Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data; Public Scientific Workshop; Request for Comments, 11454-11455 [2015-04384]

Download as PDF 11454 Federal Register / Vol. 80, No. 41 / Tuesday, March 3, 2015 / Notices TABLE 3—ESTIMATED ANNUAL REPORTING BURDEN FOR BIOLOGICS 1—Continued 312.120(b), Number of submissions to FDA of ‘‘supporting information’’ related to the use of foreign clinical studies not conducted under an IND ....................... 312.120(c), Number of waiver requests submitted to FDA related to the use of foreign clinical studies not conducted under an IND ...................................................... 312.130, Number of requests for disclosable information in an IND and for investigations involving an exception from informed consent under § 50.24 ............................ 312.310(b) and 312.305(b), Number of submissions related to expanded access and treatment of an individual patient .................................................................. 312.310(d), Number of submissions related to emergency use of an investigational new drug ..................... 312.315(c) and 312.305(b), Number of submissions related to expanded access and treatment of an intermediate-size patient population ..................................... 312.320(b), Number of submissions related to a treatment IND or treatment protocol ..................................... Total ............................................................................ 1 There Number of responses per respondent Number of respondents 21 CFR section Average burden per response Total annual responses Total hours 280 9.82 2,750 32 88,000 7 2.29 16 24 384 350 1.34 470 8 3,760 78 1.08 84 8 672 76 2.76 210 16 3,360 9 1 9 120 1,080 1 1 1 300 300 ........................ ........................ 3,254,062 ........................ .......................... are no capital costs or operating and maintenance costs associated with this collection of information. TABLE 4—ESTIMATED ANNUAL RECORDKEEPING BURDEN FOR BIOLOGICS 1 Number of recordkeepers 21 CFR section 312.52(a), Sponsor records for the transfer of obligations to a contract research organization ............................... 312.57, Sponsor recordkeeping showing the receipt, shipment, or other disposition of the investigational drug, and any financial interests .................................... 312.62(a), Investigator recordkeeping of the disposition of drugs .......................................................................... 312.62(b), Investigator recordkeeping of case histories of individuals ....................................................................... 312.160(a)(3), Records pertaining to the shipment of drugs for investigational use in laboratory research animals or in vitro tests ....................................................... 312.160(c), Shipper records of alternative disposition of unused drugs .................................................................. Total ............................................................................ Number of records per recordkeeper Total annual records Average burden per recordkeeping Total hours 75 1.40 105 2 210 335 2.70 904 100 90,400 453 1 453 40 18,120 453 1 453 40 18,120 111 1.40 155 * 0.50 78 111 1.40 155 * 0.50 78 ........................ .......................... ........................ ........................ 127,006 1 There are no capital costs or operating and maintenance costs associated with this collection of information. * Thirty (30) minutes. Dated: February 24, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015–04379 Filed 3–2–15; 8:45 am] Food and Drug Administration [Docket No. FDA–2015–N–0430] BILLING CODE 4164–01–P mstockstill on DSK4VPTVN1PROD with NOTICES DEPARTMENT OF HEALTH AND HUMAN SERVICES Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data; Public Scientific Workshop; Request for Comments AGENCY: Food and Drug Administration, HHS. Notice of public scientific workshop; request for comments. ACTION: The Food and Drug Administration (FDA) is announcing a public scientific VerDate Sep<11>2014 19:24 Mar 02, 2015 Jkt 235001 PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 workshop to discuss dystrophin protein quantification methodologies for human tissue. This workshop is being cosponsored by the National Institutes of Health (NIH). The purpose of the workshop is to discuss currently available methodologies and to identify scientific knowledge gaps and opportunities for improving dystrophin protein detection in the context of drug development. The intended audiences for this workshop are scientists and clinicians involved in the acquisition, measurement, and analysis of proteins associated with Duchenne Muscular Dystrophy (DMD). E:\FR\FM\03MRN1.SGM 03MRN1 Federal Register / Vol. 80, No. 41 / Tuesday, March 3, 2015 / Notices Dates and Time: The scientific workshop will be held on March 20, 2015, from 8:30 a.m. to 5:30 p.m. ADDRESSES: The scientific workshop will be held at FDA’s White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503A), Silver Spring, MD 20993–0002. Participants must enter through Building 1 and undergo security screening. For parking and security information, please refer to https://www.fda.gov/AboutFDA/ WorkingatFDA/BuildingsandFacilities/ WhiteOakCampusInformation/ ucm241740.htm. Contact Persons: Mary Gross, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993–0002, 301–796–3519, mary.gross@fda.hhs.gov; or Georgiann Ienzi, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993–0002, 301–796–3515, georgiann.ienzi@ fda.hhs.gov. If you need special accommodations due to a disability, contact Mary Gross or Georgiann Ienzi at least 7 days in advance. Registration: The scientific workshop is free and seating will be on a firstcome, first-served basis. It may be necessary to limit both the number of attendees from individual organizations and the total number of attendees based on space limitations. Email registrations should be sent to Dystrophin_ Workshop@fda.hhs.gov by March 17, 2015. If you cannot attend in person, the meeting will be Webcast live. Information about how to access the Webcast will be located at: https:// www.fda.gov/Drugs/NewsEvents/ ucm432429.htm. Comments and Meeting Summary: Submit electronic comments to https:// www.regulations.gov by May 20, 2015. Submit written comments to the Division of Dockets Management (HFA– 305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Please identify your comments with the docket number found in brackets in the heading of this document. It is only necessary to send one set of comments. 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 https:// www.regulations.gov. A summary of the scientific workshop’s highlights will be made available for review at the Division of Dockets Management and at https:// mstockstill on DSK4VPTVN1PROD with NOTICES DATES: VerDate Sep<11>2014 19:24 Mar 02, 2015 Jkt 235001 www.regulations.gov. You may submit a request to obtain a hard copy by sending a request to the Division of Freedom of Information (ELEM–1029), Office of Management Programs, Food and Drug Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857. FDA and NIH are cosponsoring this scientific workshop to discuss current methodologies being used in drug development and scientific research for DMD. Recent scientific advances present an opportunity for the development and validation of robust methods for the objective, reliable, and quantitative measurement of DMDassociated proteins. SUPPLEMENTARY INFORMATION: I. Background Dystrophinopathies result from genetic mutations in the dystrophin gene that decrease dystrophin protein expression levels and result in altered dystrophin function. These changes can lead to muscle degeneration and, in many patients, downstream pathologies including inflammation and fibrosis that interfere with muscle regeneration, loss of movement, orthopedic complications, and ultimately respiratory and cardiac failure. II. Scope of the Scientific Workshop The workshop will include sessions which will focus on current technologies used in the detection of dystrophin. Presentations will provide overviews of the technologies (including limitations, detection sensitivities, linearity, and reproducibility). A panel discussion will help identify development challenges for each method. Muscle biopsy collection, sample handling, reference materials, and image analysis will also be discussed. FDA will post the agenda and other background material approximately 2 days before the public scientific workshop at: https://www.fda.gov/Drugs/ NewsEvents/ucm432429.htm. Dated: February 24, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015–04384 Filed 3–2–15; 8:45 am] BILLING CODE 4164–01–P PO 00000 Frm 00069 Fmt 4703 Sfmt 4703 11455 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. 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 https:// www.fda.gov/AboutFDA/ WorkingatFDA/BuildingsandFacilities/ WhiteOakCampusInformation/ ucm241740.htm. SUMMARY: 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, richard.ishihara@ fda.hhs.gov. For questions regarding Day 2 of the workshop, contact Denise Pica-Branco, Center for Drug Evaluation and Research, Food and Drug FOR FURTHER INFORMATION CONTACT: E:\FR\FM\03MRN1.SGM 03MRN1

Agencies

[Federal Register Volume 80, Number 41 (Tuesday, March 3, 2015)]
[Notices]
[Pages 11454-11455]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2015-04384]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-N-0430]


Measuring Dystrophin in Dystrophinopathy Patients and 
Interpreting the Data; Public Scientific Workshop; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public scientific workshop; request for comments.

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

    The Food and Drug Administration (FDA) is announcing a public 
scientific workshop to discuss dystrophin protein quantification 
methodologies for human tissue. This workshop is being cosponsored by 
the National Institutes of Health (NIH). The purpose of the workshop is 
to discuss currently available methodologies and to identify scientific 
knowledge gaps and opportunities for improving dystrophin protein 
detection in the context of drug development. The intended audiences 
for this workshop are scientists and clinicians involved in the 
acquisition, measurement, and analysis of proteins associated with 
Duchenne Muscular Dystrophy (DMD).

[[Page 11455]]


DATES: Dates and Time: The scientific workshop will be held on March 
20, 2015, from 8:30 a.m. to 5:30 p.m.

ADDRESSES: The scientific workshop will be held at FDA's White Oak 
Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great 
Room (Rm. 1503A), Silver Spring, MD 20993-0002. Participants must enter 
through Building 1 and undergo security screening. For parking and 
security information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Contact Persons: Mary Gross, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Silver Spring, MD 20993-0002, 301-796-3519, mary.gross@fda.hhs.gov; or 
Georgiann Ienzi, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 
301-796-3515, georgiann.ienzi@fda.hhs.gov.
    If you need special accommodations due to a disability, contact 
Mary Gross or Georgiann Ienzi at least 7 days in advance.
    Registration: The scientific workshop is free and seating will be 
on a first-come, first-served basis. It may be necessary to limit both 
the number of attendees from individual organizations and the total 
number of attendees based on space limitations. Email registrations 
should be sent to Dystrophin_Workshop@fda.hhs.gov by March 17, 2015. If 
you cannot attend in person, the meeting will be Webcast live. 
Information about how to access the Webcast will be located at: https://www.fda.gov/Drugs/NewsEvents/ucm432429.htm.
    Comments and Meeting Summary: Submit electronic comments to https://www.regulations.gov by May 20, 2015. Submit written comments to the 
Division of Dockets Management (HFA-305), Food and Drug Administration, 
5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Please identify your 
comments with the docket number found in brackets in the heading of 
this document. It is only necessary to send one set of comments. 
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 https://www.regulations.gov.
    A summary of the scientific workshop's highlights will be made 
available for review at the Division of Dockets Management and at 
https://www.regulations.gov. You may submit a request to obtain a hard 
copy by sending a request to the Division of Freedom of Information 
(ELEM-1029), Office of Management Programs, Food and Drug 
Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857.

SUPPLEMENTARY INFORMATION: FDA and NIH are cosponsoring this scientific 
workshop to discuss current methodologies being used in drug 
development and scientific research for DMD. Recent scientific advances 
present an opportunity for the development and validation of robust 
methods for the objective, reliable, and quantitative measurement of 
DMD-associated proteins.

I. Background

    Dystrophinopathies result from genetic mutations in the dystrophin 
gene that decrease dystrophin protein expression levels and result in 
altered dystrophin function. These changes can lead to muscle 
degeneration and, in many patients, downstream pathologies including 
inflammation and fibrosis that interfere with muscle regeneration, loss 
of movement, orthopedic complications, and ultimately respiratory and 
cardiac failure.

II. Scope of the Scientific Workshop

    The workshop will include sessions which will focus on current 
technologies used in the detection of dystrophin. Presentations will 
provide overviews of the technologies (including limitations, detection 
sensitivities, linearity, and reproducibility). A panel discussion will 
help identify development challenges for each method. Muscle biopsy 
collection, sample handling, reference materials, and image analysis 
will also be discussed.
    FDA will post the agenda and other background material 
approximately 2 days before the public scientific workshop at: https://www.fda.gov/Drugs/NewsEvents/ucm432429.htm.

    Dated: February 24, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-04384 Filed 3-2-15; 8:45 am]
BILLING CODE 4164-01-P
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