[Federal Register Volume 78, Number 16 (Thursday, January 24, 2013)]
[Notices]
[Pages 5185-5186]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-01420]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2011-D-0847]
Guidance for Industry and Food and Drug Administration Staff;
Humanitarian Use Device (HUD) Designations; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing the
availability of a guidance for the industry and FDA staff entitled
``Humanitarian Use Device (HUD) Designations.'' Devices are eligible
for HUD designation if they are designed to treat or diagnose a disease
or condition that affects or is manifested in fewer than 4,000
individuals in the United States per year. Devices that receive HUD
designations may be eligible for marketing approval under the
Humanitarian Device Exemption (HDE) marketing pathway. This guidance
document is intended to assist applicants in the preparation and
submission of HUD designation requests and FDA reviewers in evaluating
such requests. This guidance finalizes the draft guidance of the same
title dated December 2011.
DATES: Submit either electronic or written comments on Agency guidances
at any time.
ADDRESSES: Submit written requests for single copies of the guidance to
the Office of Orphan Products (OOPD), Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 32, Rm. 5271, Silver Spring, MD 20993-
0002. Send one self-addressed adhesive label to assist the office in
processing your requests. The guidance may also be obtained by mail by
calling OOPD at 301-796-8660. See the SUPPLEMENTARY INFORMATION section
for electronic access to the guidance document.
Submit electronic comments on the 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: Eric Chen, Office of Orphan Products
Development (OOPD), Food and Drug Administration, Bldg. 32, Rm. 5222,
10903 New Hampshire Ave., Silver Spring, MD 20993, 301-796-6327, email:
[email protected].
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry and
FDA staff entitled ``Humanitarian Use Device (HUD) Designations.''
Devices are eligible for HUD designation if they are designed to treat
or diagnose a disease or condition that affects or is manifested in
fewer than 4,000 individuals in the United States per year. (See
section 520(m) of the Federal Food, Drug, and Cosmetic Act (FD&C Act),
21 U.S.C. 360j(m); 21 CFR 814.102.) This guidance document is intended
to assist applicants in the preparation and submission of HUD
designation requests to OOPD. This guidance is also intended to assist
FDA reviewers in the evaluation and analysis of HUD designation
requests.
Topics addressed in this guidance include: (1) Demonstrating in HUD
designation requests that the device is designed to treat or diagnose a
disease or condition that affects or is manifested in fewer than 4,000
individuals in the United States per year; (2) how this demonstration
varies depending on whether the device is intended for therapeutic or
diagnostic purposes; (3)
[[Page 5186]]
how properties of the device may affect this demonstration; and (4) for
the purpose of a HUD designation request, delineating a medically
plausible subset (``orphan subset'') of persons with a given disease or
condition that affects or is manifested in 4,000 individuals or more in
the United States per year.
Devices that receive HUD designation may be eligible for marketing
approval under an HDE application. An HDE application is a premarketing
application that is similar to a premarket approval (PMA) application
in that the applicant must demonstrate a reasonable assurance of
safety, but in an HDE application, the applicant seeks an exemption
from the PMA requirement of demonstrating a reasonable assurance of
effectiveness. A device that has received HUD designation is eligible
for HDE approval if, among other criteria, the device will not expose
patients to an unreasonable or significant risk of illness or injury
and the probable benefit to health from use of the device outweighs the
risk of injury or illness from its use, taking into account the
probable risks and benefits of currently available devices or
alternative forms of treatment. (See section 520(m)(2)(C) of the FD&C
Act; 21 CFR 814.104(b)(2).) Although a HUD designation from OOPD is a
prerequisite to submitting an HDE application to the Center for Devices
and Radiological Health or the Center for Biologics Evaluation and
Research, it does not by itself guarantee approval of the HDE
application.
In the Federal Register of December 13, 2011 (76 FR 77542), FDA
issued for public comment ``Draft Guidance for Industry and Food and
Drug Administration Staff on Humanitarian Use Devices Designations''
dated December 2011. The Agency issued this draft guidance with the aim
of assisting sponsors in the preparation and submission of HUD
designation requests by, among other things, providing clarity on
particular elements of HUD designation requests that had historically
caused confusion among sponsors. In particular, the draft guidance
focused on the disease or condition that the device treats or
diagnoses, population estimates, orphan subsets, device descriptions,
scientific rationales, and supporting documentation.
We received several comments on the draft guidance. Most comments
appreciated the clarification and explanation provided by the draft
guidance. Several comments made recommendations to improve clarity.
FDA is issuing the draft guidance in final form with minor
revisions to improve clarity. This guidance is being issued consistent
with FDA's good guidance practices regulation (21 CFR 10.115). The
guidance represents the Agency's current thinking on HUD designation
requests. 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. Comments
Interested persons may submit either written comments regarding
this document to the Division of Dockets Management (see ADDRESSES) or
electronic comments to http://www.regulations.gov. 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.
III. Electronic Access
Persons with access to the Internet may obtain this guidance
document at either: http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/default.htm, http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm, or http://www.regulations.gov.
Dated: January 18, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-01420 Filed 1-23-13; 8:45 am]
BILLING CODE 4160-01-P