Document ID: FDA-2009-D-0490-0012
Agency: fda
Document Type: Notice
Title: Draft Guidance for Industry and FDA Staff:  Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System; Availability
Posted Date: 2013-06-17T04:00Z

[Federal Register Volume 78, Number 116 (Monday, June 17, 2013)]
[Notices]
[Pages 36194-36196]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-14096]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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

Draft Guidance for Industry and FDA Staff: Investigational New 
Drug Applications for Minimally Manipulated, Unrelated Allogeneic 
Placental/Umbilical Cord Blood Intended for Hematopoietic and 
Immunologic Reconstitution in Patients with Disorders Affecting the 
Hematopoietic System; 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 draft document entitled ``Guidance for Industry and 
FDA Staff:

[[Page 36195]]

Investigational New Drug Applications for Minimally Manipulated, 
Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for 
Hematopoietic and Immunologic Reconstitution in Patients with Disorders 
Affecting the Hematopoietic System'' dated June 2013. The draft 
guidance document provides advice to potential sponsors, such as cord 
blood banks, registries, transplant centers, or individual physicians 
serving as sponsor-investigators, to assist in the submission of an 
Investigational New Drug Application (IND) for certain hematopoietic 
progenitor cells from placental/umbilical cord blood (HPC, Cord Blood), 
when such HPC, Cord Blood units are not licensed, and when a suitable 
human leukocyte antigen matched cord blood transplant is needed for 
hematopoietic and immunologic reconstitution in patients with disorders 
affecting the hematopoietic system that are inherited, acquired, or 
result from myeloablative treatment and there is no satisfactory 
alternative treatment available. If unlicensed HPC, Cord Blood units 
are made available for clinical use, they must be distributed under an 
IND. The draft guidance, when finalized, is intended to supersede the 
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.

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 either electronic or written comments on the draft 
guidance by September 16, 2013.

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 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: Valerie A. Butler, 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 for Minimally Manipulated, Unrelated Allogeneic Placental/
Umbilical Cord Blood Intended for Hematopoietic and Immunologic 
Reconstitution in Patients with Disorders Affecting the Hematopoietic 
System'' dated June 2013. The draft guidance, when finalized, will 
provide advice to potential sponsors to assist in the submission of an 
IND for certain HPC, Cord Blood, when such HPC, Cord Blood units are 
not licensed in accordance with Title 21 Code of Federal Regulations 
Part 601 (21 CFR part 601), and when a suitable human leukocyte antigen 
matched cord blood transplant is needed for hematopoietic and 
immunologic reconstitution in patients with disorders affecting the 
hematopoietic system that are inherited, acquired, or result from 
myeloablative treatment and there is no satisfactory alternative 
treatment available. If unlicensed HPC, Cord Blood units are made 
available for clinical use, they must be distributed under an IND 
meeting the applicable requirements in 21 CFR part 312. The draft 
guidance, when finalized, is intended to supersede the 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 draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent 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 requirement of the applicable statutes 
and regulations.
    Elsewhere in this issue of the Federal Register, we also are 
announcing the availability of another, related draft guidance entitled 
``Guidance for Industry: Biologics License Applications for Minimally 
Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood 
Intended for Hematopoietic and Immunologic Reconstitution in Patients 
with Disorders Affecting the Hematopoietic System.'' That draft 
guidance, when finalized, is intended to supersede the document 
entitled ``Guidance for Industry: Minimally Manipulated, Unrelated 
Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic 
Reconstitution for Specified Indications'' dated October 2009.

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 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 Form FDA 1571 has been approved under 
OMB control number 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 submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets 
Management (see ADDRESSES). It is only necessary to send one set of 
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, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the draft guidance 
at either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

[[Page 36196]]

    Dated: June 10, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-14096 Filed 6-14-13; 8:45 am]
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