81 FR 26803 - Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 81, Issue 86 (May 4, 2016)

The Food and Drug Administration (FDA) is announcing the availability of grant funds for the support of FDA's Office of Orphan Products Development (OOPD) Natural History Grants Program. The goal of the Orphan Products Natural History Grants Program is to support studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics. The ultimate goal of these natural history studies is to support 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 natural history studies that will either assist or substantially contribute to market approval of these products. Applicants must include in the application's Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the United States per year), and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.

[Federal Register Volume 81, Number 86 (Wednesday, May 4, 2016)]
[Notices]
[Pages 26803-26804]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2016-10398]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2016-N-0012]


Natural History Studies for Rare Disease Product Development: 
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 (OOPD) Natural History Grants Program. The goal of 
the Orphan Products Natural History Grants Program is to support 
studies that advance rare disease medical product development through 
characterization of the natural history of rare diseases/conditions, 
identification of genotypic and phenotypic subpopulations, and 
development and/or validation of clinical outcome measures, biomarkers 
and/or companion diagnostics. The ultimate goal of these natural 
history studies is to support 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 natural history studies that will either assist or 
substantially contribute to market approval of these products. 
Applicants must include in the application's Background and 
Significance section documentation to support that the estimated 
prevalence of the orphan disease or condition in the United States is 
less than 200,000 (or in the case of a vaccine or diagnostic, 
information to support that the product will be administered to fewer 
than 200,000 people in the United States per year), 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 October 14, 2016 and October 15, 
2018.
    2. The anticipated start dates are March 2017 and March 2019.
    3. The opening dates are August 15, 2016 and August 15, 2018.
    4. The expiration date is October 16, 2018.

ADDRESSES: Submit electronic applications to: http://www.grants.gov. 
For more information, see section III of the SUPPLEMENTARY INFORMATION 
section of this notice.

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. 5295, Silver 
Spring, MD 20993-0002, 301-796-8660, email: 
[email protected]; or Daniel Lukash, Office of 
Acquisitions and Grant Services, 5630 Fishers Lane, Rockville, MD 
20857, 240-402-7596, email: [email protected].
    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/orphan.

[[Page 26804]]


SUPPLEMENTARY INFORMATION:

I. Funding Opportunity Description

RFA-FD-16-043

93.103

A. Background

    The OOPD 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 OOPD 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.
    The natural history of a disease is the natural course of a disease 
from the time immediately prior to its inception, progressing through 
its pre-symptomatic phase and different clinical stages to the point 
where the disease has ended without external intervention. Natural 
history studies track the course of disease over time, identifying 
demographic, genetic, environmental, and other variables that correlate 
with its development and outcomes in the absence of treatment. Thorough 
understanding of disease natural history is the foundation upon which a 
clinical development program for drugs, biologics, medical foods or 
medical devices is built.
    Rare diseases, as defined in the United States Orphan Drug Act 
(ODA) (Pub. L. 97-414), are diseases or conditions with a prevalence of 
fewer than 200,000 persons in the United States. Though individually 
rare, together there are approximately 30 million Americans affected by 
7,000 known rare diseases. Unlike common diseases, there is little 
existing knowledge on the natural history of most rare diseases, which 
makes natural history studies of particular importance for rare 
diseases product development. In January 2014, the FDA organized a 
Public Workshop on Complex Issues in Developing Drugs for Rare 
Diseases. During the workshop, the lack of natural history studies was 
reconfirmed by all stakeholders (patients, industry, researchers and 
the FDA) as one of the most common and urgent issues that hinder 
treatment development for rare diseases. The need for natural history 
studies was also emphasized in the recently published (August 17, 2015) 
draft FDA Guidance for Industry, ``Rare Diseases: Common Issues in Drug 
Development,'' available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf.

B. Research Objectives

    The objective of FDA's Orphan Products Natural History Grants 
Program is to support studies that characterize the natural history of 
rare diseases/conditions, identify genotypic and phenotypic 
subpopulations, and develop and/or validate clinical outcome measures, 
biomarkers and/or companion diagnostics. The ultimate goal of these 
natural history studies is to support clinical development of products 
for use in serious 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 natural history studies that will 
either assist or substantially contribute to market approval of these 
products. Applicants must include in the application's Background and 
Significance section documentation to support that the estimated 
prevalence of the orphan disease or condition in the United States is 
less than 200,000 (or in the case of a vaccine or diagnostic, 
information to support that the product will be administered to fewer 
than 200,000 people in the United States per year), 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 may not apply.

II. Award Information/Funds Available

A. Award Amount

    Of the estimated FY 2017 funding ($17.7 million), approximately $2 
million will fund 2 to 5 new awards, subject to availability of funds. 
Prospective Natural History Studies are eligible for grants of up to 
$400,000 per year for up to 5 years. Retrospective Natural History 
Studies or Surveys are eligible for grants of up to $150,000 per year 
for up to 2 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 $150,000 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, all future 
years of noncompetitive continuation of support will depend on the 
following factors: (1) Performance during the preceding year; (2) 
compliance with regulatory requirements as applicable; 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 System for Award Management (SAM) 
(formerly Central Contractor Registration (CCR))
 Step 3: Obtain Username & Password on Grants.gov
 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: April 28, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-10398 Filed 5-3-16; 8:45 am]
 BILLING CODE 4164-01-P