Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability, 5454-5455 [2020-01703]

Download as PDF 5454 Federal Register / Vol. 85, No. 20 / Thursday, January 30, 2020 / Notices biologics-guidances or https:// www.regulations.gov. Dated: January 27, 2020. Lowell J. Schiller, Principal Associate Commissioner for Policy. [FR Doc. 2020–01710 Filed 1–29–20; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2018–D–2236] Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration is announcing the availability of a final guidance entitled ‘‘Human Gene Therapy for Retinal Disorders; Guidance for Industry.’’ The final guidance provides recommendations to stakeholders developing human gene therapy (GT) products for retinal disorders affecting adult and pediatric patients. The 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. The guidance announced in this notice finalizes the draft guidance of the same title dated July 2018. DATES: The announcement of the guidance is published in the Federal Register on January 30, 2020. ADDRESSES: You may submit either electronic or written comments on Agency guidances at any time as follows: khammond on DSKJM1Z7X2PROD with NOTICES SUMMARY: 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 VerDate Sep<11>2014 16:56 Jan 29, 2020 Jkt 250001 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; 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 PO 00000 Frm 00088 Fmt 4703 Sfmt 4703 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.govinfo.gov/content/pkg/FR-201509-18/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 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: Jenifer Stach, 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 ‘‘Human Gene Therapy for Retinal Disorders; Guidance for Industry.’’ This guidance provides recommendations to stakeholders developing human 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. This 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. E:\FR\FM\30JAN1.SGM 30JAN1 Federal Register / Vol. 85, No. 20 / Thursday, January 30, 2020 / Notices In the Federal Register of July 12, 2018 (83 FR 32302), FDA announced the availability of the draft guidance of the same title. FDA received several comments on the draft guidance and those comments were considered as the guidance was finalized. The guidance announced in this notice finalizes the draft guidance dated July 2018. Elsewhere in this issue of the Federal Register, FDA is announcing the availability of two other human gene therapy final guidance documents entitled ‘‘Human Gene Therapy for Hemophilia; Guidance for Industry’’ and ‘‘Human Gene Therapy for Rare Diseases; Guidance for Industry.’’ 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 ‘‘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. khammond on DSKJM1Z7X2PROD with NOTICES 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– 3521). 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 guidance at either https://www.fda.gov/vaccines-bloodbiologics/guidance-compliance- VerDate Sep<11>2014 16:56 Jan 29, 2020 Jkt 250001 regulatory-information-biologics or https://www.regulations.gov. Dated: January 27, 2020. Lowell J. Schiller, Principal Associate Commissioner for Policy. [FR Doc. 2020–01703 Filed 1–29–20; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Health Resources and Services Administration Agency Information Collection Activities: Submission to OMB for Review and Approval; Public Comment Request; Nurse Corps Loan Repayment Program, OMB No. 0915– 0140 Revision Health Resources and Services Administration (HRSA), Department of Health and Human Services. ACTION: Notice. AGENCY: In compliance with the Paperwork Reduction Act of 1995, HRSA submitted an Information Collection Request (ICR) to the Office of Management and Budget (OMB) for review and approval. Comments submitted during the first public review of this ICR will be provided to OMB. OMB will accept further comments from the public during the review and approval period. OMB may act on HRSA’s ICR only after the 30-day comment period for this Notice has closed. SUMMARY: Comments on this ICR should be received no later than March 2, 2020. ADDRESSES: Submit your comments, including the ICR Title, to the desk officer for HRSA, either by email to OIRA_submission@omb.eop.gov or by fax to (202) 395–5806. FOR FURTHER INFORMATION CONTACT: To request a copy of the clearance requests submitted to OMB for review, email Lisa Wright-Solomon, the HRSA Information Collection Clearance Officer at paperwork@hrsa.gov or call (301) 443– 1984. SUPPLEMENTARY INFORMATION: Information Collection Request Title: Nurse Corps Loan Repayment Program OMB No. 0915–0140—Revision. Abstract: The Nurse Corps Loan Repayment Program (Nurse Corps LRP) assists in the recruitment and retention of professional Registered Nurses (RNs) by decreasing the financial barriers associated with pursuing a nursing education. RNs in this instance include advanced practice RNs (e.g., nurse practitioners, certified registered nurse DATES: PO 00000 Frm 00089 Fmt 4703 Sfmt 4703 5455 anesthetists, certified nurse-midwives, and clinical nurse specialists) dedicated to working at eligible health care facilities with a critical shortage of nurses (i.e., a Critical Shortage Facility) or working as nurse faculty in eligible, accredited schools of nursing. The Nurse Corps LRP provides loan repayment assistance to these nurses to repay a portion of their qualifying educational loans in exchange for fulltime service at a public or private nonprofit Critical Shortage Facility (CSF) or in an eligible, accredited school of nursing. A 60-day notice was published in the Federal Register on October, 10, 2019 vol. 84, No. 197; pp. 54617–51619. Need and Proposed Use of the Information: This information collection is used by the Nurse Corps program to make award decisions about Nurse Corps LRP applicants and to monitor a participant’s compliance with the program’s service requirements. Individuals must submit an application in order to participate in the program. The application asks for personal, professional, educational, and financial information required to determine the applicant’s eligibility to participate in the Nurse Corps LRP. The revised information collection request includes a new form and updates to existing forms for the Nurse Corps LRP in order to expand the service options for awarded participants, promote the use of telehealth for delivering care throughout the nation especially in rural areas, and to reduce the application burden on respondents. New Form #1—Applicants will be asked to submit a Disadvantaged Background Form. This new form asks the applicant’s site Point of Contact to certify whether the applicant is from a disadvantaged background. The form provides eligibility criteria for the determination. Updated Form #1—The Participant Semi-Annual Employment Verification Form will be updated to include additional information about the participant’s service including information about telehealth services and whether they work at multiple CSF sites. Telehealth helps expand the reach of providers especially in rural areas where medical service sites are more remote. The information collected will assist Program with determining the impact and utilization of telehealth services in various health care settings which will be used to inform our telehealth policies. Enabling multiple CSF site service will also allow greater flexibility for providers who rotate or split time between multiple sites which E:\FR\FM\30JAN1.SGM 30JAN1

Agencies

[Federal Register Volume 85, Number 20 (Thursday, January 30, 2020)]
[Notices]
[Pages 5454-5455]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01703]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-2236]


Human Gene Therapy for Retinal Disorders; Guidance for Industry; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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

SUMMARY: The Food and Drug Administration is announcing the 
availability of a final guidance entitled ``Human Gene Therapy for 
Retinal Disorders; Guidance for Industry.'' The final guidance provides 
recommendations to stakeholders developing human gene therapy (GT) 
products for retinal disorders affecting adult and pediatric patients. 
The 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. 
The guidance announced in this notice finalizes the draft guidance of 
the same title dated July 2018.

DATES: The announcement of the guidance is published in the Federal 
Register on January 30, 2020.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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; 
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.govinfo.gov/content/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 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: Jenifer Stach, 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 ``Human 
Gene Therapy for Retinal Disorders; Guidance for Industry.'' This 
guidance provides recommendations to stakeholders developing human 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. This 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.

[[Page 5455]]

    In the Federal Register of July 12, 2018 (83 FR 32302), FDA 
announced the availability of the draft guidance of the same title. FDA 
received several comments on the draft guidance and those comments were 
considered as the guidance was finalized. The guidance announced in 
this notice finalizes the draft guidance dated July 2018.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of two other human gene therapy final guidance 
documents entitled ``Human Gene Therapy for Hemophilia; Guidance for 
Industry'' and ``Human Gene Therapy for Rare Diseases; Guidance for 
Industry.''
    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 ``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.

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. 3501-3521). 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 guidance at 
either https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics or https://www.regulations.gov.

    Dated: January 27, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01703 Filed 1-29-20; 8:45 am]
 BILLING CODE 4164-01-P
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