Notice2025-19984
Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers; Guidance for Industry; Availability
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
November 17, 2025
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers." This guidance finalizes the revised draft guidance issued on November 2, 2022, and replaces the final guidance of the same title issued in June 2016 and updated in October 2017. The guidance addresses frequently asked questions, including those related to the implementation of FDA's regulations on expanded access to investigational drugs and other topics related to expanded access that are promulgated through the 21st Century Cures Act (Cures Act) and the FDA Reauthorization Act of 2017 (FDARA). Upon publication of this guidance, FDA is withdrawing the 1998 information sheet guidance, entitled "Emergency Use of an Investigational Drug or Biologic," for institutional review boards and clinical investigators because the information provided in the 1998 guidance is included in this final guidance.
Full Text
<html>
<head>
<title>Federal Register, Volume 90 Issue 219 (Monday, November 17, 2025)</title>
</head>
<body><pre>
[Federal Register Volume 90, Number 219 (Monday, November 17, 2025)]
[Notices]
[Pages 51369-51371]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2025-19984]
=======================================================================
-----------------------------------------------------------------------
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; 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 final guidance for industry entitled ``Expanded
Access to Investigational Drugs for Treatment Use: Questions and
Answers.'' This guidance finalizes the revised draft guidance issued on
November 2, 2022, and replaces the final guidance of the same title
issued in June 2016 and updated in October 2017. The guidance addresses
frequently asked questions, including those related to the
implementation of FDA's regulations on expanded access to
investigational drugs and other topics related to expanded access that
are promulgated through the 21st Century Cures Act (Cures Act) and the
FDA Reauthorization Act of 2017 (FDARA). Upon publication of this
guidance, FDA is withdrawing the 1998 information sheet guidance,
entitled ``Emergency Use of an Investigational Drug or Biologic,'' for
institutional review boards and clinical investigators because the
information provided in the 1998 guidance is included in this final
guidance.
DATES: The announcement of the guidance is published in the Federal
Register on November 17, 2025.
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: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the
[[Page 51370]]
instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<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-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 <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 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 guidance document.
FOR FURTHER INFORMATION CONTACT: Dorothy West, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-2500; or Phillip
Kurs, Center for Biologics Evaluation and Research, Food and Drug
Administration, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry
entitled ``Expanded Access to Investigational Drugs for Treatment Use:
Questions and Answers.'' Expanded access refers to the use of an
investigational drug when the primary purpose is to diagnose, monitor,
or treat a patient's disease or condition (see section 561(a) of the
Federal Food, Drug, and Cosmetic Act (FD&C Act); 21 CFR 312.300(a)),
rather than to obtain the kind of information about the drug that is
generally derived from clinical trials. FDA issued a final guidance for
industry in 2016 in a question-and-answer format to respond to the most
frequently asked questions on various provisions of the regulation
regarding expanded access. The 2016 guidance was updated in 2017.
Since the issuance of the updated final guidance in 2017, the Cures
Act (Pub. L. 114-255) added section 561A to the 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.
FDA issued a revised draft guidance of the same title on November
2, 2022 (87 FR 66191), to respond to questions received from interested
parties since issuance of the updated final guidance in 2017. This
guidance finalizes the revised draft guidance issued in November 2022
and includes the Agency's recommendations related to new requirements
of the Cures Act and FDARA that pertain to expanded access. FDA
considered comments received on the revised draft guidance as this
guidance was finalized. Changes from the draft to the final guidance
include additional clarifications related to informed consent and the
process for submission of expanded access requests. In addition, we
made editorial changes to improve clarity. FDA considered the
applicability of Executive Order 14192, per OMB guidance in M-25-20,
and finds this action to be deregulatory in nature.
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 ``Expanded Access to Investigational Drugs
for Treatment Use: Questions and Answers.'' It does
[[Page 51371]]
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 part 312 (21 CFR part 312) and Form FDA 1571 have been approved
under OMB control number 0910-0014. The collections of information in
Sec. Sec. 312.300 through 312.320 and Form FDA 3926 have been approved
under OMB control number 0910-0814. The collections of information in
21 CFR part 201 have been approved under OMB control number 0910-0342.
The collections of information in 42 CFR part 11 have been approved
under OMB control number 0925-0586. The collections of information in
21 CFR part 11 have been approved under OMB control number 0910-0303.
The collections of information in 21 CFR parts 50 and 56 have been
approved under OMB control number 0910-0130. The collections of
information pertaining to expedited programs for serious conditions for
drugs have been approved under OMB control number 0910-0765.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
<a href="https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</a>, <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>,
<a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2025-19984 Filed 11-14-25; 8:45 am]
BILLING CODE 4164-01-P
</pre></body>
</html>Indexed from Federal Register on November 17, 2025.
This is legal information, not legal advice. Laws vary by jurisdiction and change frequently. Always verify current law with official sources and consult a licensed attorney in your jurisdiction for advice on your specific situation.