Notice2022-23785
Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers; Draft Guidance for Industry; Availability
Primary source
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Published
November 2, 2022
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a revised draft guidance for industry entitled "Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers."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.
Full Text
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<title>Federal Register, Volume 87 Issue 211 (Wednesday, November 2, 2022)</title>
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[Federal Register Volume 87, Number 211 (Wednesday, November 2, 2022)]
[Notices]
[Pages 66191-66192]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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Questions and Answers; Draft Guidance for Industry; Availability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the availability of a revised draft guidance for industry entitled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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the 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 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 <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>.
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
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