Document ID: FDA-2009-D-0490-0001
Agency: fda
Document Type: Notice
Title: Draft Guidance for Industry and Food and Drug Administration Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical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
Posted Date: 2009-10-20T04:00Z

[Federal Register: October 20, 2009 (Volume 74, Number 201)]
[Notices]               
[Page 53751-53752]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr20oc09-67]                         

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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Draft Guidance for Industry and Food and Drug Administration 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 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 In this draft guidance, we refer to these products 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 This draft guidance 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 This draft guidance document is 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 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 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 http://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]

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