[Federal Register Volume 86, Number 2 (Tuesday, January 5, 2021)]
[Notices]
[Pages 314-315]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-29119]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-D-2199]


Investigational New Drug Submissions for Individualized Antisense 
Oligonucleotide Drug Products: Administrative and Procedural 
Recommendations; Draft Guidance for Sponsor-Investigators; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``IND 
Submissions for Individualized Antisense Oligonucleotide Drug Products: 
Administrative and Procedural Recommendations.'' FDA is publishing this 
draft guidance to help sponsor-investigators (hereafter referred to as 
sponsors) developing individualized antisense oligonucleotide (ASO) 
drug products for a severely debilitating or life-threatening genetic 
disease. Most often, individuals with such diseases will not have 
adequate alternative therapy available for treating their disease. This 
draft guidance is intended to help sponsors of such development 
programs, who may be relatively unfamiliar with FDA regulations, 
processes, and practices, with the administrative and procedural 
aspects of interacting with FDA, including seeking feedback from FDA on 
their development programs and making regulatory submissions related to 
these development programs.

DATES: Submit either electronic or written comments on the draft 
guidance by March 8, 2021 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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 Submission

    Submit electronic comments in the following way:
    <bullet> 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.
    <bullet> 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:
    <bullet> 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.
    <bullet> 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-2020-D-2199 for ``IND Submissions for Individualized Antisense 
Oligonucleotide Drug Products: Administrative and Procedural 
Recommendations.'' 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, 240-402-7500.
    <bullet> 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, 240-402-7500.
    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 Division of Drug Information, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10001 New Hampshire Ave., 
Hillandale Building, 4th Floor, Silver Spring, MD 20993-0002. Send one 
self-addressed adhesive label to assist that office in processing your 
requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the draft guidance document.

[[Page 315]]


FOR FURTHER INFORMATION CONTACT: Colleen Locicero, Office of New Drugs, 
Center for Drug Evaluation and Research, Food and Drug Administration, 
10903 New Hampshire Avenue, Silver Spring, MD 20903, 301-796-1114.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``IND Submissions for Individualized Antisense Oligonucleotide 
Drug Products: Administrative and Procedural Recommendations.'' This 
draft guidance is intended to help sponsors developing individualized 
ASO drug products for a severely debilitating or life-threatening 
genetic disease.
    The draft guidance addresses the approach for obtaining feedback 
from FDA, the expectations and process for making regulatory 
submissions to FDA, and high-level recommendations related to the 
requirement for institutional review board review of protocols for 
trials of individualized ASO drug products and the informed consent of 
participants. The draft guidance discusses the importance of early 
interaction with FDA, submission expectations for pre-investigational 
new drug (IND) meeting packages and IND applications, and ethical and 
human subject protection considerations.
    The draft guidance is intended to help sponsors of such development 
programs, who may be relatively unfamiliar with FDA regulations, 
processes, and practices, seek feedback from FDA on their development 
programs and make regulatory submissions related to these development 
programs. The draft guidance is expected to facilitate the preparation 
of adequate pre-IND and IND submissions for review by the Agency, which 
may help enable prompt initiation of the investigation.
    This draft guidance represents the first of several guidances FDA 
intends to publish to advise and help sponsors developing 
individualized ASO drug products for patients who have severely 
debilitating or life-threatening diseases or conditions and no adequate 
alternative therapy available to them to treat their disease or 
condition.
    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 ``IND 
Submissions for Individualized Antisense Oligonucleotide Drug Products: 
Administrative and Procedural Recommendations.'' 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

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. Therefore, 
clearance by the Office of Management and Budget (OMB) under the 
Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3521) is not required 
for this guidance. The previously approved collections of information 
are subject to review by OMB under the PRA. The collections of 
information in 21 CFR part 312 for the submission of IND applications, 
amendments, and safety reports; for investigator brochures; and for 
requesting a pre-IND meeting have been approved under OMB control 
number 0910-0014; the collections of information for paper submissions 
of Form FDA 3500A have been approved under OMB control number 0910-
0291; the collections of information for electronic submissions of Form 
FDA 3500 have been approved under OMB control number 0910-0645; the 
collections of information in 21 CFR parts 50 and 56 for obtaining 
informed consent for prospective patients have been approved under OMB 
control number 0910-0755.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs or https://www.regulations.gov.

    Dated: December 23, 2020.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2020-29119 Filed 1-4-21; 8:45 am]
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