[Federal Register Volume 81, Number 107 (Friday, June 3, 2016)]
[Notices]
[Pages 35776-35777]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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 This guidance finalizes the draft guidance issued in February 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