Draft Guidance for Industry and Food and Drug Administration Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications; Availability, 53751-53752 [E9-25136]
Download as PDF
<![CDATA[
Federal Register / Vol. 74, No. 201 / Tuesday, October 20, 2009 / Notices
guidance document describes
recommendations for device
manufacturers submitting to FDA an
application for determination that a
cleared or approved device meets this
CLIA standard (CLIA waiver
application).
The guidance recommends that CLIA
waiver applications include a
description of the features of the device
that make it ‘‘simple’’; a report
describing a hazard analysis that
identifies potential sources of error,
including a summary of the design and
results of flex studies and conclusions
drawn from the flex studies; a
description of fail-safe and failure alert
mechanisms and a description of the
studies validating these mechanisms; a
description of clinical tests that
demonstrate the accuracy of the test in
the hands of intended operators; and
53751
statistical analyses of clinical study
results. Only new information
collections not already approved are
included in the estimate in the
following table. Quick reference
instructions are a short version of the
instructions that are written in simple
language and that can be posted.
FDA estimates the burden of this
collection as follows:
TABLE 1.—ESTIMATED ANNUAL REPORTING BURDEN1
21 CFR Section
No. of
Respondents
493.15(a) and (b)
1 There
Annual Frequency
of Response
40
Total Annual
Responses
1
Hours per
Response
40
Total Hours
780
31,200
Operating and
Maintenance Costs
$50,200
are no capital costs associated with this collection of information.
TABLE 2.—ESTIMATED ANNUAL RECORDKEEPING BURDEN1
21 CFR Section
No. of
Recordkeepers
493.15(a) and (b)
cprice-sewell on DSKGBLS3C1PROD with NOTICES
1 There
Annual Frequency
per Recordkeeping
40
Total Annual
Records
1
Hours per
Record
40
2,800
Total Hours
112,000
Operating and
Maintenance Costs
$16,000
are no capital costs associated with this collection of information.
The total number of reporting and
recordkeeping hours is 143,200 hours.
FDA bases the burden on an agency
analysis of premarket submissions with
clinical trials similar to the waived
laboratory tests. Based on previous
years’ experience with CLIA waiver
applications, FDA expects 40
manufacturers to submit one CLIA
waiver application per year. The time
required to prepare and submit a waiver
application, including the time needed
to assemble supporting data, averages
780 hours per waiver application for a
total of 31,200 hours for reporting.
Based on previous years experience
with CLIA waiver applications, FDA
expects that each manufacturer will
spend 2,800 hours creating and
maintaining the record for a total of
112,000 hours.
The total operating and maintenance
cost associated with the waiver
application is estimated at $66,200. The
cost consists of specimen collection for
the clinical study (estimated $23,500);
laboratory supplies, reference testing
and study oversight (estimated $26,700);
shipping and office supplies (estimated
$6,000); and educational materials,
including quick reference instructions
(estimated $10,000).
This guidance also refers to
previously approved collections of
information found in FDA regulations.
The collections of information in 21
CFR part 801 and § 809.10 have been
approved under OMB control number
0910–0485 and the collections of
information in 21 CFR part 803 have
VerDate Nov<24>2008
14:46 Oct 19, 2009
Jkt 220001
been approved under OMB control
number 0910–0437.
Dated: October 9, 2009.
David Horowitz,
Assistant Commissioner for Policy.
[FR Doc. E9–25177 Filed 10–19–09; 8:45 am]
BILLING CODE 4160–01–S
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2009–D–0490]
Draft Guidance for Industry and Food
and Drug Administration Staff:
Investigational New Drug Applications
for Minimally Manipulated, Unrelated
Allogeneic Placental/Umbilical Cord
Blood Intended for Hematopoietic
Reconstitution for Specified
Indications; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
SUMMARY: The Food and Drug
Administration (FDA) is announcing the
availability of a draft document entitled
‘‘Guidance for Industry and FDA Staff:
Investigational New Drug Applications
(INDs) for Minimally Manipulated,
Unrelated Allogeneic Placental/
Umbilical Cord Blood Intended for
Hematopoietic Reconstitution for
Specified Indications,’’ dated October
2009. In this draft guidance, we refer to
these products for hematopoietic
PO 00000
Frm 00055
Fmt 4703
Sfmt 4703
reconstitution for specified indications
as hematopoietic progenitor cells, cord
(HPC–C). This draft guidance provides
advice to potential sponsors (e.g.,
generally cord blood banks, or registries,
and individual physicians serving as
sponsor-investigators) to assist in the
submission of an IND for certain HPC–
Cs, when such HPC–Cs are not licensed
in accordance with certain FDA
regulations, and when a suitable human
leukocyte antigen (HLA) matched cord
blood transplant is needed for treatment
of a patient with a serious or lifethreatening disease or condition and
there is no satisfactory alternative
treatment. This draft guidance
document is applicable only to HPC–Cs
intended for hematopoietic
reconstitution in patients with the
clinical indications listed in the
guidance entitled ‘‘Guidance for
Industry: Minimally Manipulated,
Unrelated Allogeneic Placental/
Umbilical Cord Blood Intended for
Hematopoietic Reconstitution for
Specified Indications’’ (HPC–C
licensure guidance), published
elsewhere in this issue of the Federal
Register. FDA is also announcing that it
no longer intends to exercise
enforcement discretion with respect to
the IND and biologics license
application (BLA) requirements for
minimally manipulated, unrelated
allogeneic hematopoietic stem/
progenitor cell products and the phasein implementation period for IND and
license application requirements will
end as of October 20, 2011.
E:\FR\FM\20OCN1.SGM
20OCN1
53752
Federal Register / Vol. 74, No. 201 / Tuesday, October 20, 2009 / Notices
cprice-sewell on DSKGBLS3C1PROD with NOTICES
DATES: Although you can comment on
any guidance at any time (see 21 CFR
10.115(g)(5)), to ensure that the agency
considers your comment on this draft
guidance before it begins work on the
final version of the guidance, submit
electronic or written comments on the
draft guidance by January 19, 2010.
ADDRESSES: Submit written requests for
single copies of the draft guidance to the
Office of Communication, Outreach and
Development (HFM–40), Center for
Biologics Evaluation and Research
(CBER), Food and Drug Administration,
1401 Rockville Pike, suite 200N,
Rockville, MD 20852–1448. Send one
self-addressed adhesive label to assist
the office in processing your requests.
The draft guidance may also be obtained
by mail by calling CBER at 1–800–835–
4709 or 301–827–1800. See the
SUPPLEMENTARY INFORMATION section for
electronic access to the draft guidance
document.
Submit electronic comments on the
draft guidance to https://
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.
FOR FURTHER INFORMATION CONTACT:
Tami Belouin, Center for Biologics
Evaluation and Research (HFM–17),
Food and Drug Administration, 1401
Rockville Pike, suite 200N, Rockville,
MD 20852–1448, 301–827–6210.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a draft document entitled ‘‘Guidance for
Industry and FDA Staff: Investigational
New Drug Applications (INDs) for
Minimally Manipulated, Unrelated
Allogeneic Placental/Umbilical Cord
Blood Intended for Hematopoietic
Reconstitution for Specified
Indications,’’ dated October 2009. This
draft guidance provides advice to
potential sponsors (e.g., generally cord
blood banks, or registries, and
individual physicians serving as
sponsor-investigators), to assist in the
submission of an IND for certain HPC–
Cs, when such HPC–Cs are not licensed
in accordance with 21 CFR part 601,
and when a suitable HLA matched cord
blood transplant is needed for treatment
of a patient with a serious or lifethreatening disease or condition and
there is no satisfactory alternative
treatment. This draft guidance
document is applicable only to HPC–Cs
intended for hematopoietic
reconstitution in patients with the
clinical indications as listed in the
guidance entitled ‘‘Guidance for
VerDate Nov<24>2008
14:46 Oct 19, 2009
Jkt 220001
Industry: Minimally Manipulated,
Unrelated Allogeneic Placental/
Umbilical Cord Blood Intended for
Hematopoietic Reconstitution in
Patients with Specified Indications’’
(HPC–C licensure guidance), published
elsewhere in this issue of the Federal
Register.
FDA is also announcing that it no
longer intends to exercise enforcement
discretion with respect to IND and BLA
requirements for minimally
manipulated unrelated allogeneic
hematopoietic stem/progenitor cell
products and the phase-in
implementation period for IND and
license application requirements for
these products will end (see the
SUMMARY for the ending date). We
encourage sponsors to send in
applications as soon as possible to allow
sufficient time for review, comment,
and resubmission as needed to complete
all actions by the end of this 2-year
period.
In the Federal Register notice of
January 20, 1998 (63 FR 2985), FDA
requested submission of comments
proposing establishment controls,
process controls, and product standards
designed to ensure the safety and
effectiveness of minimally manipulated
unrelated allogeneic hematopoietic
stem/progenitor cell products derived
from peripheral and cord blood for
hematopoietic reconstitution. Also, in
the January 20, 1998, notice, FDA
announced its intention to phase in
implementation of IND and license
application requirements for minimally
manipulated unrelated allogeneic
hematopoietic stem/progenitor cell
products to permit the development of
licensing standards for those products
where possible.
In the Federal Register notice of
January 17, 2007 (72 FR 1999), FDA
announced the availability of the draft
guidance entitled ‘‘Guidance for
Industry: Minimally Manipulated,
Unrelated, Allogeneic Placental/
Umbilical Cord Blood Intended for
Hematopoietic Reconstitution in
Patients with Hematological
Malignancies,’’ dated December 2006.
FDA received comments on the
December 2006 draft guidance and those
comments were considered as the
guidance was finalized. The HPC–C
licensure guidance finalizes the
December 2006 draft guidance. Some of
the comments received by FDA
expressed the importance of access and
availability of HPC–C products that do
not meet the standards for licensure and
therefore, cannot be licensed. FDA
recognizes the importance of providing
guidance for such products and is
publishing this IND draft guidance for
PO 00000
Frm 00056
Fmt 4703
Sfmt 4703
comment. The HPC–C licensure
guidance document is effective on its
date of publication.
This draft guidance is consistent with
FDA’s good guidance practices
regulation (21 CFR 10.115). The draft
guidance when finalized will represent
FDA’s current thinking on these topics.
It does not create or confer any rights for
or on any person and does not operate
to bind FDA or the public. Alternative
approaches may be used if such
approaches satisfy the requirements of
the applicable statutes and regulations.
II. Paperwork Reduction Act of 1995
This draft guidance refers to
previously approved collections of
information found in FDA regulations.
These collections of information are
subject to review by the Office of
Management and Budget (OMB) under
the Paperwork Reduction Act of 1995
(44 U.S.C. 3501–3520). The collections
of information in 21 CFR part 312 have
been approved under OMB Control No.
0910–0014; 21 CFR part 1271 have been
approved under OMB Control Nos.
0910–0559, 0910–0469, and 0910–0543;
and FDA Form 1571 has been approved
under OMB Control No. 0910–0014.
III. Comments
The draft guidance is being
distributed for comment purposes only
and is not intended for implementation
at this time. Interested persons may still,
at any time, submit to the Division of
Dockets Management (see ADDRESSES)
electronic or written comments
regarding the draft guidance. Submit a
single copy of electronic comments or
two paper copies of any mailed
comments, except that individuals may
submit one paper copy. Comments are
to be identified with the docket number
found in brackets in the heading of this
document. A copy of the draft guidance
and received comments are available for
public examination in the Division of
Dockets Management between 9 a.m.
and 4 p.m., Monday through Friday.
IV. Electronic Access
Persons with access to the Internet
may obtain the draft guidance at either
https://www.fda.gov/BiologicsBlood
Vaccines/GuidanceCompliance
RegulatoryInformation/Guidances/
default.htm.
Dated: October 14, 2009.
David Horowitz,
Assistant Commissioner for Policy.
[FR Doc. E9–25136 Filed 10–19–09; 8:45 am]
BILLING CODE 4160–01–S
E:\FR\FM\20OCN1.SGM
20OCN1
]]>Agencies
[Federal Register Volume 74, Number 201 (Tuesday, October 20, 2009)]
[Notices]
[Pages 53751-53752]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: E9-25136]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2009-D-0490]
Draft Guidance for Industry and Food and Drug Administration
Staff: Investigational New Drug Applications for Minimally Manipulated,
Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for
Hematopoietic Reconstitution for Specified Indications; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing the
availability of a draft document entitled ``Guidance for Industry and
FDA Staff: Investigational New Drug Applications (INDs) for Minimally
Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood
Intended for Hematopoietic Reconstitution for Specified Indications,''
dated October 2009. In this draft guidance, we refer to these products
for hematopoietic reconstitution for specified indications as
hematopoietic progenitor cells, cord (HPC-C). This draft guidance
provides advice to potential sponsors (e.g., generally cord blood
banks, or registries, and individual physicians serving as sponsor-
investigators) to assist in the submission of an IND for certain HPC-
Cs, when such HPC-Cs are not licensed in accordance with certain FDA
regulations, and when a suitable human leukocyte antigen (HLA) matched
cord blood transplant is needed for treatment of a patient with a
serious or life-threatening disease or condition and there is no
satisfactory alternative treatment. This draft guidance document is
applicable only to HPC-Cs intended for hematopoietic reconstitution in
patients with the clinical indications listed in the guidance entitled
``Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic
Placental/Umbilical Cord Blood Intended for Hematopoietic
Reconstitution for Specified Indications'' (HPC-C licensure guidance),
published elsewhere in this issue of the Federal Register. FDA is also
announcing that it no longer intends to exercise enforcement discretion
with respect to the IND and biologics license application (BLA)
requirements for minimally manipulated, unrelated allogeneic
hematopoietic stem/progenitor cell products and the phase-in
implementation period for IND and license application requirements will
end as of October 20, 2011.
[[Page 53752]]
DATES: Although you can comment on any guidance at any time (see 21
CFR 10.115(g)(5)), to ensure that the agency considers your comment on
this draft guidance before it begins work on the final version of the
guidance, submit electronic or written comments on the draft guidance
by January 19, 2010.
ADDRESSES: Submit written requests for single copies of the draft
guidance to the Office of Communication, Outreach and Development (HFM-
40), Center for Biologics Evaluation and Research (CBER), Food and Drug
Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-
1448. Send one self-addressed adhesive label to assist the office in
processing your requests. The draft guidance may also be obtained by
mail by calling CBER at 1-800-835-4709 or 301-827-1800. See the
SUPPLEMENTARY INFORMATION section for electronic access to the draft
guidance document.
Submit electronic comments on the draft guidance to https://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.
FOR FURTHER INFORMATION CONTACT: Tami Belouin, Center for Biologics
Evaluation and Research (HFM-17), Food and Drug Administration, 1401
Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-6210.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft document entitled
``Guidance for Industry and FDA Staff: Investigational New Drug
Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic
Placental/Umbilical Cord Blood Intended for Hematopoietic
Reconstitution for Specified Indications,'' dated October 2009. This
draft guidance provides advice to potential sponsors (e.g., generally
cord blood banks, or registries, and individual physicians serving as
sponsor-investigators), to assist in the submission of an IND for
certain HPC-Cs, when such HPC-Cs are not licensed in accordance with 21
CFR part 601, and when a suitable HLA matched cord blood transplant is
needed for treatment of a patient with a serious or life-threatening
disease or condition and there is no satisfactory alternative
treatment. This draft guidance document is applicable only to HPC-Cs
intended for hematopoietic reconstitution in patients with the clinical
indications as listed in the guidance entitled ``Guidance for Industry:
Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord
Blood Intended for Hematopoietic Reconstitution in Patients with
Specified Indications'' (HPC-C licensure guidance), published elsewhere
in this issue of the Federal Register.
FDA is also announcing that it no longer intends to exercise
enforcement discretion with respect to IND and BLA requirements for
minimally manipulated unrelated allogeneic hematopoietic stem/
progenitor cell products and the phase-in implementation period for IND
and license application requirements for these products will end (see
the SUMMARY for the ending date). We encourage sponsors to send in
applications as soon as possible to allow sufficient time for review,
comment, and resubmission as needed to complete all actions by the end
of this 2-year period.
In the Federal Register notice of January 20, 1998 (63 FR 2985),
FDA requested submission of comments proposing establishment controls,
process controls, and product standards designed to ensure the safety
and effectiveness of minimally manipulated unrelated allogeneic
hematopoietic stem/progenitor cell products derived from peripheral and
cord blood for hematopoietic reconstitution. Also, in the January 20,
1998, notice, FDA announced its intention to phase in implementation of
IND and license application requirements for minimally manipulated
unrelated allogeneic hematopoietic stem/progenitor cell products to
permit the development of licensing standards for those products where
possible.
In the Federal Register notice of January 17, 2007 (72 FR 1999),
FDA announced the availability of the draft guidance entitled
``Guidance for Industry: Minimally Manipulated, Unrelated, Allogeneic
Placental/Umbilical Cord Blood Intended for Hematopoietic
Reconstitution in Patients with Hematological Malignancies,'' dated
December 2006. FDA received comments on the December 2006 draft
guidance and those comments were considered as the guidance was
finalized. The HPC-C licensure guidance finalizes the December 2006
draft guidance. Some of the comments received by FDA expressed the
importance of access and availability of HPC-C products that do not
meet the standards for licensure and therefore, cannot be licensed. FDA
recognizes the importance of providing guidance for such products and
is publishing this IND draft guidance for comment. The HPC-C licensure
guidance document is effective on its date of publication.
This draft guidance is consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The draft guidance when finalized
will represent FDA's current thinking on these topics. It does not
create or confer any rights for or on any person and does not operate
to bind FDA or the public. Alternative approaches may be used if such
approaches satisfy the requirements of the applicable statutes and
regulations.
II. Paperwork Reduction Act of 1995
This draft guidance refers to previously approved collections of
information found in FDA regulations. These collections of information
are subject to review by the Office of Management and Budget (OMB)
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The
collections of information in 21 CFR part 312 have been approved under
OMB Control No. 0910-0014; 21 CFR part 1271 have been approved under
OMB Control Nos. 0910-0559, 0910-0469, and 0910-0543; and FDA Form 1571
has been approved under OMB Control No. 0910-0014.
III. Comments
The draft guidance is being distributed for comment purposes only
and is not intended for implementation at this time. Interested persons
may still, at any time, submit to the Division of Dockets Management
(see ADDRESSES) electronic or written comments regarding the draft
guidance. Submit a single copy of electronic comments or two paper
copies of any mailed comments, except that individuals may submit one
paper copy. Comments are to be identified with the docket number found
in brackets in the heading of this document. A copy of the draft
guidance and received comments are available for public examination in
the Division of Dockets Management between 9 a.m. and 4 p.m., Monday
through Friday.
IV. Electronic Access
Persons with access to the Internet may obtain the draft guidance
at either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm.
Dated: October 14, 2009.
David Horowitz,
Assistant Commissioner for Policy.
[FR Doc. E9-25136 Filed 10-19-09; 8:45 am]
BILLING CODE 4160-01-S
