83 FR 32309 - Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 134 (July 12, 2018)

Page Range		32309-32310
FR Document		2018-14868

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ``Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry.'' The draft guidance document provides sponsors of retroviral vector-based human gene therapy products recommendations regarding the testing for replication competent retrovirus (RCR) during the manufacture of retroviral vector-based products, and during follow- up monitoring of patients who have received retroviral vector-based products. Recommendations include the identification and amount of material to be tested, and general testing methods. In addition, recommendations are provided on monitoring patients for evidence of retroviral infection after administration of retroviral vector-based gene therapy products. The draft guidance, when finalized, is intended to supersede the document entitled ``Guidance for Industry: Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and During Follow-up of Patients in Clinical Trials Using Retroviral Vectors,'' dated November 2006. The draft guidance, when finalized, is also intended to supplement the documents entitled ``Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry'' and ``Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Draft Guidance for Industry,'' when these draft guidance documents are finalized.

Federal Register, Volume 83 Issue 134 (Thursday, July 12, 2018)

[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)]
[Notices]
[Pages 32309-32310]
From the Federal Register Online [www.thefederalregister.org]
[FR Doc No: 2018-14868]

[[Page 32309]]

-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-1999-D-0081]

Testing of Retroviral Vector-Based Human Gene Therapy Products
for Replication Competent Retrovirus During Product Manufacture and
Patient Follow-up; Draft Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a draft document entitled ``Testing of Retroviral
Vector-Based Human Gene Therapy Products for Replication Competent
Retrovirus During Product Manufacture and Patient Follow-up; Draft
Guidance for Industry.'' The draft guidance document provides sponsors
of retroviral vector-based human gene therapy products recommendations
regarding the testing for replication competent retrovirus (RCR) during
the manufacture of retroviral vector-based products, and during follow-
up monitoring of patients who have received retroviral vector-based
products. Recommendations include the identification and amount of
material to be tested, and general testing methods. In addition,
recommendations are provided on monitoring patients for evidence of
retroviral infection after administration of retroviral vector-based
gene therapy products. The draft guidance, when finalized, is intended
to supersede the document entitled ``Guidance for Industry:
Supplemental Guidance on Testing for Replication Competent Retrovirus
in Retroviral Vector Based Gene Therapy Products and During Follow-up
of Patients in Clinical Trials Using Retroviral Vectors,'' dated
November 2006. The draft guidance, when finalized, is also intended to
supplement the documents entitled ``Long Term Follow-Up After
Administration of Human Gene Therapy Products; Draft Guidance for
Industry'' and ``Chemistry, Manufacturing, and Control Information for
Human Gene Therapy Investigational New Drug Applications; Draft
Guidance for Industry,'' when these draft guidance documents are
finalized.

DATES: Submit either electronic or written comments on the draft
guidance by October 10, 2018 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as
follows:

Electronic Submissions

Submit electronic comments in the following way:
Federal eRulemaking Portal: https://www.regulations.gov.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to https://www.regulations.gov
will be posted to the docket unchanged. Because your comment will be
made public, you are solely responsible for ensuring that your comment
does not include any confidential information that you or a third party
may not wish to be posted, such as medical information, your or anyone
else's Social Security number, or confidential business information,
such as a manufacturing process. Please note that if you include your
name, contact information, or other information that identifies you in
the body of your comments, that information will be posted on https://www.regulations.gov.
If you want to submit a comment with confidential
information that you do not wish to be made available to the public,
submit the comment as a written/paper submission and in the manner
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

Submit written/paper submissions as follows:
Mail/Hand Delivery/Courier (for Written/Paper
Submissions): Dockets Management Staff (HFA-305), Food and Drug
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
For written/paper comments submitted to the Dockets
Management Staff, FDA will post your comment, as well as any
attachments, except for information submitted, marked and identified,
as confidential, if submitted as detailed in ``Instructions.''
Instructions: All submissions received must include the Docket No.
FDA-1999-D-0081 for ``Testing of Retroviral Vector-Based Human Gene
Therapy Products for Replication Competent Retrovirus During Product
Manufacture and Patient Follow-up; Draft Guidance for Industry.''
Received comments will be placed in the docket and, except for those
submitted as ``Confidential Submissions,'' publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m.
and 4 p.m., Monday through Friday.
Confidential Submissions--To submit a comment with
confidential information that you do not wish to be made publicly
available, submit your comments only as a written/paper submission. You
should submit two copies total. One copy will include the information
you claim to be confidential with a heading or cover note that states
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will
review this copy, including the claimed confidential information, in
its consideration of comments. The second copy, which will have the
claimed confidential information redacted/blacked out, will be
available for public viewing and posted on https://www.regulations.gov.
Submit both copies to the Dockets Management Staff. If you do not wish
your name and contact information to be made publicly available, you
can provide this information on the cover sheet and not in the body of
your comments and you must identify this information as
``confidential.'' Any information marked as ``confidential'' will not
be disclosed except in accordance with 21 CFR 10.20 and other
applicable disclosure law. For more information about FDA's posting of
comments to public dockets, see 80 FR 56469, September 18, 2015, or
access the information at: https://www.thefederalregister.org/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in
the heading of this document, into the ``Search'' box and follow the
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852.
You may submit comments on any guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single copies of the draft guidance to
the Office of Communication, Outreach and Development, Center for
Biologics Evaluation and Research (CBER), Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-
0002. 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 240-402-8010. See the
SUPPLEMENTARY INFORMATION section for electronic access to the draft
guidance document.

FOR FURTHER INFORMATION CONTACT: Melissa Segal, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7301,

[[Page 32310]]

Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a draft document entitled
``Testing of Retroviral Vector-Based Human Gene Therapy Products for
Replication Competent Retrovirus During Product Manufacture and Patient
Follow-up; Draft Guidance for Industry.'' The draft guidance document
provides sponsors of retroviral vector-based human gene therapy
products recommendations regarding the testing for RCR during the
manufacture of retroviral vector-based products, and during follow-up
monitoring of patients who have received retroviral vector-based
products. Recommendations are also provided for RCR testing during
manufacture, including identification and amount of material to be
tested, and general testing methods. In addition, recommendations are
provided on monitoring patients for evidence of retroviral infection
after administration of retroviral vector-based gene therapy products.
The draft guidance, when finalized, is intended to supersede the
document entitled ``Guidance for Industry: Supplemental Guidance on
Testing for Replication Competent Retrovirus in Retroviral Vector Based
Gene Therapy Products and During Follow-up of Patients in Clinical
Trials Using Retroviral Vectors,'' dated November 2006. The draft
guidance, when finalized, is also intended to supplement the ``Long
Term Follow-Up After Administration of Human Gene Therapy Products;
Draft Guidance for Industry'' and ``Chemistry, Manufacturing, and
Control Information for Human Gene Therapy Investigational New Drug
Applications; Draft Guidance for Industry,'' when these draft guidance
documents are finalized. Elsewhere in this issue of the Federal
Register, FDA is announcing the availability of these other two draft
guidance documents.
This 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 testing of
retroviral vector-based human gene therapy products for replication
competent retrovirus during product manufacture and patient follow-up.
It does not establish any rights for any person and is not binding on
FDA or the public. You can use an alternative approach if it satisfies
the requirements of the applicable statutes and regulations. This
guidance is not subject to Executive Order 12866.

II. Paperwork Reduction Act of 1995

The 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; and the collections of information in 21
CFR part 601 have been approved under OMB control number 0910-0338.

III. Electronic Access

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

Dated: July 5, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-14868 Filed 7-11-18; 8:45 am]
BILLING CODE 4164-01-P

32310 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices

Silver Spring, MD 20993–0002, 240– regulations. This guidance is not subject Scientific Advisors and Consultants,
402–7911. to Executive Order 12866. Center for Biologics Evaluation and
Research, Food and Drug
SUPPLEMENTARY INFORMATION: II. Paperwork Reduction Act of 1995
Administration, 10903 New Hampshire
I. Background The draft guidance refers to Ave., Bldg. 71, Rm. 6338, Silver Spring,
previously approved collections of MD 20993–0002; 240–402–5771,
FDA is announcing the availability of information found in FDA regulations. serina.hunter-thomas@fda.hhs.gov.
a draft document entitled ‘‘Testing of These collections of information are SUPPLEMENTARY INFORMATION: Pursuant
Retroviral Vector-Based Human Gene subject to review by the Office of to 41 CFR 102–3.65 and approval by the
Therapy Products for Replication Management and Budget (OMB) under Department of Health and Human
Competent Retrovirus During Product the Paperwork Reduction Act of 1995 Services pursuant to 45 CFR part 11 and
Manufacture and Patient Follow-up; (44 U.S.C. 3501–3520). The collections by the General Services Administration,
Draft Guidance for Industry.’’ The draft of information in 21 CFR part 312 have FDA is announcing the renewal of the
guidance document provides sponsors been approved under OMB control Allergenic Products Advisory
of retroviral vector-based human gene number 0910–0014; and the collections Committee. The committee is a
therapy products recommendations of information in 21 CFR part 601 have discretionary Federal advisory
regarding the testing for RCR during the been approved under OMB control committee established to provide advice
manufacture of retroviral vector-based number 0910–0338. to the Commissioner. The committee
products, and during follow-up advises the Commissioner or designee
III. Electronic Access
monitoring of patients who have in discharging responsibilities as they
received retroviral vector-based Persons with access to the internet relate to helping to ensure safe and
products. Recommendations are also may obtain the draft guidance at either effective drugs for human use and, as
provided for RCR testing during https://www.fda.gov/BiologicsBlood required, any other product for which
manufacture, including identification Vaccines/GuidanceCompliance FDA has regulatory responsibility.
and amount of material to be tested, and RegulatoryInformation/Guidances/ The Committee reviews and evaluates
general testing methods. In addition, default.htm or https:// available data concerning the safety,
recommendations are provided on www.regulations.gov. effectiveness, and adequacy of labeling
monitoring patients for evidence of Dated: July 5, 2018. of marketed and investigational
retroviral infection after administration Leslie Kux, allergenic biological products or
of retroviral vector-based gene therapy Associate Commissioner for Policy. materials that are administered to
products. The draft guidance, when humans for the diagnosis, prevention, or
[FR Doc. 2018–14868 Filed 7–11–18; 8:45 am]
finalized, is intended to supersede the treatment of allergies and allergic
BILLING CODE 4164–01–P
document entitled ‘‘Guidance for disease, and makes appropriate
Industry: Supplemental Guidance on recommendations to the Commissioner
Testing for Replication Competent DEPARTMENT OF HEALTH AND of its findings regarding the affirmation
Retrovirus in Retroviral Vector Based HUMAN SERVICES or revocation of biological product
Gene Therapy Products and During licenses; on the safety, effectiveness,
Follow-up of Patients in Clinical Trials Food and Drug Administration and labeling of the products; on clinical
Using Retroviral Vectors,’’ dated and laboratory studies of such products;
November 2006. The draft guidance, [Docket No. FDA–2018–N–2475]
on amendments or revisions to
when finalized, is also intended to regulations governing the manufacture,
Advisory Committee; Allergenic
supplement the ‘‘Long Term Follow-Up testing, and licensing of allergenic
Products Advisory Committee;
After Administration of Human Gene biological products; and on the quality
Renewal
Therapy Products; Draft Guidance for and relevance of FDA’s research
Industry’’ and ‘‘Chemistry, AGENCY: Food and Drug Administration, programs that provide the scientific
Manufacturing, and Control Information HHS. support for regulating these agents.
for Human Gene Therapy ACTION:Notice; renewal of advisory The Committee shall consist of a core
Investigational New Drug Applications; committee. of nine voting members including the
Draft Guidance for Industry,’’ when Chair. Members and the Chair are
these draft guidance documents are SUMMARY: The Food and Drug selected by the Commissioner or
finalized. Elsewhere in this issue of the Administration (FDA) is announcing the designee from among authorities
Federal Register, FDA is announcing renewal of the Allergenic Products knowledgeable in the fields of allergy,
the availability of these other two draft Advisory Committee by the immunology, pediatrics, internal
guidance documents. Commissioner of Food and Drugs (the medicine, biochemistry, and related
This draft guidance is being issued Commissioner). The Commissioner has specialties. Members will be invited to
consistent with FDA’s good guidance determined that it is in the public serve for overlapping terms of up to 4
practices regulation (21 CFR 10.115). interest to renew the Allergenic years. Almost all non-Federal members
The draft guidance, when finalized, will Products Advisory Committee for an of this committee serve as Special
represent FDA’s current thinking on additional 2 years beyond the charter Government Employees. The core of
testing of retroviral vector-based human expiration date. The new charter will be voting members may include one
gene therapy products for replication in effect until July 9, 2020. technically qualified member, selected
amozie on DSK3GDR082PROD with NOTICES1

competent retrovirus during product DATES: Authority for the Allergenic by the Commissioner or designee, who
manufacture and patient follow-up. It Products Advisory Committee expired is identified with consumer interests
does not establish any rights for any on July 9, 2018; however, the and is recommended by either a
person and is not binding on FDA or the Commissioner formally determined that consortium of consumer-oriented
public. You can use an alternative renewal is in the public interest. organizations or other interested
approach if it satisfies the requirements FOR FURTHER INFORMATION CONTACT: persons. In addition to the voting
of the applicable statutes and Serina Hunter-Thomas, Division of members, the Committee may include

VerDate Sep<11>2014 17:21 Jul 11, 2018 Jkt 244001 PO 00000 Frm 00051 Fmt 4703 Sfmt 4703 E:\FR\FM\12JYN1.SGM 12JYN1

Document Created: 2018-11-06 10:22:13
Document Modified: 2018-11-06 10:22:13

Category		Regulatory Information
Collection		Federal Register
sudoc Class		AE 2.7: GS 4.107: AE 2.106:
Publisher		Office of the Federal Register, National Archives and Records Administration
Section		Notices
Action		Notice of availability.
Dates		Submit either electronic or written comments on the draft guidance by October 10, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.
Contact		Melissa Segal, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911.
FR Citation		83 FR 32309

83 FR 32309 - Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 83, Issue 134 (July 12, 2018)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration