Document ID: FDA-2012-N-0011-0003
Agency: fda
Document Type: Notice
Title: Research Project Grants: Clinical Studies of Safety and Effectiveness of Orphan Products
Posted Date: 2012-08-06T04:00Z

[Federal Register Volume 77, Number 151 (Monday, August 6, 2012)]
[Notices]
[Pages 46764-46765]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-19086]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0011]

Clinical Studies of Safety and Effectiveness of Orphan Products 
Research Project Grant (R01)

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of grant funds for the support of FDA's Office of Orphan 
Products Development (OPD) grant program. The goal of FDA's OPD grant 
program is to support the clinical development of products for use in 
rare diseases or conditions where no current therapy exists or where 
the proposed product will be superior to the existing therapy. FDA 
provides grants for clinical studies on safety and/or effectiveness 
that will either result in, or substantially contribute to, market 
approval of these products. Applicants must include in the 
application's Background and Significance section documentation to 
support the assertion that the product to be studied meets the 
statutory criteria to qualify for the grant and an explanation of how 
the proposed study will either help support product approval or provide 
essential data needed for product development.

DATES: Important dates are as follows:
    1. The application due dates are February 6, 2013; February 5, 
2014. The resubmission due dates are October 15, 2013; October 15, 
2014.
    2. The anticipated start dates are November 2013; November 2014.
    3. The opening date is December 6, 2013.
    4. The expiration date is February 6, 2014; October 16, 2014 
(resubmission).
    For Further Information and Additional Requirements Contact: 
Katherine Needleman, Office of Orphan Products Development, Food and 
Drug Administration, 10903 New Hampshire Ave., Bldg. 32, rm. 5271, 
Silver Spring, MD 20993-0002, Phone: 301-796-8660, Email: 
katherine.needleman@fda.hhs.gov; or Vieda Hubbard, Office of 
Acquisitions & Grant Services, 5630 Fishers Lane, rm. 2034, Rockville, 
MD 20857, Phone: 301-827-7177, Email: vieda.hubbard@fda.hhs.gov.
    For more information on this funding opportunity announcement (FOA) 
and to obtain detailed requirements, please refer to the full FOA 
located at http://grants.nih.gov/grants/guide (select the ``Request for 
Applications'' link), http://www.grants.gov (see ``For Applicants'' 
section), and http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/WhomtoContactaboutOrphanProductDevelopment/ucm134580.htm.

SUPPLEMENTARY INFORMATION:

I. Funding Opportunity Description

RFA-FD-13-001
93.103

A. Background

    The OPD was created to identify and promote the development of 
orphan products. Orphan products are drugs, biologics, medical devices, 
and medical foods that are indicated for a rare disease or condition. 
The term ``rare disease or condition'' is defined in section 528 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ee). FDA generally 
considers drugs, devices, and medical foods potentially eligible for 
grants under the OPD grant program if they are indicated for a disease 
or condition that has a prevalence, not incidence, of fewer than 
200,000 people in the United States. Diagnostics and vaccines are 
considered potentially eligible for such grants only if the U.S. 
population to whom they will be administered is fewer than 200,000 
people in the United States per year.

B. Research Objectives

    The goal of FDA's OPD grant program is to support the clinical 
development of products for use in rare diseases or conditions where no 
current therapy exists or where the proposed product will be superior 
to the existing therapy. FDA provides grants for clinical studies on 
safety and/or effectiveness that will either result in, or 
substantially contribute to, market approval of these products. 
Applicants must include in the application's Background and 
Significance section documentation to support the assertion that the 
product to be studied meets the statutory criteria to qualify for the 
grant and an explanation of how the proposed study will either help 
support product approval or provide essential data needed for product 
development.

C. Eligibility Information

    The grants are available to any foreign or domestic, public or 
private, for-profit or nonprofit entity (including State and local 
units of government). Federal Agencies that are not part of the 
Department of Health and Human Services (HHS) may apply. Agencies that 
are part of HHS may not apply. For-profit entities must commit to 
excluding fees or profit in their request for support to receive grant 
awards. Organizations that engage in lobbying activities, as described 
in section 501(c)(4) of the Internal Revenue Code of 1968, are not 
eligible to receive grant awards.

II. Award Information/Funds Available

A. Award Amount

    Of the estimated FY 2014 funding ($14.1 million), approximately $10 
million will fund noncompeting continuation awards, and approximately 
$4.1 million will fund 5 to 10 new awards, subject to availability of 
funds. It is anticipated that funding for the number of noncompeting 
continuation awards and new awards in FY 2015 will be similar to FY 
2014. Phase 1 studies are eligible for grants of up to $200,000 per 
year for up to 3 years. Phase 2 and 3 studies are eligible for grants 
of up to $400,000 per year for up to 4 years. Please note that the 
dollar limitation will apply to total costs (direct plus indirect). 
Budgets for each year of requested support may not exceed the $200,000 
or $400,000 total cost limit, whichever is applicable.

B. Length of Support

    The length of support will depend on the nature of the study. For 
those studies with an expected duration of more than 1 year, a second, 
third, or fourth year of noncompetitive continuation of support will 
depend on the following factors: (1) Performance during the preceding 
year; (2) compliance with regulatory requirements of IND/
investigational device exemption (IDE); and (3) availability of Federal 
funds.

III. Electronic Application, Registration, and Submission

    Only electronic applications will be accepted. To submit an 
electronic application in response to this FOA, applicants should first 
review the full

[[Page 46765]]

announcement located at http://grants.nih.gov/grants/guide. For all 
electronically submitted applications, the following steps are 
required.
     Step 1: Obtain a Dun and Bradstreet (DUNS) Number
     Step 2: Register With Central Contractor Registration
     Step 3: Obtain Username & Password
     Step 4: Authorized Organization Representative (AOR) 
Authorization
     Step 5: Track AOR Status
     Step 6: Register With Electronic Research Administration 
(eRA) Commons
    Steps 1 through 5, in detail, can be found at http://www07.grants.gov/applicants/organization_registration.jsp. Step 6, in 
detail, can be found at https://commons.era.nih.gov/commons/registration/registrationInstructions.jsp. After you have followed 
these steps, submit electronic applications to: http://www.grants.gov.

    Dated: July 31, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-19086 Filed 8-3-12; 8:45 am]
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