81 FR 35776 - Individual Patient Expanded Access Applications: Form FDA 3926; 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		35776-35777
FR Document		2016-13167

The Food and Drug Administration (FDA or Agency) 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. Individual patient expanded access allows for the use of an investigational new drug outside of a clinical investigation, or the use of an approved drug where availability is limited by a risk evaluation and mitigation strategy (REMS), for an individual patient who has a serious or immediately life-threatening disease or condition when there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition. Form FDA 3926 provides a streamlined alternative for submitting an IND for use in cases of individual patient expanded access, including for emergency use. This guidance finalizes the draft guidance issued in February 2015.

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

[Federal Register Volume 81, Number 107 (Friday, June 3, 2016)]
[Notices]
[Pages 35776-35777]
From the Federal Register Online [www.thefederalregister.org]
[FR Doc No: 2016-13167]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-0268]

Individual Patient Expanded Access Applications: Form FDA 3926;
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 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. Individual patient expanded access allows for the use of
an investigational new drug outside of a clinical investigation, or the
use of an approved drug where availability is limited by a risk
evaluation and mitigation strategy (REMS), for an individual patient
who has a serious or immediately life-threatening disease or condition
when there is no comparable or satisfactory alternative therapy to
diagnose, monitor, or treat the disease or condition. Form FDA 3926
provides a streamlined alternative for submitting an IND for use in
cases of individual patient expanded access, including for emergency
use. This guidance finalizes the draft guidance issued in February
2015.

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-2015-D-0268 for ``Individual Patient Expanded Access Applications:
Form FDA 3926; 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: Larry Lim, Center for Drug Evaluation
and Research, Food and Drug Administration, 10001 New Hampshire Ave.,
Hillandale Bldg., Rm. 4134, Silver Spring, MD 20993, 301-796-3146; 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

[[Page 35777]]

``Individual Patient Expanded Access Applications: Form FDA 3926.'' The
guidance describes Form FDA 3926, which is available for licensed
physicians to use for expanded access requests for individual patient
INDs. FDA's current 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 protocol amendment to an existing IND by the sponsor of the
existing IND); and (3) expanded access for widespread treatment use
through a treatment protocol or treatment IND (designed for use in
larger patient populations). The regulations are intended to facilitate
the availability of investigational new drugs outside of a clinical
investigation, or approved drugs where availability is limited by a
REMS, to patients with serious or immediately life-threatening diseases
or conditions when there is no comparable or satisfactory alternative
therapy to diagnose, monitor, or treat the disease or condition.
Elsewhere in this issue of the Federal Register, FDA is announcing
the availability of a guidance for industry entitled ``Expanded Access
to Investigational Drugs for Treatment Use--Questions and Answers,''
which provides answers to questions concerning the implementation of
FDA's regulations on expanded access to investigational drugs for
treatment use (21 CFR part 312, subpart I). (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
``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 may permit expanded access to an investigational new drug
outside of a clinical investigation, or to an approved drug where
availability is limited by a REMS, for an individual patient when the
applicable criteria in Sec. 312.305(a) (which apply to all types of
expanded access) and in Sec. 312.310(a) (which apply specifically to
individual patient expanded access, including for emergency use) are
met. In addition, Sec. 312.305(b) sets forth the submission
requirements for all types of expanded access use requests. One of the
requirements under Sec. 312.305(b)(2) is that a ``cover sheet'' must
be included ``meeting the requirements of Sec. 312.23(a).'' This
provision applies to several types of submissions under part 312,
ranging from commercial INDs under Sec. 312.23 that involve large
groups of patients enrolled in clinical trials to requests from
physicians to use an investigational drug for an individual patient.
Form FDA 1571 is currently used by sponsors for all types of IND
submissions. However, FDA is concerned that physicians requesting
expanded access for an individual patient may have encountered
difficulty in completing Form FDA 1571 and providing the associated
documents because Form FDA 1571 is not tailored to requests for
individual patient expanded access.
To streamline the submission process for individual patient
expanded access INDs, FDA developed Form FDA 3926, which is available
for licensed physicians to use to request expanded access to an
investigational drug outside of a clinical investigation, or to an
approved drug where availability is limited by a REMS, for an
individual patient who has a serious or immediately life-threatening
disease or condition when there is no comparable or satisfactory
alternative therapy to diagnose, monitor, or treat the disease or
condition.
In an emergency situation that requires the patient to be treated
before a written submission can be made, the request to use the
investigational drug for individual patient expanded access may be made
by telephone (or other rapid means of communication) to the appropriate
FDA review division. Authorization of the emergency use may be given by
an FDA official by telephone, provided the physician explains how the
expanded access use will meet the requirements of Sec. Sec. 312.305
and 312.310 and agrees to submit an expanded access request within 15
working days of FDA's initial authorization of the expanded access use
(Sec. 312.310(d)). The physician may choose to use Form FDA 3926 for
the expanded access application.
In the Federal Register of February 10, 2015 (80 FR 7318), FDA
announced the availability of the draft guidance. FDA received several
comments on the draft guidance and those comments were considered as
the guidance was finalized. Both the guidance and Form FDA 3926 were
revised based on public comments and editorial changes were made
primarily for clarification. One notable change includes the ability to
use Form FDA 3926 for subsequent submissions to an existing individual
patient expanded access IND.
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 the use of Form FDA 3926 by licensed
physicians to submit requests for individual patient expanded access
INDs. 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 contains information collection provisions that 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
collection of information in this guidance was approved under OMB
control number 0910-0814.
This guidance also refers to previously approved collections of
information found in FDA regulations. The collections of information in
21 CFR part 312 have been approved under OMB control number 0910-0014.

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. 2016-13167 Filed 6-2-16; 8:45 am]
BILLING CODE 4164-01-P

Federal Register / Vol. 81, No. 107 / Friday, June 3, 2016 / Notices 35777

‘‘Individual Patient Expanded Access an IND, including investigational drugs use (§ 312.310(d)). The physician may
Applications: Form FDA 3926.’’ The made available for expanded access use. choose to use Form FDA 3926 for the
guidance describes Form FDA 3926, FDA may permit expanded access to expanded access application.
which is available for licensed an investigational new drug outside of
In the Federal Register of February
physicians to use for expanded access a clinical investigation, or to an
approved drug where availability is 10, 2015 (80 FR 7318), FDA announced
requests for individual patient INDs.
limited by a REMS, for an individual the availability of the draft guidance.
FDA’s current expanded access
regulations (21 CFR part 312, subpart I) patient when the applicable criteria in FDA received several comments on the
went into effect on October 13, 2009 (74 § 312.305(a) (which apply to all types of draft guidance and those comments
FR 40900). Expanded access refers to expanded access) and in § 312.310(a) were considered as the guidance was
the use of an investigational drug when (which apply specifically to individual finalized. Both the guidance and Form
the primary purpose is to diagnose, patient expanded access, including for FDA 3926 were revised based on public
monitor, or treat a patient rather than to emergency use) are met. In addition, comments and editorial changes were
obtain the kind of information about the § 312.305(b) sets forth the submission made primarily for clarification. One
drug that is generally derived from requirements for all types of expanded notable change includes the ability to
clinical trials. Under the regulations, access use requests. One of the use Form FDA 3926 for subsequent
there are three categories of expanded requirements under § 312.305(b)(2) is submissions to an existing individual
access: (1) Expanded access for that a ‘‘cover sheet’’ must be included patient expanded access IND.
individual patients, including for ‘‘meeting the requirements of
This guidance is being issued
emergency use; (2) expanded access for § 312.23(a).’’ This provision applies to
several types of submissions under part consistent with FDA’s good guidance
intermediate-size patient populations
312, ranging from commercial INDs practices regulation (21 CFR 10.115).
(generally smaller than those typical of
a treatment IND or treatment protocol— under § 312.23 that involve large groups The guidance represents the current
a treatment protocol is submitted as a of patients enrolled in clinical trials to thinking of FDA on the use of Form
protocol amendment to an existing IND requests from physicians to use an FDA 3926 by licensed physicians to
by the sponsor of the existing IND); and investigational drug for an individual submit requests for individual patient
(3) expanded access for widespread patient. Form FDA 1571 is currently expanded access INDs. It does not
treatment use through a treatment used by sponsors for all types of IND establish any rights for any person and
protocol or treatment IND (designed for submissions. However, FDA is is not binding on FDA or the public.
use in larger patient populations). The concerned that physicians requesting You can use an alternative approach if
regulations are intended to facilitate the expanded access for an individual it satisfies the requirements of the
availability of investigational new drugs patient may have encountered difficulty applicable statutes and regulations.
outside of a clinical investigation, or in completing Form FDA 1571 and
approved drugs where availability is providing the associated documents II. Paperwork Reduction Act of 1995
limited by a REMS, to patients with because Form FDA 1571 is not tailored This guidance contains information
serious or immediately life-threatening to requests for individual patient
collection provisions that are subject to
diseases or conditions when there is no expanded access.
To streamline the submission process review by the Office of Management and
comparable or satisfactory alternative
for individual patient expanded access Budget (OMB) under the Paperwork
therapy to diagnose, monitor, or treat
the disease or condition. INDs, FDA developed Form FDA 3926, Reduction Act of 1995 (44 U.S.C. 3501–
Elsewhere in this issue of the Federal which is available for licensed 3520). The collection of information in
Register, FDA is announcing the physicians to use to request expanded this guidance was approved under OMB
availability of a guidance for industry access to an investigational drug outside control number 0910–0814.
entitled ‘‘Expanded Access to of a clinical investigation, or to an This guidance also refers to
Investigational Drugs for Treatment approved drug where availability is previously approved collections of
Use—Questions and Answers,’’ which limited by a REMS, for an individual information found in FDA regulations.
provides answers to questions patient who has a serious or The collections of information in 21
concerning the implementation of immediately life-threatening disease or CFR part 312 have been approved under
FDA’s regulations on expanded access condition when there is no comparable
OMB control number 0910–0014.
to investigational drugs for treatment or satisfactory alternative therapy to
use (21 CFR part 312, subpart I). (FDA’s diagnose, monitor, or treat the disease or III. Electronic Access
guidance documents are available at condition.
http://www.fda.gov/Drugs/Guidance In an emergency situation that Persons with access to the Internet
ComplianceRegulatoryInformation/ requires the patient to be treated before may obtain the document at http://www.
Guidances/default.htm. FDA has a written submission can be made, the fda.gov/RegulatoryInformation/
verified the Web site addresses request to use the investigational drug Guidances/default.htm, http://www.fda.
throughout this document, as of the date for individual patient expanded access gov/BiologicsBloodVaccines/Guidance
this document publishes in the Federal may be made by telephone (or other ComplianceRegulatoryInformation/
Register, but Web sites are subject to rapid means of communication) to the Guidances/default.htm, or http://
change over time.) appropriate FDA review division. www.regulations.gov.
Additionally, in this issue of the Authorization of the emergency use may
Dated: May 31, 2016.
Federal Register, FDA is announcing be given by an FDA official by
mstockstill on DSK3G9T082PROD with NOTICES

the availability of a guidance for telephone, provided the physician Leslie Kux,
industry entitled ‘‘Charging for explains how the expanded access use Associate Commissioner for Policy.
Investigational Drugs Under an IND— will meet the requirements of [FR Doc. 2016–13167 Filed 6–2–16; 8:45 am]
Questions and Answers,’’ which §§ 312.305 and 312.310 and agrees to BILLING CODE 4164–01–P
provides information about the submit an expanded access request
implementation of FDA’s regulation on within 15 working days of FDA’s initial
charging for investigational drugs under authorization of the expanded access

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Document Created: 2018-02-08 07:30:00
Document Modified: 2018-02-08 07:30:00

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		Larry Lim, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Bldg., Rm. 4134, Silver Spring, MD 20993, 301-796-3146; 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 35776

81 FR 35776 - Individual Patient Expanded Access Applications: Form FDA 3926; 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