83 FR 32311 - Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 134 (July 12, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 134 (July 12, 2018)
| Page Range | 32311-32312 | |
| FR Document | 2018-14867 |
[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)] [Notices] [Pages 32311-32312] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-14867] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2018-D-2173] Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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availability of a draft document entitled ``Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry.'' The draft guidance provides sponsors, who are developing a human gene therapy (GT) product, recommendations regarding the design of long term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a GT product. The draft guidance, when finalized, is intended to supersede the document entitled ``Guidance for Industry: Gene Therapy Clinical Trials--Observing Participants for Delayed Adverse Events'' dated November 2006. This draft guidance, when finalized, is also intended to supplement the guidance entitled ``Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Draft Guidance for Industry.'' DATES: Submit either electronic or written comments on the draft guidance by October 10, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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-2018-D-2173 for ``Long Term Follow-Up After Administration of Human Gene Therapy Products; 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 [[Page 32312]] the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. FOR FURTHER INFORMATION CONTACT: Jonathan McKnight, 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 draft document entitled ``Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry.'' The draft guidance provides a brief introduction of the product characteristics, patient-related factors, and the preclinical and clinical data that should be considered when assessing the need for LTFU observations for your GT product. The draft guidance also describes the Agency's current recommendations for the conduct of LTFU studies, specifically the information/data to support a sponsor's rationale for the duration and design of a LTFU protocol when clinical trials are initiated. Also included in the draft guidance are GT product-specific clinical considerations for monitoring subjects under a LTFU protocol and recommendations on patient monitoring for licensed GT products. The draft guidance, when finalized, is intended to supersede the guidance entitled ``Guidance for Industry: Gene Therapy Clinical Trials-- Observing Participants for Delayed Adverse Events'' dated November 2006. The draft guidance, when finalized, is also intended to supplement the guidance 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,'' published elsewhere in this issue of the Federal Register. Also, elsewhere in this issue of the Federal Register, FDA is announcing the availability of another draft guidance entitled ``Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Draft Guidance for Industry.'' 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 the current thinking of FDA on long term follow-up after administration of human gene therapy products. 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 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 parts 50 and 56 have been approved under OMB control number 0910-0755; the collections of information in 21 CFR part 58 have been approved under OMB control number 0910-0119; and the collections of information in 21 CFR part 312 have been approved under OMB control number 0910-0014. 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. 2018-14867 Filed 7-11-18; 8:45 am] BILLING CODE 4164-01-P
![Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 32311
one non-voting member who is Human Gene Therapy Products for those submitted as ‘‘Confidential
identified with industry interests. Replication Competent Retrovirus Submissions,’’ publicly viewable at
Further information regarding the during Product Manufacture and Patient https://www.regulations.gov or at the
most recent charter and other Follow-up; Draft Guidance for Dockets Management Staff between 9
information can be found at https:// Industry.’’ a.m. and 4 p.m., Monday through
www.fda.gov/AdvisoryCommittees/ DATES: Submit either electronic or Friday.
CommitteesMeetingMaterials/ • Confidential Submissions—To
written comments on the draft guidance
BloodVaccinesandOtherBiologics/ submit a comment with confidential
by October 10, 2018 to ensure that the
AllergenicProductsAdvisoryCommittee/ information that you do not wish to be
Agency considers your comment on this
ucm129360.htm or by contacting the made publicly available, submit your
draft guidance before it begins work on
Designated Federal Officer (see FOR comments only as a written/paper
the final version of the guidance. submission. You should submit two
FURTHER INFORMATION CONTACT). In light
ADDRESSES: You may submit comments copies total. One copy will include the
of the fact that no change has been made
on any guidance at any time as follows: information you claim to be confidential
to the committee name or description of
duties, no amendment will be made to Electronic Submissions with a heading or cover note that states
21 CFR 14.100. ‘‘THIS DOCUMENT CONTAINS
Submit electronic comments in the CONFIDENTIAL INFORMATION.’’ The
This document is issued under the following way:
Federal Advisory Committee Act (5 Agency will review this copy, including
• Federal eRulemaking Portal: the claimed confidential information, in
U.S.C. app.). For general information https://www.regulations.gov. Follow the
related to FDA advisory committees, its consideration of comments. The
instructions for submitting comments. second copy, which will have the
please check https://www.fda.gov/ Comments submitted electronically,
AdvisoryCommittees/default.htm. claimed confidential information
including attachments, to https:// redacted/blacked out, will be available
Dated: July 9, 2018. www.regulations.gov will be posted to for public viewing and posted on
Leslie Kux, the docket unchanged. Because your https://www.regulations.gov. Submit
Associate Commissioner for Policy. comment will be made public, you are both copies to the Dockets Management
[FR Doc. 2018–14942 Filed 7–11–18; 8:45 am] solely responsible for ensuring that your Staff. If you do not wish your name and
BILLING CODE 4164–01–P
comment does not include any contact information to be made publicly
confidential information that you or a available, you can provide this
third party may not wish to be posted, information on the cover sheet and not
DEPARTMENT OF HEALTH AND such as medical information, your or in the body of your comments and you
HUMAN SERVICES anyone else’s Social Security number, or must identify this information as
confidential business information, such ‘‘confidential.’’ Any information marked
Food and Drug Administration as a manufacturing process. Please note as ‘‘confidential’’ will not be disclosed
that if you include your name, contact except in accordance with 21 CFR 10.20
[Docket No. FDA–2018–D–2173]
information, or other information that and other applicable disclosure law. For
Long Term Follow-Up After identifies you in the body of your more information about FDA’s posting
Administration of Human Gene comments, that information will be of comments to public dockets, see 80
Therapy Products; Draft Guidance for posted on https://www.regulations.gov. FR 56469, September 18, 2015, or access
Industry; Availability • If you want to submit a comment the information at: https://www.gpo.gov/
with confidential information that you fdsys/pkg/FR-2015-09-18/pdf/2015-
AGENCY: Food and Drug Administration, do not wish to be made available to the 23389.pdf.
HHS. public, submit the comment as a Docket: For access to the docket to
ACTION: Notice of availability. written/paper submission and in the read background documents or the
manner detailed (see ‘‘Written/Paper electronic and written/paper comments
SUMMARY: The Food and Drug Submissions’’ and ‘‘Instructions’’). received, go to https://
Administration (FDA or Agency) is www.regulations.gov and insert the
announcing the availability of a draft Written/Paper Submissions
docket number, found in brackets in the
document entitled ‘‘Long Term Follow- Submit written/paper submissions as heading of this document, into the
Up After Administration of Human follows: ‘‘Search’’ box and follow the prompts
Gene Therapy Products; Draft Guidance • Mail/Hand Delivery/Courier (for and/or go to the Dockets Management
for Industry.’’ The draft guidance Written/Paper Submissions): Dockets Staff, 5630 Fishers Lane, Rm. 1061,
provides sponsors, who are developing Management Staff (HFA–305), Food and Rockville, MD 20852.
a human gene therapy (GT) product, Drug Administration, 5630 Fishers You may submit comments on any
recommendations regarding the design Lane, Rm. 1061, Rockville, MD 20852. guidance at any time (see 21 CFR
of long term follow-up (LTFU) • For written/paper comments 10.115(g)(5)).
observational studies for the collection submitted to the Dockets Management Submit written requests for single
of data on delayed adverse events Staff, FDA will post your comment, as copies of the draft guidance to the Office
following administration of a GT well as any attachments, except for of Communication, Outreach and
product. The draft guidance, when information submitted, marked and Development, Center for Biologics
finalized, is intended to supersede the identified, as confidential, if submitted Evaluation and Research (CBER), Food
document entitled ‘‘Guidance for as detailed in ‘‘Instructions.’’ and Drug Administration, 10903 New
amozie on DSK3GDR082PROD with NOTICES1
Industry: Gene Therapy Clinical Instructions: All submissions received Hampshire Ave., Bldg. 71, Rm. 3128,
Trials—Observing Participants for must include the Docket No. FDA– Silver Spring, MD 20993–0002. Send
Delayed Adverse Events’’ dated 2018–D–2173 for ‘‘Long Term Follow- one self-addressed adhesive label to
November 2006. This draft guidance, Up After Administration of Human assist the office in processing your
when finalized, is also intended to Gene Therapy Products; Draft Guidance requests. The draft guidance may also be
supplement the guidance entitled for Industry.’’ Received comments will obtained by mail by calling CBER at 1–
‘‘Testing of Retroviral Vector-Based be placed in the docket and, except for 800–835–4709 or 240–402–8010. See
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![32312 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices
the SUPPLEMENTARY INFORMATION section for any person and is not binding on FOR FURTHER INFORMATION CONTACT: Ann
for electronic access to the draft FDA or the public. You can use an Ferrero, Maternal and Child Health
guidance document. alternative approach if it satisfies the Bureau (MCHB), HRSA, in one of three
FOR FURTHER INFORMATION CONTACT: requirements of the applicable statutes ways: (1) Send a request to the following
Jonathan McKnight, Center for Biologics and regulations. This guidance is not address: Ann Ferrero, MCHB, HRSA
Evaluation and Research, Food and subject to Executive Order 12866. 5600 Fishers Lane, Room 18N100C,
Drug Administration, 10903 New II. Paperwork Reduction Act of 1995 Rockville, MD 20857; (2) call 301–443–
Hampshire Ave., Bldg. 71, Rm. 7301, 3999; or (3) send an email to AFerrero@
Silver Spring, MD 20993–0002, 240– This draft guidance refers to hrsa.gov.
402–7911. previously approved collections of
SUPPLEMENTARY INFORMATION:
information found in FDA regulations.
SUPPLEMENTARY INFORMATION: Background: The ACHDNC provides
These collections of information are
advice and recommendations to the
I. Background subject to review by the Office of
Secretary of HHS on the development of
Management and Budget (OMB) under
FDA is announcing the availability of newborn screening activities,
the Paperwork Reduction Act of 1995
a draft document entitled ‘‘Long Term technologies, policies, guidelines, and
(44 U.S.C. 3501–3520). The collections
Follow-Up After Administration of programs for effectively reducing
of information in 21 CFR parts 50 and
Human Gene Therapy Products; Draft morbidity and mortality in newborns
56 have been approved under OMB
Guidance for Industry.’’ The draft and children having, or at risk for,
control number 0910–0755; the
guidance provides a brief introduction heritable disorders. In addition,
collections of information in 21 CFR
of the product characteristics, patient- ACHDNC’s recommendations regarding
part 58 have been approved under OMB
related factors, and the preclinical and inclusion of additional conditions for
control number 0910–0119; and the
clinical data that should be considered screening, following adoption by the
collections of information in 21 CFR
when assessing the need for LTFU Secretary, are evidence-informed
part 312 have been approved under
observations for your GT product. The preventive health services provided for
OMB control number 0910–0014.
draft guidance also describes the in the comprehensive guidelines
Agency’s current recommendations for III. Electronic Access supported by HRSA through the
the conduct of LTFU studies, Persons with access to the internet Recommended Uniform Screening Panel
specifically the information/data to may obtain the draft guidance at either (RUSP) pursuant to section 2713 of the
support a sponsor’s rationale for the https://www.fda.gov/BiologicsBlood Public Health Service Act (42 U.S.C.
duration and design of a LTFU protocol Vaccines/GuidanceCompliance 300gg–13). Under this provision, non-
when clinical trials are initiated. Also RegulatoryInformation/Guidances/ grandfathered group health plans and
included in the draft guidance are GT default.htm or https:// health insurance issuers offering group
product-specific clinical considerations www.regulations.gov. or individual health insurance are
for monitoring subjects under a LTFU required to provide insurance coverage
Dated: July 5, 2018.
protocol and recommendations on without cost-sharing (a co-payment, co-
patient monitoring for licensed GT Leslie Kux, insurance, or deductible) for preventive
products. The draft guidance, when Associate Commissioner for Policy. services for plan years (i.e., policy years)
finalized, is intended to supersede the [FR Doc. 2018–14867 Filed 7–11–18; 8:45 am] beginning on or after the date that is one
guidance entitled ‘‘Guidance for BILLING CODE 4164–01–P year from the Secretary’s adoption of the
Industry: Gene Therapy Clinical condition for screening.
Trials—Observing Participants for Agenda: During the August 2, 2018,
Delayed Adverse Events’’ dated DEPARTMENT OF HEALTH AND meeting, the ACHDNC will discuss
November 2006. The draft guidance, HUMAN SERVICES issues related to long-term follow-up,
when finalized, is also intended to timeliness, education and training, the
Health Resources and Services
supplement the guidance entitled evidence-based review process, and risk
Administration
‘‘Testing of Retroviral Vector-Based assessment in newborn screening.
Human Gene Therapy Products for Advisory Committee on Heritable Information about the ACHDNC, a roster
Replication Competent Retrovirus Disorders in Newborns and Children of members, and the meeting agenda, as
during Product Manufacture and Patient well as past meeting summaries, is
Follow-up; Draft Guidance for AGENCY: Health Resources and Services located on the ACHDNC website:
Industry,’’ published elsewhere in this Administration (HRSA), Department of https://www.hrsa.gov/advisory-
issue of the Federal Register. Also, Health and Human Services (HHS). committees/heritable-disorders/
elsewhere in this issue of the Federal ACTION: Notice of meeting. index.html.
Register, FDA is announcing the Public Participation: Members of the
SUMMARY: In accordance with the public will have the opportunity to
availability of another draft guidance
Federal Advisory Committee Act, this provide comments, which are part of the
entitled ‘‘Chemistry, Manufacturing,
notice announces that the Advisory official Committee record. To submit
and Control Information for Human
Committee on Heritable Disorders in written comments or request time
Gene Therapy Investigational New Drug
Newborns and Children (ACHDNC) will for an oral comment at the meeting,
Applications; Draft Guidance for
hold a public meeting. please register online by 12:00 p.m. ET
Industry.’’
This draft guidance is being issued DATES: Thursday, August 2, 2018, from on July 27, 2018, at http://
amozie on DSK3GDR082PROD with NOTICES1
consistent with FDA’s good guidance 9:30 a.m. to 5:00 p.m. Eastern Time www.achdncmeetings.org. Oral
practices regulation (21 CFR 10.115). (ET). comments will be honored in the order
The draft guidance, when finalized, will ADDRESSES: This meeting is a webinar they are requested and may be limited
represent the current thinking of FDA only and requires advanced registration. as time allows. Individuals associated
on long term follow-up after Please register online at http:// with groups or who plan to provide
administration of human gene therapy www.achdncmeetings.org/ by 12:00 p.m. comments on similar topics may be
products. It does not establish any rights ET on July 30, 2018. asked to combine their comments and
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Document Created: 2018-11-06 10:22:34
Document Modified: 2018-11-06 10:22:34
| 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 | Jonathan McKnight, 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 32311 |
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