[Federal Register Volume 88, Number 174 (Monday, September 11, 2023)]
[Notices]
[Pages 62384-62385]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-19501]



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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-D-3452]


Institutional Review Board Review of Individual Patient Expanded 
Access Submissions for Investigational Drugs and Biological Products; 
Guidance for Institutional Review Boards and Clinical 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 final guidance for institutional review boards 
(IRBs) and clinical investigators entitled ``Institutional Review Board 
(IRB) Review of Individual Patient Expanded Access Submissions for 
Investigational Drugs and Biological Products.'' FDA is issuing this 
final guidance to provide recommendations regarding the key factors and 
procedures IRBs should consider when reviewing individual patient 
expanded access submissions, including for reviews conducted by a 
single member of the IRB, to fulfill its obligations under FDA 
regulations. Although FDA has issued guidance on expanded access 
requests, including expanded access for individual patients, the Agency 
is aware that IRBs seek further clarity on this topic.

DATES: The announcement of the guidance is published in the Federal 
Register on September 11, 2023.

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:
    <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-2023-D-3452 for ``Institutional Review Board (IRB) Review of 
Individual Patient Expanded Access Submissions for Investigational 
Drugs and Biological Products.'' 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 this final 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; or the 
Office of Communication, Outreach and Development, Center for Biologics 
Evaluation and Research, 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 that office in processing 
your requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the guidance document.

FOR FURTHER INFORMATION CONTACT: Dat Doan, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 51, Rm. 3334, Silver Spring, MD 20993-0002, 240-402-8926; or Anne 
Taylor, Center for Biologics Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver 
Spring, MD 20993-0002, 240-402-5683.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a final guidance for IRBs and 
clinical investigators entitled ``Institutional Review Board (IRB) 
Review of Individual Patient Expanded Access Submissions for 
Investigational Drugs and Biological Products.'' FDA is issuing this 
guidance to provide recommendations regarding the key factors and 
procedures IRBs should consider when reviewing individual patient 
expanded access submissions, including for reviews conducted by a 
single member of the IRB, to fulfill its obligations under 21 CFR part 
56.

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Although FDA has issued guidance on expanded access requests, including 
expanded access for individual patients, the Agency is aware that IRBs 
seek further clarity on this topic.
    Under FDA regulations, there are three categories of expanded 
access: individual (also known as single) patient, including for 
emergency use; intermediate-size for intermediate-size patient 
populations; and ``treatment'' for larger populations. This guidance 
only applies to IRB review of individual patient expanded access 
submissions, as outlined in 21 CFR 312.310. The recommendations in this 
guidance are intended to provide additional clarity to assist IRBs in 
conducting efficient reviews of individual patient expanded access 
requests.
    In the Federal Register of June 9, 2020 (85 FR 35311), FDA 
announced the availability of a guidance for IRBs and investigators 
entitled ``Institutional Review Board (IRB) Review of Individual 
Patient Expanded Access Requests for Investigational Drugs and 
Biological Products During the COVID-19 Public Health Emergency: 
Guidance for IRBs and Clinical Investigators'' (2020 COVID-19 guidance) 
to support public health efforts following a determination, under 
section 319 of the Public Health Service (PHS) Act (42 U.S.C. 247d), by 
the Secretary of Health and Human Services that a public health 
emergency existed related to Coronavirus Disease 2019 (COVID-19 public 
health emergency). The 2020 COVID-19 guidance focused on addressing the 
COVID-19 public health emergency and was intended to remain in effect 
only for the duration of the COVID-19 public health emergency. However, 
the 2020 COVID-19 guidance explained we expected the recommendations 
would assist the Agency more broadly in its continued efforts to 
facilitate access to drugs through expanded access for individual 
patients beyond the COVID-19 public health emergency and that FDA would 
replace the 2020 COVID-19 guidance with any appropriate changes based 
on comments received and the Agency's experience with implementation. 
FDA continues to believe that many of the recommendations set forth in 
the 2020 COVID-19 guidance are applicable outside the context of the 
COVID-19 public health emergency and are applicable to key factors and 
procedures IRBs should consider when reviewing individual patient 
expanded access submissions. In addition, in the Federal Register of 
March 13, 2023 (88 FR 15417), FDA listed the 2020 COVID-19 guidance as 
one of the guidances FDA was revising to continue in effect for 180 
days after the COVID-19 PHE declaration issued under the PHS Act 
expired on May 11, 2023, during which time FDA planned to further 
revise those guidances. Consistent with what we said in the Federal 
Register of March 13, 2023, FDA is therefore issuing this revised final 
guidance, which supersedes the 2020 COVID-19 guidance. FDA revised the 
guidance to remove references to the COVID-19 public health emergency 
and made editorial changes to improve clarity. FDA also clarified 
recommendations on IRB procedures and factors to consider for 
individual patient expanded access submissions.
    FDA is issuing this guidance for immediate implementation in 
accordance with our good guidance practices regulation (21 CFR 
10.115(g)(3)) without initially seeking prior comment because the 
Agency has determined that prior public participation is not feasible 
or appropriate (see 21 CFR 10.115(g)(2) and section 701(h)(1)(C)(i) of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 371(h)(1)(C)(i))). 
Specifically, we are not seeking prior comment because public health 
emergencies (PHEs) and the need to address individual patient expanded 
access submissions for patient access to investigational drugs for 
diagnosing, monitoring, or treating diseases or conditions related to 
PHEs may occur without notice and, as we have learned from experience 
during the COVID-19 PHE, may hinder physicians seeking to treat their 
patients in a timely manner. It is thus important to public health to 
provide recommendations regarding the key factors and procedures IRBs 
should consider when reviewing requests. Interested parties had an 
opportunity to comment on the recommendations in the 2020 COVID-19 
guidance, and FDA considered those comments when revising the guidance. 
Although this guidance document is being implemented immediately, it 
remains subject to comment in accordance with FDA's good guidance 
practices regulation (Sec.  10.115(g)(3)(D)).
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). This guidance represents the 
current thinking of FDA on ``Institutional Review Board (IRB) Review of 
Individual Patient Expanded Access Submissions for Investigational 
Drugs and Biological 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.

II. Paperwork Reduction Act of 1995

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. The 
previously approved collections of information are subject to review by 
the Office of Management and Budget (OMB) under the Paperwork Reduction 
Act of 1995 (PRA) (44 U.S.C. 3501-3521). The collections of information 
in 21 CFR part 312 and Form FDA 1571 have been approved under OMB 
control number 0910-0014. The collections of information in 21 CFR 
parts 50 and 56 relating to the protection of human subjects, informed 
consent, and IRBs have been approved under OMB control number 0910-
0130. The collections of information in 21 CFR 312.300 through 312.320 
relating to expanded access to investigational drugs for treatment use 
and Form FDA 3926 have been approved under OMB control number 0910-
0814. The collections of information in 21 CFR part 11 relating to 
electronic records and signatures have been approved under OMB control 
number 0910-0303.

III. Electronic Access

    Persons with access to the internet may obtain the guidance at 
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: September 6, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-19501 Filed 9-8-23; 8:45 am]
BILLING CODE 4164-01-P