81 FR 63766 - Recommendations for Microbial Vectors Used for Gene Therapy; Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 81, Issue 180 (September 16, 2016)

Page Range		63766-63767
FR Document		2016-22353

The Food and Drug Administration (FDA or Agency) is announcing the availability of a document entitled ``Recommendations for Microbial Vectors Used for Gene Therapy; Guidance for Industry.'' The guidance document provides investigational new drug application (IND) sponsors, with recommendations concerning IND submissions for microbial vectors used for gene therapy (MVGTs) in early phase clinical trials. The guidance focuses on the chemistry, manufacturing, and control (CMC) information that sponsors should submit in an IND for MVGTs and provides an overview of preclinical and clinical considerations for these products. The guidance announced in this notice finalizes the draft guidance of the same title dated October 2015 and supplements the guidance entitled ``Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs),'' dated April 2008.

Federal Register, Volume 81 Issue 180 (Friday, September 16, 2016)

[Federal Register Volume 81, Number 180 (Friday, September 16, 2016)]
[Notices]
[Pages 63766-63767]
From the Federal Register Online [www.thefederalregister.org]
[FR Doc No: 2016-22353]

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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-3399]

Recommendations for Microbial Vectors Used for Gene Therapy;
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 document entitled ``Recommendations for Microbial
Vectors Used for Gene Therapy; Guidance for Industry.'' The guidance
document provides investigational new drug application (IND) sponsors,
with recommendations concerning IND submissions for microbial vectors
used for gene therapy (MVGTs) in early phase clinical trials. The
guidance focuses on the chemistry, manufacturing, and control (CMC)
information that sponsors should submit in an IND for MVGTs and
provides an overview of preclinical and clinical considerations for
these products. The guidance announced in this notice finalizes the
draft guidance of the same title dated October 2015 and supplements the
guidance entitled ``Guidance for FDA Reviewers and Sponsors: Content
and Review of Chemistry, Manufacturing, and Control (CMC) Information
for Human Gene Therapy Investigational New Drug Applications (INDs),''
dated April 2008.

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

ADDRESSES: You may submit comments as follows:

Electronic Submissions

Submit electronic comments in the following way:
Federal eRulemaking Portal: http://www.regulations.gov.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to http://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 http://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): Division of Dockets Management (HFA-305), Food and Drug
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
For written/paper comments submitted to the Division of
Dockets Management, 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-2015-D-3399 for ``Recommendations for Microbial Vectors Used for
Gene Therapy; Guidance for Industry.'' Received comments will be placed
in the docket and, except for those submitted as ``Confidential
Submissions,'' publicly viewable at http://www.regulations.gov or at
the Division of Dockets Management 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 http://www.regulations.gov.
Submit both copies to the Division of Dockets Management. 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: http://www.fda.gov/regulatoryinformation/dockets/default.htm.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to http://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 Division of Dockets Management, 5630 Fishers
Lane, Rm. 1061, Rockville, MD 20852.
Submit written requests for single copies of the 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 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 guidance document.

FOR FURTHER INFORMATION CONTACT: Tami Belouin, 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.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a document entitled
``Recommendations for Microbial Vectors Used for Gene Therapy; Guidance
for Industry.'' The guidance provides IND sponsors, with
recommendations concerning IND submissions for microbial vectors used
for MVGTs in early phase clinical trials. The guidance focuses on the
CMC information that sponsors should submit in an IND for MVGTs and
provides an overview of preclinical and

[[Page 63767]]

clinical considerations for these products.
In the Federal Register of October 14, 2015 (80 FR 61822), FDA
announced the availability of the draft guidance of the same title
dated October 2015. FDA received one comment on the draft guidance and
that comment was considered as the guidance was finalized. Minor
editorial changes were made in response to the comment to improve
clarity. The guidance announced in this notice finalizes the draft
guidance dated October 2015 and supplements the guidance entitled
``Guidance for FDA Reviewers and Sponsors: Content and Review of
Chemistry, Manufacturing, and Control (CMC) Information for Human Gene
Therapy Investigational New Drug Applications (INDs),'' dated April
2008.
This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on recommendations for MVGTs. 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.

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 21 CFR parts 211 and 610 have been
approved under OMB control number 0910-0139 and in 21 CFR part 312
under OMB control number 0910-0014.

III. 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: September 12, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-22353 Filed 9-15-16; 8:45 am]
BILLING CODE 4164-01-P

Federal Register / Vol. 81, No. 180 / Friday, September 16, 2016 / Notices 63767

clinical considerations for these DEPARTMENT OF HEALTH AND ethical treatment of study subjects and
products. HUMAN SERVICES for acquiring adequate and reliable data
In the Federal Register of October 14, to support regulatory decisions. This
Food and Drug Administration course is intended to train clinical
2015 (80 FR 61822), FDA announced the
availability of the draft guidance of the [Docket No. FDA–2013–N–1214] investigators in all elements of clinical
trials, including the preclinical and
same title dated October 2015. FDA
Clinical Investigator Training Course clinical information needed to support
received one comment on the draft
the investigational use of medical
guidance and that comment was AGENCY: Food and Drug Administration, products; the statistical design of trials;
considered as the guidance was HHS. and scientific, regulatory, and ethical
finalized. Minor editorial changes were ACTION: Notice. considerations related to conduct of
made in response to the comment to clinical trials. The course lecturers will
improve clarity. The guidance SUMMARY: The Food and Drug include a diverse representation of
announced in this notice finalizes the Administration (FDA), in collaboration senior FDA staff and other experts,
draft guidance dated October 2015 and with the University of Maryland Center enabling communication on issues
supplements the guidance entitled of Excellence in Regulatory Science and critical for successful conduct of clinical
‘‘Guidance for FDA Reviewers and Innovation (M–CERSI), is announcing a research.
Sponsors: Content and Review of 3-day training course for clinical
investigators on the scientific, ethical, II. Description of the Training Course
Chemistry, Manufacturing, and Control
(CMC) Information for Human Gene and regulatory aspects of clinical trials A. Purpose
Therapy Investigational New Drug for medical products. This training
course is intended to provide clinical The training course is designed to
Applications (INDs),’’ dated April 2008. provide clinical investigators with an
investigators, such as clinicians, nurses,
This guidance is being issued pharmacists, and other health care overview of the following information:
consistent with FDA’s good guidance providers involved in conducting • The essential toxicological,
practices regulation (21 CFR 10.115). clinical trials, with expertise in the pharmacological, and manufacturing
The guidance represents the current design, conduct, and analysis of clinical data to support investigational use in
thinking of FDA on recommendations trials; to improve the quality of clinical humans;
for MVGTs. It does not establish any trials; and to enhance the safety of trial • Fundamental issues in the design
rights for any person and is not binding participants. Senior FDA staff, along and conduct of clinical trials;
• Statistical and analytic
on FDA or the public. You can use an with other experts, will present on
considerations in the interpretation of
alternative approach if it satisfies the issues critical for successful conduct of
trial data;
requirements of the applicable statutes clinical research. • Appropriate safety evaluation
and regulations. DATES: The training course will be held during studies; and
II. Paperwork Reduction Act of 1995
on November 7, 2016, from 8:20 a.m. to • The ethical considerations and
5:30 p.m. (registration begins at 7:30 regulatory requirements for clinical
This guidance refers to previously a.m.); on November 8, 2016, from 8:30 trials.
approved collections of information a.m. to 4:45 p.m.; and on November 9, In addition, the course aims to:
found in FDA regulations. These 2016, from 8:30 a.m. to 3:30 p.m. • Foster a cadre of clinical
collections of information are subject to ADDRESSES: The course will be held at investigators with knowledge,
review by the Office of Management and the Silver Spring Civic Building at experience, and commitment to
Veterans Plaza, One Veterans Place, investigational medicine;
Budget (OMB) under the Paperwork
Silver Spring, MD 20910. GPS device • Promote communication between
Reduction Act of 1995 (44 U.S.C. 3501– clinical investigators and FDA;
3520). The collections of information in address: 8525 Fenton St., Silver Spring,
MD 20910. For additional information, • Enhance investigators’
21 CFR parts 211 and 610 have been understanding of FDA’s role in
please refer to http://
approved under OMB control number experimental medicine;
www.silverspringdowntown.com/go/
0910–0139 and in 21 CFR part 312
silver-spring-civic-building-and- • Improve the quality of clinical trial
under OMB control number 0910–0014. veterans-plaza. (FDA has verified the data; and
Web address, but FDA is not responsible • Enhance protection of subjects in
III. Electronic Access
for subsequent changes to the Web site clinical trials.
Persons with access to the Internet after this document publishes in the B. Agenda
may obtain the guidance at either http:// Federal Register.)
The course will be conducted over 3
www.fda.gov/BiologicsBloodVaccines/ FOR FURTHER INFORMATION CONTACT: days and will be presented mainly by
GuidanceCompliance Nicole Silva, Office of Medical Policy, senior FDA staff with other lecturers
RegulatoryInformation/Guidances/ Center for Drug Evaluation and presenting on selected topics. The
default.htm or http:// Research, Food and Drug agenda is available at http://www.fda.
www.regulations.gov. Administration, 10903 New Hampshire gov/Training/ClinicalInvestigator
Dated: September 12, 2016. Ave., Bldg. 51, Rm. 6323, Silver Spring, TrainingCourse/default.htm.
MD 20993, 301–796–3419,
Leslie Kux, C. Target Audience
Nicole.Silva@fda.hhs.gov.
mstockstill on DSK3G9T082PROD with NOTICES

Associate Commissioner for Policy.
SUPPLEMENTARY INFORMATION: The course is targeted toward
[FR Doc. 2016–22353 Filed 9–15–16; 8:45 am] clinicians, nurses, pharmacists and
BILLING CODE 4164–01–P I. Background other health care professionals
Clinical trial investigators play a responsible for, or involved in, the
critical role in the development of conduct and/or design of clinical trials.
medical products. They bear the Registration: There is no registration
responsibility for ensuring the safe and fee to attend this in-person training

VerDate Sep<11>2014 17:55 Sep 15, 2016 Jkt 238001 PO 00000 Frm 00033 Fmt 4703 Sfmt 4703 E:\FR\FM\16SEN1.SGM 16SEN1

Document Created: 2016-09-16 00:24:30
Document Modified: 2016-09-16 00:24:30

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 Agency guidances at any time.
Contact		Tami Belouin, 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		81 FR 63766

81 FR 63766 - Recommendations for Microbial Vectors Used for Gene Therapy; Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 81, Issue 180 (September 16, 2016)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration