Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data; Public Scientific Workshop; Request for Comments, 11454-11455 [2015-04384]
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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
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19:24 Mar 02, 2015
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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
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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]
-----------------------------------------------------------------------
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