80 FR 11454 - Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data; Public Scientific Workshop; Request for Comments

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

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

Page Range		11454-11455
FR Document		2015-04384

Submit electronic comments to http:// 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 http://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 http://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.

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

[Federal Register Volume 80, Number 41 (Tuesday, March 3, 2015)]
[Notices]
[Pages 11454-11455]
From the Federal Register Online  [www.thefederalregister.org]
[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 http://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, [email protected]; 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, [email protected].
    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 [email protected] 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: http://www.fda.gov/Drugs/NewsEvents/ucm432429.htm.
    Comments and Meeting Summary: Submit electronic comments to http://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 http://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 
http://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: http://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

11454 Federal Register / Vol. 80, No. 41 / Tuesday, March 3, 2015 / Notices

TABLE 3—ESTIMATED ANNUAL REPORTING BURDEN FOR BIOLOGICS 1—Continued
Number of Average
Number of Total annual
21 CFR section responses per burden per Total hours
respondents responses
respondent response

312.120(b), Number of submissions to FDA of ‘‘sup-
porting information’’ related to the use of foreign clin-
ical studies not conducted under an IND ....................... 280 9.82 2,750 32 88,000
312.120(c), Number of waiver requests submitted to FDA
related to the use of foreign clinical studies not con-
ducted under an IND ...................................................... 7 2.29 16 24 384
312.130, Number of requests for disclosable information
in an IND and for investigations involving an exception
from informed consent under § 50.24 ............................ 350 1.34 470 8 3,760
312.310(b) and 312.305(b), Number of submissions re-
lated to expanded access and treatment of an indi-
vidual patient .................................................................. 78 1.08 84 8 672
312.310(d), Number of submissions related to emer-
gency use of an investigational new drug ..................... 76 2.76 210 16 3,360
312.315(c) and 312.305(b), Number of submissions re-
lated to expanded access and treatment of an inter-
mediate-size patient population ..................................... 9 1 9 120 1,080
312.320(b), Number of submissions related to a treat-
ment IND or treatment protocol ..................................... 1 1 1 300 300

Total ............................................................................ ........................ .......................... ........................ ........................ 3,254,062
1 There 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 Average
Number of Total annual
21 CFR section records per burden per Total hours
recordkeepers records
recordkeeper recordkeeping

312.52(a), Sponsor records for the transfer of obligations
to a contract research organization ............................... 75 1.40 105 2 210
312.57, Sponsor recordkeeping showing the receipt,
shipment, or other disposition of the investigational
drug, and any financial interests .................................... 335 2.70 904 100 90,400
312.62(a), Investigator recordkeeping of the disposition
of drugs .......................................................................... 453 1 453 40 18,120
312.62(b), Investigator recordkeeping of case histories of
individuals ....................................................................... 453 1 453 40 18,120
312.160(a)(3), Records pertaining to the shipment of
drugs for investigational use in laboratory research ani-
mals or in vitro tests ....................................................... 111 1.40 155 * 0.50 78
312.160(c), Shipper records of alternative disposition of
unused drugs .................................................................. 111 1.40 155 * 0.50 78

Total ............................................................................ ........................ .......................... ........................ ........................ 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. DEPARTMENT OF HEALTH AND workshop to discuss dystrophin protein
Leslie Kux, HUMAN SERVICES quantification methodologies for human
Associate Commissioner for Policy. tissue. This workshop is being
Food and Drug Administration cosponsored by the National Institutes
[FR Doc. 2015–04379 Filed 3–2–15; 8:45 am]
BILLING CODE 4164–01–P [Docket No. FDA–2015–N–0430] of Health (NIH). The purpose of the
workshop is to discuss currently
Measuring Dystrophin in available methodologies and to identify
Dystrophinopathy Patients and scientific knowledge gaps and
Interpreting the Data; Public Scientific opportunities for improving dystrophin
Workshop; Request for Comments protein detection in the context of drug
mstockstill on DSK4VPTVN1PROD with NOTICES

AGENCY: Food and Drug Administration, development. The intended audiences
HHS. for this workshop are scientists and
clinicians involved in the acquisition,
ACTION:Notice of public scientific
measurement, and analysis of proteins
workshop; request for comments.
associated with Duchenne Muscular
The Food and Drug Administration Dystrophy (DMD).
(FDA) is announcing a public scientific

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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

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Document Created: 2015-12-18 11:56:04
Document Modified: 2015-12-18 11:56:04

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 scientific workshop; request for comments.
Dates		Dates and Time: The scientific workshop will be held on March 20, 2015, from 8:30 a.m. to 5:30 p.m.
FR Citation		80 FR 11454

80 FR 11454 - Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data; Public Scientific Workshop; Request for Comments

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

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

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration