[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Draft Guidance for Industry on Expanded Access to Investigational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availability of a draft guidance for industry entitled ``Expanded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providing this draft guidance in a question and answer format,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draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance by July 8, 2013.
ADDRESSES: Submit written requests for single copies of the draft
guidance to the Division of Drug Information, Center for Drug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access to the draft guidance document.
Submit electronic comments on the draft guidance to http://www.regulations.gov.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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