[Federal Register Volume 87, Number 211 (Wednesday, November 2, 2022)]
[Notices]
[Pages 66191-66192]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-23785]



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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-D-0446]


Expanded Access to Investigational Drugs for Treatment Use: 
Questions and Answers; Draft Guidance for Industry; Availability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the availability of a revised draft guidance for industry entitled 
``Expanded Access to Investigational Drugs for Treatment Use: Questions 
and Answers.'' Since 2017, FDA has received many questions concerning 
implementation of the regulatory requirements of the expanded access 
program. In addition, FDA developed recommendations for fulfilling the 
new requirements for expanded access submissions promulgated in the 
21st Century Cures Act (Cures Act) (2016) and the FDA Reauthorization 
Act of 2017 (FDARA). FDA is providing this guidance in a question-and-
answer format, addressing the most recent frequently asked questions 
and sharing recommendations to fulfill the new statutory requirements. 
This guidance revises the guidance of the same title issued in June 
2016 and updated in October 2017.

DATES: Submit either electronic or written comments on the draft 
guidance by January 3, 2023 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-2013-D-0446 for ``Expanded Access to Investigational Drugs for 
Treatment Use: Questions and Answers.'' 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.
     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; 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 draft 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 
Stephen Ripley, 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 revised draft guidance for 
industry entitled ``Expanded Access to Investigational Drugs for 
Treatment Use: Questions and Answers.'' Under the expanded access 
regulation provided in 21 CFR part 312, subpart I, FDA allows

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use of investigational drugs for treatment of patients with serious or 
immediately life-threatening diseases or conditions who lack 
therapeutic alternatives. FDA issued a guidance in 2016 (updated in 
2017) in a question-and-answer format to respond to the most frequently 
asked questions on various provisions of the regulation regarding 
expanded access.
    The Cures Act added section 561A to the Federal Food, Drug, and 
Cosmetic Act (FD&C Act) (21 U.S.C. 360bbb-0) to include new 
requirements regarding expanded access. Under section 561A of the FD&C 
Act, a manufacturer or distributor of one or more investigational drugs 
for the diagnosis, monitoring, or treatment of one or more serious 
diseases or conditions is required to make its policy for evaluating 
and responding to expanded access requests (expanded access policy) 
readily available to the public, such as by posting the policy on a 
publicly available website. In addition, FDARA (Pub. L. 115-52) amended 
the FD&C Act to require that the expanded access policy for an 
investigational drug be posted by the earlier of (1) the first 
initiation of a phase 2 or phase 3 study with respect to such 
investigational drug or (2) within 15 days after the drug receives a 
fast track, breakthrough, or regenerative advanced therapy designation.
    This revised draft guidance includes responses to stakeholder 
questions received since publication of the updated final guidance in 
2017 and includes the Agency's recommendations related to new 
requirements of the Cures Act and FDARA that are related to expanded 
access. This guidance revises the guidance for industry of the same 
title issued in June 2016 and updated in October 2017. Significant 
changes to the 2017 version of the guidance include additional 
recommendations related to institutional review board review, informed 
consent, and new requirements established by the Cures Act and FDARA.
    This revised draft guidance is being issued consistent with FDA's 
good guidance practices regulation (21 CFR 10.115). The revised draft 
guidance, when finalized, will represent the current thinking of FDA on 
``Expanded Access to Investigational Drugs for Treatment Use: Questions 
and Answers.'' 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 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 (PRA) (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 have been approved under OMB control 
number 0910-0014; the collections of information in 21 CFR parts 50 and 
56 have been approved under OMB control number 0910-0130; the 
collections of information under the Expanded Access regulations (21 
CFR 312.300 through 312.320) have been approved under OMB control 
number 0910-0814; and the collections of information in 42 CFR part 11 
have been approved under OMB control number 0925-0586.

III. Electronic Access

    Persons with access to the internet may obtain the revised draft 
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/biologics-guidances, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: October 27, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-23785 Filed 11-1-22; 8:45 am]
BILLING CODE 4164-01-P