcast will be located at: http:// 1503A), Silver Spring, MD 20993–0002. development challenges for each Entrance for the public workshop www.fda.gov/Drugs/NewsEvents/ method. Muscle biopsy collection, ucm432429.htm. participants (non-FDA employees) is sample handling, reference materials, through Building 1 where routine Comments and Meeting Summary: and image analysis will also be security check procedures will be Submit electronic comments to http:// discussed. performed. For parking and security www.regulations.gov by May 20, 2015. Submit written comments to the FDA will post the agenda and other information, please refer to http:// Division of Dockets Management (HFA– background material approximately 2 www.fda.gov/AboutFDA/ 305), Food and Drug Administration, days before the public scientific WorkingatFDA/BuildingsandFacilities/ 5630 Fishers Lane, Rm. 1061, Rockville, workshop at: http://www.fda.gov/Drugs/ WhiteOakCampusInformation/ MD 20852. Please identify your NewsEvents/ucm432429.htm. ucm241740.htm. comments with the docket number Dated: February 24, 2015. FOR FURTHER INFORMATION CONTACT: For found in brackets in the heading of this questions regarding Day 1 of the Leslie Kux, document. It is only necessary to send workshop, contact Richard (Wes) one set of comments. Received Associate Commissioner for Policy. Ishihara, Center for Drug Evaluation and comments may be seen in the Division [FR Doc. 2015–04384 Filed 3–2–15; 8:45 am] Research, Food and Drug mstockstill on DSK4VPTVN1PROD with NOTICES of Dockets Management between 9 a.m. BILLING CODE 4164–01–P Administration, 10903 New Hampshire and 4 p.m., Monday through Friday, and Ave., Silver Spring, MD 20993–0002, will be posted to the docket at http:// 301–796–0069, richard.ishihara@ www.regulations.gov. fda.hhs.gov. A summary of the scientific For questions regarding Day 2 of the workshop’s highlights will be made workshop, contact Denise Pica-Branco, available for review at the Division of Center for Drug Evaluation and Dockets Management and at http:// Research, Food and Drug VerDate Sep<11>2014 19:24 Mar 02, 2015 Jkt 235001 PO 00000 Frm 00069 Fmt 4703 Sfmt 4703 E:\FR\FM\03MRN1.SGM 03MRN1

11456 Federal Register / Vol. 80, No. 41 / Tuesday, March 3, 2015 / Notices Administration, 10903 New Hampshire Dated: February 24, 2015. Agenda: To review and evaluate grant Ave., Silver Spring, MD 20993– Leslie Kux, applications. 0002,Telephone: 301–796–1732, FAX: Place: National Institutes of Health, Room Associate Commissioner for Policy. 4F100, 5601 Fishers Lane, Rockville, MD 301–796–9858, denise.picabranco@ [FR Doc. 2015–04376 Filed 3–2–15; 8:45 am] 20892. fda.hhs.gov. BILLING CODE 4164–01–P Contact Person: Jay R Radke, Ph.D., AIDS Review Branch, Scientific Review Program, SUPPLEMENTARY INFORMATION: The first DEA/NIAID/NIH/DHHS, Room 3G11B, 5601 day of the workshop will focus on DEPARTMENT OF HEALTH AND Fishers Lane, Rockville, MD 20892, 240–669– approaches for assessing the efficacy of HUMAN SERVICES 5046, jay.radke@nih.gov. therapeutic products on neurocognitive (Catalogue of Federal Domestic Assistance outcomes in patients diagnosed with National Institutes of Health Program Nos. 93.855, Allergy, Immunology, inborn errors of metabolism disorders. and Transplantation Research; 93.856, The session will address the role of National Institute of Allergy and Microbiology and Infectious Diseases Infectious Diseases; Notice of Closed Research, National Institutes of Health, HHS) natural history studies and methodological approaches for selecting Meetings Dated: February 25, 2015. appropriate assessment scales and Pursuant to section 10(d) of the David Clary, standardizing neurocognitive Federal Advisory Committee Act, as Program Analyst, Office of Federal Advisory assessments. The second day of the amended (5 U.S.C. App.), notice is Committee Policy. workshop will discuss identification of hereby given of the following meetings. [FR Doc. 2015–04328 Filed 3–2–15; 8:45 am] signals in animal studies and clinical The meetings will be closed to the BILLING CODE 4140–01–P trials that warrant further clinical public in accordance with the investigation and testing that may be provisions set forth in sections predictive of neurocognitive outcome in 552b(c)(4) and 552b(c)(6), title 5 U.S.C., DEPARTMENT OF HEALTH AND children. Additionally, strategies and as amended. The grant applications and HUMAN SERVICES methods to address the challenges of the discussions could disclose National Institutes of Health assessing long-term neurocognitive confidential trade secrets or commercial outcomes for products used to treat property such as patentable material, Center for Scientific Review; Notice of pediatric patients will be discussed. and personal information concerning Closed Meetings individuals associated with the grant Participation in the Public Workshop applications, the disclosure of which Pursuant to section 10(d) of the would constitute a clearly unwarranted Federal Advisory Committee Act, as Registration: There is no fee to attend amended (5 U.S.C. App.), notice is invasion of personal privacy. the public workshop, but attendees hereby given of the following meetings. should register in advance. Space is Name of Committee: National Institute of The meetings will be closed to the limited, and registration will be on a Allergy and Infectious Diseases Special Emphasis Panel; Immunobiology of public in accordance with the first-come, first-served basis. Persons Xenotransplantation (U01, U19). provisions set forth in sections interested in attending this workshop Date: March 23–24, 2015. 552b(c)(4) and 552b(c)(6), title 5 U.S.C., must register online at neurocognitive_ Time: 9:00 a.m. to 6:00 p.m. as amended. The grant applications and workshop@fda.hhs.gov before March 31, Agenda: To review and evaluate grant the discussions could disclose 2015. For those without Internet access, applications. confidential trade secrets or commercial please contact Denise Pica-Branco (see Place: National Institutes of Health, Room property such as patentable material, FOR FURTHER INFORMATION CONTACT) to 3F100, 5601 Fishers Lane, Rockville, MD and personal information concerning 20892, (Telephone Conference Call). individuals associated with the grant register. Onsite registration will not be Contact Person: Nancy Vazquez- available. Maldonado, Ph.D., Scientific Review Officer, applications, the disclosure of which Scientific Review Program, DEA/NIAID/NIH/ would constitute a clearly unwarranted If you need special accommodations DHHS, 6700B Rockledge Drive, MSC–7616, invasion of personal privacy. due to a disability, please contact Denise Pica-Branco (see FOR FURTHER Bethesda, MD 20892–7616, 301–496–3253, Name of Committee: Center for Scientific nvazquez@niaid.nih.gov. Review Special Emphasis Panel; Program INFORMATION CONTACT) at least 7 days in Name of Committee: National Institute of Project: Structure/Function studies of advance. Allergy and Infectious Diseases Special Secondary Transporters in a Lipid Transcripts: Transcripts of the Emphasis Panel; Mucosal Environment and Environment. workshop will be available for review at HIV Prevention (MEHP II (R01)). Date: March 23–25, 2015. the Division of Dockets Management Date: March 23–24, 2015. Time: 10:00 a.m. to 5:00 p.m. (HFA–305), Food and Drug Time: 12:00 p.m. to 4:00 p.m. Agenda: To review and evaluate grant Agenda: To review and evaluate grant applications. Administration, 5630 Fishers Lane, Rm. applications. Place: National Institutes of Health, 6701 1061, Rockville, MD 20852, and at Place: National Institutes of Health, Room Rockledge Drive, Bethesda, MD 20892, http://www.regulations.gov 3C100, 5601 Fishers Lane, Rockville, MD (Virtual Meeting). approximately 30 days after the 20892, (Telephone Conference Call). Contact Person: Albert Wang, Scientific workshop. A transcript will also be Contact Person: Kelly Y. Poe, Ph.D., Review Officer, Center for Scientific Review, available in either hard copy or on CD– Scientific Review Officer, Scientific Review National Institutes of Health, 6701 Rockledge ROM, after submission of a Freedom of Program, DEA/NIAID/NIH/DHHS, 5601 Drive, Room 4146, MSC 7806, Bethesda, MD mstockstill on DSK4VPTVN1PROD with NOTICES Information request. Send written Fishers Lane, Rockville, MD 20892, 240–669– 20892, 301–435–1016, wangca@csr.nih.gov. 5036, Kelly.poe@nih.gov. Name of Committee: Center for Scientific requests to the Division of Freedom of Name of Committee: National Institute of Review Special Emphasis Panel; Member Information (ELEM–1029), Food and Allergy and Infectious Diseases Special Conflict: AIDS and AIDS Related Research. Drug Administration, 12420 Parklawn Emphasis Panel; Innovative Assays to Date: March 24–25, 2015. Dr., Element Bldg., Rockville, MD Quantify the Latent HIV Reservoir (R01). Time: 10:00 a.m. to 5:00 p.m. 20857. Send faxed requests to 301–827– Date: March 26, 2015. Agenda: To review and evaluate grant 9267. Time: 9:00 a.m. to 6:00 p.m. applications. VerDate Sep<11>2014 19:24 Mar 02, 2015 Jkt 235001 PO 00000 Frm 00070 Fmt 4703 Sfmt 4703 E:\FR\FM\03MRN1.SGM 03MRN1


Document Created: 2015-12-18 11:56:04
Document Modified: 2015-12-18 11:56:04
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice of public workshop.
DatesThe public workshop will be held on April 16 and 17, 2015, from 8 a.m. to 5 p.m.
ContactFor questions regarding Day 1 of the workshop, contact Richard (Wes) Ishihara, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-0069, [email protected]
FR Citation80 FR 11455 
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