[Federal Register Volume 78, Number 90 (Thursday, May 9, 2013)]
[Proposed Rules]
[Pages 27115-27116]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-11005]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

21 CFR Part 312

[Docket No. FDA-2013-D-0446]


Draft Guidance for Industry on Expanded Access to Investigational 
Drugs for Treatment Use--Questions and Answers; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a draft guidance for industry entitled ``Expanded 
Access to Investigational Drugs for Treatment Use--Qs & As.'' This 
guidance is intended to provide information for industry, researchers, 
physicians, and patients about certain aspects of FDA's implementation 
of its regulations on expanded access to investigational drugs for 
treatment use. FDA has received a number of questions about 
implementation of its expanded access regulations. Therefore, FDA is 
providing this draft guidance in a question and answer format, 
addressing the most frequently asked questions.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by July 8, 2013.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 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, 10903 New 
Hampshire Ave., Bldg. 51, rm. 2201, Silver Spring, MD 20993-0002 or 
Office of Communication, Outreach, and Development (HFM-40), Center for 
Biologics Evaluation and Research, Food and Drug Administration, 1401 
Rockville Pike, suite 200N, Rockville, MD 20852-1448. 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access to the draft guidance document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: For the Center for Drug Evaluation and 
Research: Colleen L. Locicero, Center for Drug Evaluation and Research, 
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, rm. 
4200, Silver Spring, MD 20993-0002, 301-796-2270.
    For the Center for Biologics Evaluation and Research: Stephen M. 
Ripley, Center for Biologics Evaluation and Research (HFM-17), Food and 
Drug Administration, 1401 Rockville Pike, Rockville, MD 20852-1448, 
301-827-6210.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Expanded Access to Investigational Drugs for Treatment Use--
Qs & As.'' FDA's expanded access regulations (21 CFR part 312, subpart 
I) went into effect on October 13, 2009 (74 FR 40900).
    These regulations contain the requirements for the use of 
investigational new drugs or approved drugs where availability is 
limited by a risk evaluation and mitigation strategy (REMS), when the 
primary purpose is to diagnose, monitor, or treat a patient's disease 
or condition. Under these regulations, there are three categories of 
expanded access based on the size of the patient population to be 
treated: (1) Individual patient access, including for emergency use; 
(2) intermediate-size patient population access; and (3) larger 
population access under a treatment protocol or treatment 
investigational new drug application (IND). These regulations are 
intended to facilitate the availability of investigational new drugs, 
or approved drugs where availability is limited by a REMS, to patients 
with serious or immediately life-threatening diseases or conditions who 
lack other therapeutic options and may benefit from investigational 
therapies.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of the draft guidance entitled ``Charging for 
Investigational Drugs Under an IND--Qs & As,'' which is intended to 
provide information about FDA's implementation of its regulation on 
charging for investigational drugs under an investigational new drug 
applications, including investigational drugs made available under 
expanded access programs.
    One of FDA's major goals in promulgating these expanded access 
regulations was to make expanded access a more transparent process by 
increasing awareness and knowledge of expanded access programs and the 
procedures for obtaining investigational drugs for treatment use. Since 
these expanded access regulations went into effect in 2009, FDA has 
received a number of questions concerning its implementation of the 
regulations. Consistent with the goal of making expanded access 
processes more transparent, FDA is providing this draft guidance to 
address frequently asked questions about how it is interpreting various 
provisions in the expanded access regulations, including questions 
about when it is appropriate to request access under each of the three 
access categories, the types and content of access submissions, IRB 
review of individual patient expanded access, and the onset and 
duration of access use.
    Although FDA is inviting comment on the entire draft guidance (21 
CFR 10.115(g)(1)(ii)(C)), FDA notes that it is particularly interested 
in receiving comments on question 10. Question 10 asks, ``Is 
Institutional Review Board (IRB) review and approval required for 
individual patient expanded access?'' In the draft guidance, FDA 
explains that under current regulations for all expanded access uses, 
including individual patient access uses, investigators are required to 
ensure that IRB review and approval is obtained consistent with 21 CFR 
part 56 (21 CFR 312.305(c)(4)). 21 CFR part 56 requires, among other 
things, that an IRB review the expanded access use at a convened 
meeting at which a majority of the IRB members are present (``full IRB 
review'') (21 CFR 56.108(c)). However, FDA is aware of concerns that 
this requirement for full IRB review may deter individual patient 
access to investigational drugs for treatment use. FDA has encouraged 
use of central IRBs for review of expanded access uses to address these 
concerns. However, other options may be needed. Therefore, FDA is 
particularly interested in receiving comments on this issue, including 
to what extent the requirement for full IRB review of individual 
patient expanded access is a deterrent to patient access,

[[Page 27116]]

whether FDA should consider alternatives to full IRB review of 
individual patient expanded access, and what alternative approaches may 
better facilitate access while providing appropriate ethical oversight.
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the Agency's current thinking on expanded 
access to investigational drugs for treatment use. It does not create 
or confer any rights for or on any person and does not operate to bind 
FDA or the public. An alternative approach may be used if such approach 
satisfies the requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    This draft 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 Sec. Sec.  312.305, 312.310, 312.315, and 
312.320 have been approved under OMB control number 0910-0014.

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

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

    Dated: May 3, 2013.
Peter Lurie,
Acting Associate Commissioner for Policy and Planning.
[FR Doc. 2013-11005 Filed 5-8-13; 8:45 am]
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