83 FR 32302 - Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry; Availability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 | 32302-32303 | |
| FR Document | 2018-14870 |
[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)] [Notices] [Pages 32302-32303] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-14870] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2018-D-2236] Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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(FDA or Agency) is announcing the availability of a draft document entitled ``Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry.'' The draft guidance provides recommendations to stakeholders developing human gene therapy (GT) products for retinal disorders affecting adult and pediatric patients. The draft guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products. DATES: Submit either electronic or written comments on the draft guidance by October 10, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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-2236 for ``Human Gene Therapy for Retinal Disorders; 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 [[Page 32303]] 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: Angela Moy, 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 ``Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry.'' The draft guidance provides recommendations to stakeholders developing GT products for retinal disorders affecting adult and pediatric patients. These disorders vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. These disorders manifest with central or peripheral visual impairment and often with progressive visual loss. The draft guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products. Elsewhere in this issue of the Federal Register, FDA is announcing the availability of two other human gene therapy draft guidance documents entitled ``Human Gene Therapy for Hemophilia; Draft Guidance for Industry'' and ``Human Gene Therapy for Rare Diseases; 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 ``Human Gene Therapy for Retinal Disorders.'' 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 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 50 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; the collections of information in 21 CFR part 211 have been approved under OMB control number 0910-0139; the collections of information in 21 CFR part 312 have been approved under OMB control number 0910-0014; the collections of information in 21 CFR part 601 have been approved under OMB control number 0910-0338; the collections of information in the guidance entitled ``Expedited Programs for Serious Conditions-- Drugs and Biologics'' have been approved under OMB control number 0910- 0765; and the collections of information in the guidance entitled ``Formal Meetings Between the FDA and Sponsors or Applicants'' have been approved under OMB control number 0910-0429. 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. 2018-14870 Filed 7-11-18; 8:45 am] BILLING CODE 4164-01-P
![32302 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices
DATES: Authority for the AdvisoryCommittee/default.htm or by including attachments, to https://
Psychopharmacologic Drugs Advisory contacting the Designated Federal www.regulations.gov will be posted to
Committee will expire on June 4, 2020, Officer (see FOR FURTHER INFORMATION the docket unchanged. Because your
unless the Commissioner formally CONTACT). In light of the fact that no comment will be made public, you are
determines that renewal is in the public change has been made to the committee solely responsible for ensuring that your
interest. name or description of duties, no comment does not include any
FOR FURTHER INFORMATION CONTACT: amendment will be made to 21 CFR confidential information that you or a
Kalyani Bhatt, Center for Drug 14.100. third party may not wish to be posted,
Evaluation and Research, Food and This document is issued under the such as medical information, your or
Drug Administration, 10903 New Federal Advisory Committee Act (5 anyone else’s Social Security number, or
Hampshire Ave., Bldg. 31, Rm. 2417, U.S.C. app.). For general information confidential business information, such
Silver Spring, MD 20993–0002, 301– related to FDA advisory committees, as a manufacturing process. Please note
796–9001, email: PDAC@fda.hhs.gov. please check https://www.fda.gov/ that if you include your name, contact
AdvisoryCommittees/default.htm. information, or other information that
SUPPLEMENTARY INFORMATION: Pursuant
identifies you in the body of your
to 41 CFR 102–3.65 and approval by the Dated: July 9, 2018.
comments, that information will be
Department of Health and Human Leslie Kux,
posted on https://www.regulations.gov.
Services pursuant to 45 CFR part 11 and Associate Commissioner for Policy. • If you want to submit a comment
by the General Services Administration, [FR Doc. 2018–14934 Filed 7–11–18; 8:45 am] with confidential information that you
FDA is announcing the renewal of the BILLING CODE 4164–01–P do not wish to be made available to the
Psychopharmacologic Drugs Advisory public, submit the comment as a
Committee (the Committee). The written/paper submission and in the
Committee is a discretionary Federal DEPARTMENT OF HEALTH AND manner detailed (see ‘‘Written/Paper
advisory committee established to HUMAN SERVICES Submissions’’ and ‘‘Instructions’’).
provide advice to the Commissioner.
The Committee advises the Food and Drug Administration Written/Paper Submissions
Commissioner or designee in [Docket No. FDA–2018–D–2236] Submit written/paper submissions as
discharging responsibilities as they follows:
relate to helping to ensure safe and Human Gene Therapy for Retinal • Mail/Hand Delivery/Courier (for
effective drugs for human use and, as Disorders; Draft Guidance for Industry; Written/Paper Submissions): Dockets
required, any other product for which Availability Management Staff (HFA–305), Food and
FDA has regulatory responsibility. Drug Administration, 5630 Fishers
The Committee reviews and evaluates AGENCY: Food and Drug Administration, Lane, Rm. 1061, Rockville, MD 20852.
data concerning the safety and HHS. • For written/paper comments
effectiveness of marketed and ACTION: Notice of availability. submitted to the Dockets Management
investigational human drug products for Staff, FDA will post your comment, as
use in the practice of psychiatry and SUMMARY: The Food and Drug
well as any attachments, except for
related fields and makes appropriate Administration (FDA or Agency) is
information submitted, marked and
recommendations to the Commissioner. announcing the availability of a draft
identified, as confidential, if submitted
The Committee shall consist of a core document entitled ‘‘Human Gene
as detailed in ‘‘Instructions.’’
of nine voting members including the Therapy for Retinal Disorders; Draft Instructions: All submissions received
Chair. Members and the Chair are Guidance for Industry.’’ The draft must include the Docket No. FDA–
selected by the Commissioner or guidance provides recommendations to 2018–D–2236 for ‘‘Human Gene
designee from among authorities stakeholders developing human gene Therapy for Retinal Disorders; Draft
knowledgeable in the fields of therapy (GT) products for retinal Guidance for Industry.’’ Received
psychopharmacology, psychiatry, disorders affecting adult and pediatric comments will be placed in the docket
epidemiology or statistics, and related patients. The draft guidance focuses on and, except for those submitted as
specialties. Members will be invited to issues specific to GT products for retinal ‘‘Confidential Submissions,’’ publicly
serve for overlapping terms of up to 4 disorders and provides viewable at https://www.regulations.gov
years. Almost all non-Federal members recommendations related to product or at the Dockets Management Staff
of this committee serve as Special development, preclinical testing, and between 9 a.m. and 4 p.m., Monday
Government Employees. The core of clinical trial design for such GT through Friday.
voting members may include one products. • Confidential Submissions—To
technically qualified member, selected DATES: Submit either electronic or submit a comment with confidential
by the Commissioner or designee, who written comments on the draft guidance information that you do not wish to be
is identified with consumer interests by October 10, 2018 to ensure that the made publicly available, submit your
and is recommended by either a Agency considers your comment on this comments only as a written/paper
consortium of consumer-oriented draft guidance before it begins work on submission. You should submit two
organizations or other interested the final version of the guidance. copies total. One copy will include the
persons. In addition to the voting ADDRESSES: You may submit comments information you claim to be confidential
members, the Committee may include on any guidance at any time as follows: with a heading or cover note that states
one non-voting member who is ‘‘THIS DOCUMENT CONTAINS
amozie on DSK3GDR082PROD with NOTICES1
identified with industry interests. Electronic Submissions CONFIDENTIAL INFORMATION.’’ The
Further information regarding the Submit electronic comments in the Agency will review this copy, including
most recent charter and other following way: the claimed confidential information, in
information can be found at https:// • Federal eRulemaking Portal: its consideration of comments. The
www.fda.gov/AdvisoryCommittees/ https://www.regulations.gov. Follow the second copy, which will have the
CommitteesMeetingMaterials/Drugs/ instructions for submitting comments. claimed confidential information
PsychopharmacologicDrugs Comments submitted electronically, redacted/blacked out, will be available
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![Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 32303
for public viewing and posted on management, and include genetic as III. Electronic Access
https://www.regulations.gov. Submit well as age-related diseases. These Persons with access to the internet
both copies to the Dockets Management disorders manifest with central or may obtain the draft guidance at either
Staff. If you do not wish your name and peripheral visual impairment and often https://www.fda.gov/BiologicsBlood
contact information to be made publicly with progressive visual loss. The draft Vaccines/GuidanceCompliance
available, you can provide this guidance focuses on issues specific to RegulatoryInformation/Guidances/
information on the cover sheet and not GT products for retinal disorders and default.htm or https://
in the body of your comments and you provides recommendations related to www.regulations.gov.
must identify this information as product development, preclinical
‘‘confidential.’’ Any information marked Dated: July 5, 2018.
testing, and clinical trial design for such
as ‘‘confidential’’ will not be disclosed Leslie Kux,
GT products.
except in accordance with 21 CFR 10.20 Associate Commissioner for Policy.
and other applicable disclosure law. For Elsewhere in this issue of the Federal [FR Doc. 2018–14870 Filed 7–11–18; 8:45 am]
more information about FDA’s posting Register, FDA is announcing the BILLING CODE 4164–01–P
of comments to public dockets, see 80 availability of two other human gene
FR 56469, September 18, 2015, or access therapy draft guidance documents
the information at: https://www.gpo.gov/ entitled ‘‘Human Gene Therapy for DEPARTMENT OF HEALTH AND
fdsys/pkg/FR-2015-09-18/pdf/2015- Hemophilia; Draft Guidance for HUMAN SERVICES
23389.pdf. Industry’’ and ‘‘Human Gene Therapy
Docket: For access to the docket to for Rare Diseases; Draft Guidance for Food and Drug Administration
read background documents or the Industry.’’ [Docket No. FDA–2018–D–2258]
electronic and written/paper comments This draft guidance is being issued
received, go to https:// consistent with FDA’s good guidance Human Gene Therapy for Rare
www.regulations.gov and insert the practices regulation (21 CFR 10.115). Diseases; Draft Guidance for Industry;
docket number, found in brackets in the The draft guidance, when finalized, will Availability
heading of this document, into the represent the current thinking of FDA AGENCY: Food and Drug Administration,
‘‘Search’’ box and follow the prompts on ‘‘Human Gene Therapy for Retinal HHS.
and/or go to the Dockets Management Disorders.’’ It does not establish any
Staff, 5630 Fishers Lane, Rm. 1061, ACTION: Notice of availability.
rights for any person and is not binding
Rockville, MD 20852. on FDA or the public. You can use an SUMMARY: The Food and Drug
You may submit comments on any alternative approach if it satisfies the Administration (FDA or Agency) is
guidance at any time (see 21 CFR requirements of the applicable statutes announcing the availability of a draft
10.115(g)(5)). and regulations. This guidance is not document entitled ‘‘Human Gene
Submit written requests for single subject to Executive Order 12866. Therapy for Rare Diseases; Draft
copies of the draft guidance to the Office Guidance for Industry.’’ The draft
of Communication, Outreach and II. Paperwork Reduction Act of 1995 guidance document provides
Development, Center for Biologics recommendations to stakeholders
Evaluation and Research (CBER), Food This draft guidance refers to
previously approved collections of developing a human gene therapy (GT)
and Drug Administration, 10903 New product intended to treat a rare disease
Hampshire Ave., Bldg. 71, Rm. 3128, information subject to review by the
in adult and/or pediatric patients
Silver Spring, MD 20993–0002. Send Office of Management and Budget
regarding the manufacturing,
one self-addressed adhesive label to (OMB) under the Paperwork Reduction
preclinical, and clinical trial design
assist the office in processing your Act of 1995 (44 U.S.C. 3501–3520). The
issues for all phases of the clinical
requests. The draft guidance may also be collections of information in 21 CFR
development program. Such
obtained by mail by calling CBER at 1– part 50 have been approved under OMB
information is intended to assist
800–835–4709 or 240–402–8010. See control number 0910–0755; the sponsors in designing clinical
the SUPPLEMENTARY INFORMATION section collections of information in 21 CFR development programs for such
for electronic access to the draft part 58 have been approved under OMB products, where there may be limited
guidance document. control number 0910–0119; the study population size and potential
FOR FURTHER INFORMATION CONTACT: collections of information in 21 CFR feasibility and safety issues as well as
Angela Moy, Center for Biologics part 211 have been approved under issues relating to the interpretability of
Evaluation and Research, Food and OMB control number 0910–0139; the bioactivity/efficacy outcomes that may
Drug Administration, 10903 New collections of information in 21 CFR be unique to rare diseases or to the
Hampshire Ave., Bldg. 71, Rm. 7301, part 312 have been approved under nature of the GT product itself.
Silver Spring, MD 20993–0002, 240– OMB control number 0910–0014; the DATES: Submit either electronic or
402–7911. collections of information in 21 CFR written comments on the draft guidance
SUPPLEMENTARY INFORMATION: part 601 have been approved under by October 10, 2018 to ensure that the
OMB control number 0910–0338; the Agency considers your comment on this
I. Background collections of information in the draft guidance before it begins work on
FDA is announcing the availability of guidance entitled ‘‘Expedited Programs the final version of the guidance.
a draft document entitled ‘‘Human Gene for Serious Conditions—Drugs and ADDRESSES: You may submit comments
amozie on DSK3GDR082PROD with NOTICES1
Therapy for Retinal Disorders; Draft Biologics’’ have been approved under on any guidance at any time as follows:
Guidance for Industry.’’ The draft OMB control number 0910–0765; and
guidance provides recommendations to the collections of information in the Electronic Submissions
stakeholders developing GT products guidance entitled ‘‘Formal Meetings Submit electronic comments in the
for retinal disorders affecting adult and Between the FDA and Sponsors or following way:
pediatric patients. These disorders vary Applicants’’ have been approved under • Federal eRulemaking Portal:
in etiology, prevalence, diagnosis, and OMB control number 0910–0429. https://www.regulations.gov. Follow the
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Document Created: 2018-11-06 10:22:51
Document Modified: 2018-11-06 10:22:51
| 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 | Angela Moy, 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 32302 |
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