[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]


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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.

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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 This guidance finalizes the draft guidance of the same 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]
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