Document ID: FDA-2010-N-0394-0001
Agency: fda
Document Type: Notice
Title: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant
Posted Date: 2010-08-06T04:00Z

[Federal Register: August 6, 2010 (Volume 75, Number 151)]
[Notices]               
[Page 47602-47603]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr06au10-83]                         

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2010-N-0394]

 
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 estimated prevalence of the orphan disease or condition (or 
in the case of a vaccine or diagnostic, information to support the 
estimates of how many people will be administered the diagnostic or 
vaccine annually) 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 2, 2011; February 1, 
2012. The resubmission due dates are October 14, 2011; October 15, 
2012.
    2. The anticipated start dates are November 2010; November 2012.
    3. The opening date is December 2, 2010.
    4. The expiration date is February 2, 2012; October 16, 2012 
(resubmission).

FOR FURTHER INFORMATION AND ADDITIONAL REQUIREMENTS CONTACT:
    Katherine Needleman, Orphan Products Grants Program, Office of 
Orphan Products Development, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 32, rm. 5271, Silver Spring, MD 20993-0002, 301-
796-8660, e-mail: katherine.needleman@fda.hhs.gov.
    Vieda Hubbard, Division of Acquisition Support and Grants, Office 
of Acquisitions & Grant Services, 5630 Fishers Lane, Rockville, MD 
20857, 301-827-7177, e-mail: 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.\1\
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    \1\ FDA has verified the Web site addresses throughout this 
document, but FDA is not responsible for any subsequent changes to 
the Web site after this document publishes in the Federal Register.)

SUPPLEMENTARY INFORMATION:

I. Funding Opportunity Description

RFA-FD-11-001
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 
(that is, one with prevalence, not incidence, of fewer than 200,000 
people in the United States). Diagnostics and vaccines will qualify for 
orphan status only if the U.S. population to whom they will be 
administered is fewer than 200,000 people 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

[[Page 47603]]

products. Applicants must include in the application's Background and 
Significance section documentation to support the estimated prevalence 
of the orphan disease or condition (or in the case of a vaccine or 
diagnostic, information to support the estimates of how many people 
will be administered the diagnostic or vaccine annually) 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 fiscal year (FY) 2012 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 2013 will be 
similar to FY 2012. 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 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 and 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 30, 2010.
Leslie Kux,
Acting Assistant Commissioner for Policy.
[FR Doc. 2010-19354 Filed 8-5-10; 8:45 am]
BILLING CODE 4160-01-S