Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Draft Guidance for Industry; Availability, 5445-5446 [2020-01705]

Download as PDF Federal Register / Vol. 85, No. 20 / Thursday, January 30, 2020 / Notices therapies for on-demand treatment of bleeding to prophylaxis to reduce the frequency of bleeding. GT products for the treatment of hemophilia are being developed as single-dose treatments that may provide long-term expression of the deficient coagulation factor at steady levels to reduce or eliminate the need for exogenous factor replacement. Elsewhere in this issue of the Federal Register, FDA is announcing the availability of two other human gene therapy guidance documents entitled ‘‘Human Gene Therapy for Retinal Disorders; Guidance for Industry’’ and ‘‘Human Gene Therapy for Rare Diseases; Guidance for Industry.’’ In the Federal Register of July 12, 2018 (83 FR 32306), FDA announced the availability of the draft guidance of the same title. FDA received several comments on the draft guidance, and those comments were considered as the guidance was finalized. The guidance announced in this notice finalizes the draft guidance dated July 2018. 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 Hemophilia.’’ 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 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. VerDate Sep<11>2014 16:56 Jan 29, 2020 Jkt 250001 III. Electronic Access Persons with access to the internet may obtain the guidance at either https://www.fda.gov/vaccines-bloodbiologics/guidance-complianceregulatory-information-biologics/ biologics-guidances or https:// www.regulations.gov. Dated: January 27, 2020. Lowell J. Schiller, Principal Associate Commissioner for Policy. [FR Doc. 2020–01702 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–2019–D–5392] Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ‘‘Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.’’ The draft guidance document provides FDA’s current thinking on the criteria to determine sameness of human gene therapy products for the purpose of orphan drug designation and orphan drug exclusivity. The draft guidance is intended to assist stakeholders, including industry and academic sponsors who seek orphan drug designation and orphan drug exclusivity, in the development of gene therapies for rare diseases. DATES: Submit either electronic or written comments on the draft guidance by April 29, 2020 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: 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 PO 00000 Frm 00079 Fmt 4703 Sfmt 4703 5445 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– 2019–D–5392 for ‘‘Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.’’ 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 E:\FR\FM\30JAN1.SGM 30JAN1 5446 Federal Register / Vol. 85, No. 20 / Thursday, January 30, 2020 / Notices 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-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 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: Jenifer Roe, 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: khammond on DSKJM1Z7X2PROD with NOTICES I. Background FDA is announcing the availability of a draft guidance document entitled ‘‘Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.’’ The draft guidance provides FDA’s current thinking on the criteria to determine sameness of human gene therapy products for the purpose of orphan drug designation and orphan drug exclusivity. The draft guidance is intended to assist stakeholders, including industry and academic sponsors who seek orphan drug designation and orphan drug exclusivity, in the development of gene therapies for rare diseases. The draft guidance focuses specifically on factors FDA intends to consider when determining sameness for gene therapy products and does not address sameness determinations for other types of products. 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 ‘‘Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.’’ 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 draft guidance refers to previously approved FDA collections of information. 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 316 have been approved under OMB control number 0910–0167, 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/Biologics BloodVaccines/GuidanceCompliance RegulatoryInformation/Guidances/ default.htm or https:// www.regulations.gov. Dated: January 27, 2020. Lowell J. Schiller, Principal Associate Commissioner for Policy. [FR Doc. 2020–01705 Filed 1–29–20; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket Nos. FDA–2018–N–3442; FDA– 2013–N–0557; FDA–2013–N–0514; FDA– 2013–N–0190; FDA–2013–N–1428; FDA– 2019–N–0075; FDA–2016–N–2544; FDA– 2019–N–2778; FDA–2012–N–0977; FDA– 2013–N–0823; FDA–2009–N–0380; FDA– 2013–N–1147; FDA–2010–N–0117; FDA– 2010–D–0350; FDA–2010–D–0319; FDA– 2012–D–0530; and FDA–2016–N–2683] Agency Information Collection Activities; Announcement of Office of Management and Budget Approvals AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA) is publishing a list of information collections that have been approved by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995. FOR FURTHER INFORMATION CONTACT: Ila S. Mizrachi, Office of Operations, Food and Drug Administration, Three White Flint North, 10A–12M, 11601 Landsdown St. North Bethesda, MD 20852, 301–796–7726, PRAStaff@ fda.hhs.gov. SUMMARY: The following is a list of FDA information collections recently approved by OMB under section 3507 of the Paperwork Reduction Act of 1995 (44 U.S.C. 3507). The OMB control number and expiration date of OMB approval for each information collection are shown in table 1. Copies of the supporting statements for the information collections are available on the internet at https://www.reginfo.gov/public/do/ PRAMain. An Agency may not conduct or sponsor, and a person is not required to respond to, a collection of information unless it displays a currently valid OMB control number. SUPPLEMENTARY INFORMATION: TABLE 1—LIST OF INFORMATION COLLECTIONS APPROVED BY OMB OMB control number Title of collection Web-Based Pilot Survey to Assess Allergy to Cosmetics in the United States ..................................................... Postmarket Surveillance of Medical Devices .......................................................................................................... Administrative Procedures for Clinical Laboratory Improvement Amendments Categorization ............................. VerDate Sep<11>2014 16:56 Jan 29, 2020 Jkt 250001 PO 00000 Frm 00080 Fmt 4703 Sfmt 4703 E:\FR\FM\30JAN1.SGM 30JAN1 0910–0881 0910–0449 0910–0607 Date approval expires 1/31/2021 11/30/2022 11/30/2022

Agencies

[Federal Register Volume 85, Number 20 (Thursday, January 30, 2020)]
[Notices]
[Pages 5445-5446]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01705]

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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-5392]

Interpreting Sameness of Gene Therapy Products Under the Orphan
Drug Regulations; 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 ``Interpreting Sameness
of Gene Therapy Products Under the Orphan Drug Regulations.'' The draft
guidance document provides FDA's current thinking on the criteria to
determine sameness of human gene therapy products for the purpose of
orphan drug designation and orphan drug exclusivity. The draft guidance
is intended to assist stakeholders, including industry and academic
sponsors who seek orphan drug designation and orphan drug exclusivity,
in the development of gene therapies for rare diseases.

DATES: Submit either electronic or written comments on the draft
guidance by April 29, 2020 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-2019-D-5392 for ``Interpreting Sameness of Gene Therapy Products
Under the Orphan Drug Regulations.'' 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

[[Page 5446]]

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 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: Jenifer Roe, 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 guidance document
entitled ``Interpreting Sameness of Gene Therapy Products Under the
Orphan Drug Regulations.'' The draft guidance provides FDA's current
thinking on the criteria to determine sameness of human gene therapy
products for the purpose of orphan drug designation and orphan drug
exclusivity. The draft guidance is intended to assist stakeholders,
including industry and academic sponsors who seek orphan drug
designation and orphan drug exclusivity, in the development of gene
therapies for rare diseases. The draft guidance focuses specifically on
factors FDA intends to consider when determining sameness for gene
therapy products and does not address sameness determinations for other
types of products.
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 ``Interpreting
Sameness of Gene Therapy Products Under the Orphan Drug Regulations.''
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 draft guidance refers to previously approved FDA collections
of information. 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 316 have been approved under OMB control
number 0910-0167, 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: January 27, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01705 Filed 1-29-20; 8:45 am]
BILLING CODE 4164-01-P

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