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)

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 This guidance finalizes the draft guidance issued in May 2013.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, 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.

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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 This guidance finalizes the draft guidance issued in 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