81 FR 35778 - Expanded Access to Investigational Drugs for Treatment Use-Questions and Answers; Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 81, Issue 107 (June 3, 2016)

Page Range		35778-35779
FR Document		2016-13165

The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry entitled ``Expanded Access to Investigational Drugs for Treatment Use--Questions and Answers.'' The guidance provides information for industry, researchers, physicians, institutional review boards (IRBs), and patients about the implementation of FDA's regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (IND). FDA received a number of questions concerning implementation of its expanded access regulations and is providing guidance in a question and answer format to address the most frequently asked questions. This guidance finalizes the draft guidance issued in May 2013.

Federal Register, Volume 81 Issue 107 (Friday, June 3, 2016)

[Federal Register Volume 81, Number 107 (Friday, June 3, 2016)]
[Notices]
[Pages 35778-35779]
From the Federal Register Online [www.thefederalregister.org]
[FR Doc No: 2016-13165]

[[Page 35778]]

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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; 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 guidance for industry entitled ``Expanded Access
to Investigational Drugs for Treatment Use--Questions and Answers.''
The guidance provides information for industry, researchers,
physicians, institutional review boards (IRBs), and patients about the
implementation of FDA's regulations on expanded access to
investigational drugs for treatment use under an investigational new
drug application (IND). FDA received a number of questions concerning
implementation of its expanded access regulations and is providing
guidance in a question and answer format to address the most frequently
asked questions. This guidance finalizes the draft guidance issued in
May 2013.

DATES: Submit either electronic or written comments on Agency guidances
at any time.

ADDRESSES: You may submit comments as follows:

Electronic Submissions

Submit electronic comments in the following way:
Federal eRulemaking Portal: http://www.regulations.gov.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to http://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 http://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): Division of Dockets Management (HFA-305), Food and Drug
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
For written/paper comments submitted to the Division of
Dockets Management, 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; Guidance for Industry;
Availability.'' Received comments will be placed in the docket and,
except for those submitted as ``Confidential Submissions,'' publicly
viewable at http://www.regulations.gov or at the Division of Dockets
Management between 9 a.m. and 4 p.m., Monday through Friday.
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 http://www.regulations.gov.
Submit both copies to the Division of Dockets Management. 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: http://www.fda.gov/regulatoryinformation/dockets/default.htm.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to http://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 Division of Dockets Management, 5630 Fishers
Lane, Rm. 1061, Rockville, MD 20852.
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 or to 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: Ebla Ali Ibrahim, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 6302, Silver Spring, MD 20993, 301-796-
3691; 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 guidance for industry
entitled ``Expanded Access to Investigational Drugs for Treatment Use--
Questions and Answers.'' FDA's expanded access regulations (21 CFR part
312, subpart I) went into effect on October 13, 2009 (74 FR 40900).
Expanded access refers to the use of an investigational drug when the
primary purpose is to diagnose, monitor, or treat a patient rather than
to obtain the kind of information about the drug that is generally
derived from clinical trials. Under the regulations, there are three
categories of expanded access: (1) Expanded access for individual
patients, including for emergency use; (2) expanded access for
intermediate-size patient populations (generally smaller than those
typical of a treatment IND or treatment protocol--a treatment protocol
is submitted as a

[[Page 35779]]

protocol amendment to an existing IND by the sponsor of the existing
IND); and (3) expanded access for widespread treatment use through a
treatment IND or treatment protocol (designed for use in larger patient
populations). The regulations are intended to facilitate, when
appropriate, the availability of investigational new drugs outside of a
clinical investigation or approved drugs where availability is limited
by a risk evaluation and mitigation strategy (REMS) to patients with
serious or immediately life-threatening diseases or conditions who lack
other therapeutic options.
Elsewhere in this issue of the Federal Register, FDA is announcing
the availability of a guidance for industry entitled ``Charging for
Investigational Drugs Under an IND--Questions and Answers,'' which
provides information about the implementation of FDA's regulation on
charging for investigational drugs under an IND, including
investigational drugs made available for expanded access use. (FDA's
guidance documents are available at http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. FDA has
verified the Web site addresses throughout this document, as of the
date this document publishes in the Federal Register, but Web sites are
subject to change over time.)
Additionally, in this issue of the Federal Register, FDA is
announcing the availability of a guidance for industry entitled
``Individual Patient Expanded Access Applications: Form FDA 3926.'' The
guidance describes Form FDA 3926 (Individual Patient Expanded Access--
Investigational New Drug Application (IND)), which is available for
licensed physicians to use for expanded access requests for individual
patient INDs as a streamlined alternative to Form FDA 1571
(Investigational New Drug Application (IND)), and describes the process
for submitting expanded access requests for individual patient expanded
access INDs.
One of FDA's major goals in promulgating the expanded access
regulations was to make expanded access a more transparent process by
increasing awareness and knowledge about expanded access and the
procedures for obtaining investigational drugs for treatment use. Since
the expanded access regulations went into effect in 2009, FDA has
received a number of questions concerning implementation of the
regulations. Consistent with the goal of making expanded access
processes more transparent, FDA is providing guidance in a question and
answer format to address questions about how FDA is implementing its
expanded access regulations, including questions about when it is
appropriate to request expanded access under each of the three expanded
access categories, the types and content of expanded access
submissions, IRB review of individual patient expanded access, and the
onset and duration of expanded access use.
In the Federal Register of May 9, 2013 (78 FR 27115), FDA announced
the availability of the draft guidance entitled ``Expanded Access to
Investigational Drugs for Treatment Use--Questions & Answers.'' FDA
received several comments on the draft guidance, and those comments
were considered as the guidance was finalized. Based on public
comments, in addition to editorial changes made primarily for
clarification, the final guidance includes significant clarification on
the types of expanded access and when each type should be used.
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. 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

This guidance refers to previously approved collections of
information found in FDA regulations. These collections of information
are subject to review by the Office of Management and Budget (OMB)
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The
collections of information in 21 CFR part 312 have been approved under
OMB control number 0910-0014, and the collection of information
resulting from the submission of Form FDA 3926 has been approved under
OMB control number 0910-0814.

III. Electronic Access

Persons with access to the Internet may obtain the document at
http://www.fda.gov/RegulatoryInformation/Guidances/default.htm, http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

Dated: May 31, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-13165 Filed 6-2-16; 8:45 am]
BILLING CODE 4164-01-P

Federal Register / Vol. 81, No. 107 / Friday, June 3, 2016 / Notices 35779

protocol amendment to an existing IND in a question and answer format to Dated: May 31, 2016.
by the sponsor of the existing IND); and address questions about how FDA is Leslie Kux,
(3) expanded access for widespread implementing its expanded access Associate Commissioner for Policy.
treatment use through a treatment IND regulations, including questions about [FR Doc. 2016–13165 Filed 6–2–16; 8:45 am]
or treatment protocol (designed for use when it is appropriate to request BILLING CODE 4164–01–P
in larger patient populations). The expanded access under each of the three
regulations are intended to facilitate, expanded access categories, the types
when appropriate, the availability of and content of expanded access DEPARTMENT OF HEALTH AND
investigational new drugs outside of a submissions, IRB review of individual HUMAN SERVICES
clinical investigation or approved drugs
patient expanded access, and the onset
where availability is limited by a risk Food and Drug Administration
and duration of expanded access use.
evaluation and mitigation strategy [Docket No. FDA–2013–D–0447]
(REMS) to patients with serious or In the Federal Register of May 9, 2013
immediately life-threatening diseases or (78 FR 27115), FDA announced the Charging for Investigational Drugs
conditions who lack other therapeutic availability of the draft guidance Under an Investigational New Drug
options. entitled ‘‘Expanded Access to Application—Questions and Answers;
Elsewhere in this issue of the Federal Investigational Drugs for Treatment Guidance for Industry; Availability
Register, FDA is announcing the Use—Questions & Answers.’’ FDA
availability of a guidance for industry received several comments on the draft AGENCY: Food and Drug Administration,
entitled ‘‘Charging for Investigational HHS.
guidance, and those comments were
Drugs Under an IND—Questions and considered as the guidance was ACTION: Notice of availability.
Answers,’’ which provides information finalized. Based on public comments, in
about the implementation of FDA’s SUMMARY: The Food and Drug
addition to editorial changes made Administration (FDA or Agency) is
regulation on charging for
primarily for clarification, the final announcing the availability of a
investigational drugs under an IND,
guidance includes significant guidance for industry entitled ‘‘Charging
including investigational drugs made
available for expanded access use. clarification on the types of expanded for Investigational Drugs Under an
(FDA’s guidance documents are access and when each type should be IND—Questions and Answers.’’ The
available at http://www.fda.gov/Drugs/ used. guidance provides information for
GuidanceComplianceRegulatory This guidance is being issued industry, researchers, physicians,
Information/Guidances/default.htm. consistent with FDA’s good guidance institutional review boards (IRBs), and
FDA has verified the Web site addresses patients about the implementation of
practices regulation (21 CFR 10.115).
throughout this document, as of the date FDA’s regulation on charging for
The guidance represents the current
this document publishes in the Federal investigational drugs under an
thinking of FDA on expanded access to
Register, but Web sites are subject to investigational new drug application
investigational drugs for treatment use. (IND) for the purpose of either clinical
change over time.) It does not establish any rights for any
Additionally, in this issue of the trials or expanded access for treatment
person and is not binding on FDA or the use. FDA received a number of
Federal Register, FDA is announcing public. You can use an alternative
the availability of a guidance for questions concerning its
approach if it satisfies the requirements implementation of the charging
industry entitled ‘‘Individual Patient
of the applicable statutes and regulation. FDA is providing guidance
Expanded Access Applications: Form
regulations. in a question and answer format to
FDA 3926.’’ The guidance describes
Form FDA 3926 (Individual Patient II. Paperwork Reduction Act of 1995 address the most frequently asked
Expanded Access—Investigational New questions about charging for
Drug Application (IND)), which is This guidance refers to previously investigational drugs under an IND.
available for licensed physicians to use approved collections of information This guidance finalizes the draft
for expanded access requests for found in FDA regulations. These guidance issued in May 2013.
individual patient INDs as a streamlined collections of information are subject to DATES: Submit either electronic or
alternative to Form FDA 1571 review by the Office of Management and written comments on Agency guidances
(Investigational New Drug Application Budget (OMB) under the Paperwork at any time.
(IND)), and describes the process for Reduction Act of 1995 (44 U.S.C. 3501– ADDRESSES: You may submit comments
submitting expanded access requests for 3520). The collections of information in as follows:
individual patient expanded access 21 CFR part 312 have been approved
INDs. Electronic Submissions
under OMB control number 0910–0014,
One of FDA’s major goals in and the collection of information Submit electronic comments in the
promulgating the expanded access resulting from the submission of Form following way:
regulations was to make expanded FDA 3926 has been approved under • Federal eRulemaking Portal: http://
access a more transparent process by www.regulations.gov. Follow the
OMB control number 0910–0814.
increasing awareness and knowledge instructions for submitting comments.
about expanded access and the III. Electronic Access Comments submitted electronically,
procedures for obtaining investigational including attachments, to http://
drugs for treatment use. Since the Persons with access to the Internet www.regulations.gov will be posted to
mstockstill on DSK3G9T082PROD with NOTICES

expanded access regulations went into may obtain the document at http:// the docket unchanged. Because your
effect in 2009, FDA has received a www.fda.gov/RegulatoryInformation/ comment will be made public, you are
number of questions concerning Guidances/default.htm, http:// solely responsible for ensuring that your
implementation of the regulations. www.fda.gov/BiologicsBloodVaccines/ comment does not include any
Consistent with the goal of making GuidanceComplianceRegulatory confidential information that you or a
expanded access processes more Information/Guidances/default.htm or third party may not wish to be posted,
transparent, FDA is providing guidance http://www.regulations.gov. such as medical information, your or

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Document Created: 2018-02-08 07:29:56
Document Modified: 2018-02-08 07:29:56

Category		Regulatory Information
Collection		Federal Register
sudoc Class		AE 2.7: GS 4.107: AE 2.106:
Publisher		Office of the Federal Register, National Archives and Records Administration
Section		Notices
Action		Notice of availability.
Dates		Submit either electronic or written comments on Agency guidances at any time.
Contact		Ebla Ali Ibrahim, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6302, Silver Spring, MD 20993, 301-796- 3691; 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.
FR Citation		81 FR 35778

81 FR 35778 - Expanded Access to Investigational Drugs for Treatment Use-Questions and Answers; Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 81, Issue 107 (June 3, 2016)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration