Document ID: FDA-2009-D-0490-0010
Agency: fda
Document Type: Notice
Title: Guidance for Industry and Food and Drug Administration Staff; Availability: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental-Umbilical Cord Blood, etc.
Posted Date: 2011-08-02T04:00Z

[Federal Register Volume 76, Number 148 (Tuesday, August 2, 2011)]
[Notices]
[Pages 46303-46304]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2011-19483]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2009-D-0490]

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.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a 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 June 2011. The guidance document provides advice to potential 
sponsors to assist in the submission of an IND for certain minimally 
manipulated hematopoietic stem/progenitor cells from placental/
umbilical cord blood, from an unrelated allogeneic cord blood donor and 
intended for hematopoietic reconstitution in patients with specified 
indications (HPC-Cs), when such HPC-Cs are not licensed 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. If such HPC-Cs are made available for clinical use, they 
must be distributed under an IND. The guidance announced in this notice 
finalizes the draft guidance of the same title dated October 2009.

DATES: Submit either electronic or written comments on Agency guidances 
at any time.

ADDRESSES: Submit written requests for single copies of the 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 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 guidance document.
    Submit electronic comments on the guidance to http://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 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 June 2011. The 
guidance document provides advice to potential sponsors (e.g., cord 
blood banks or registries, transplant centers, 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. The guidance document is applicable 
only to HPC-Cs intended for hematopoietic reconstitution in patients 
with the clinical indications listed in the guidance. If such HPC-Cs 
are made available for clinical use, they must be distributed under an 
IND meeting all of the applicable requirements in part 312 (21 CFR Part 
312).
    In the Federal Register of October 20, 2009 (74 FR 53751), FDA 
announced the availability of the draft guidance of the same title 
dated October 2009. FDA received a few comments on the draft guidance, 
and those comments were considered as the guidance was finalized. 
Changes incorporated in the final guidance include simplifying table A, 
which sets forth certain regulatory requirements and current best 
practices with respect to what should be included in an IND. In 
addition, organizational and editorial revisions were made to improve 
clarity. The guidance announced in this notice finalizes the draft 
guidance dated October 2009.
    In the October 20, 2009, notice announcing the availability of the 
draft

[[Page 46304]]

guidance, FDA also announced that it no longer intends to exercise 
enforcement discretion with respect to the IND and biologics license 
application (BLA) requirements, and the phase-in implementation period 
for IND and license application requirements will end as of October 20, 
2011. FDA also encouraged 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. FDA 
continues to encourage potential sponsors to submit new protocols as 
needed to their existing INDs, or new INDs if needed, or BLAs as soon 
as possible, so that FDA may work with them to ensure that the 
protocols are in effect or that the BLAs are approved, if appropriate, 
by the end of the phase-in implementation period.
    We acknowledge that there will be cord blood banks that are not 
able to achieve licensure by October 20, 2011. Furthermore, we 
acknowledge that should we approve a bank's BLA, our approval may not 
include all the HPC-Cs in that bank's inventory. We note that if a bank 
is unable to obtain a BLA by October 20, 2011, or if its BLA does not 
include all the HPC-Cs in that bank's inventory, its unlicensed units 
may be released for use only under an IND.
    The guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents FDA's 
current thinking on this topic. It does not create or confer any rights 
for or on any person and does not operate to bind FDA or the public. An 
alternative approach may be used if such approach satisfies the 
requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    This 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 part 312 have been approved under OMB 
control number 0910-0014; 21 CFR Part 56 have been approved under OMB 
control number 0910-0130; 21 CFR Part 1271 have been approved under OMB 
control number 0910-0543; and FDA Form 1571 has been approved under OMB 
control number 0910-0014.

III. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) either electronic or written comments regarding this 
document. It is only necessary to send one set of comments. It is no 
longer necessary to send two copies of mailed comments. Identify 
comments with the docket number found in brackets in the heading of 
this document. Received comments may be seen 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 guidance at 
either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: July 26, 2011.
David Dorsey,
Acting Deputy Commissioner for Policy, Planning and Budget.
[FR Doc. 2011-19483 Filed 8-1-11; 8:45 am]
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