2011-19483. 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 ...

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

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 The guidance announced in this notice finalizes the draft guidance of the same title dated October 2009.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 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.