Document ID: FDA-2015-N-0012-0005
Agency: fda
Document Type: Notice
Title: Disease Natural History Database Development—(U24)
Posted Date: 2015-08-17T04:00Z

[Federal Register Volume 80, Number 158 (Monday, August 17, 2015)]
[Notices]
[Pages 49242-49243]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-20130]

-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-N-0012]

Disease Natural History Database Development--(U24)

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of grant funds for the support of Natural History Database 
Development. The National Organization for Rare Disorders (NORD) is 
developing an Internet-based data collection tool with promise to 
further the accumulation of natural history data for many rare 
diseases. The goal of this grant is to enable NORD to further develop, 
refine, and disseminate the database tool.

DATES: Important dates are as follows:
    1. The application due date is September 4, 2015.
    2. The anticipated start date is September 2015.
    3. The opening date is July 2015.
    4. The expiration date is September 5, 2015.

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 CONTACT: James Kaiser, Office of Translational 
Sciences, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993, 301-
796-1237, james.kaiser@fda.hhs.gov.
    Vieda Hubbard, Office of Acquisition and Grants Services, Food and 
Drug Administration, 5630 Fishers Lane, Rockville, MD 20857, 240-402-
7588, 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 www.grants.gov. Search by Funding Opportunity Number: RFA-
FD-15-038.

SUPPLEMENTARY INFORMATION:

I. Funding Opportunity Description

RFA-FD-15-038
93.103

A. Background

    There are an estimated 7,000 rare diseases, in total affecting 
approximately 30 million Americans. Most of these are serious 
conditions with no approved therapies. Rare diseases constitute an 
enormous unmet medical need.
    Drug development for rare diseases, as well as for common diseases, 
relies on an indepth knowledge of the diseases' natural histories. 
Natural history is the course of the disease in the absence of a 
clinical intervention (that is, treatment under clinical care or 
study). Natural history knowledge makes possible the design of 
successful and efficient drug development programs. This knowledge has 
wide-ranging applications at every stage of drug development, for 
example, insight into the mechanism of disease, which can inform proof-
of-concept studies; development of biomarkers that can expedite 
clinical studies at every stage of drug development; recognition and 
understanding of phenotypes of disease that may respond more (or less) 
to a therapy; and knowledge of the aspects of disease that matter to 
patients, with an impact on developing drugs that have a meaningful 
impact on how a patient feels, functions, or survives. The lack of 
natural history knowledge can result in the failure of drug programs, 
even for drugs with great promise. Unfortunately, the natural history 
of rare diseases is often poorly understood.
    Impediments to the understanding of the natural history of a rare 
disease include the small numbers of patients and the sparse dispersal 
of clinical experience even among the chief clinical referral centers. 
The rare disease community is largely composed of small, diverse groups 
including patient and patient-family support, nonprofit disease groups 
(including umbrella groups), academic researchers, and small- to 
medium-sized biotechnology and pharmaceutical companies. For most rare 
diseases there has been no mechanism to systematically collect rare 
disease knowledge. In addition, it has become increasingly clear that 
it is vitally important to collect more knowledge from living patients 
over time, not simply to collect currently available information. This 
``longitudinal'' information about individual patients is invaluable to 
the design of a drug development program. The rare disease community is 
in need of a means of collecting and analyzing this knowledge: A 
natural history database tool.

B. Research Objectives

    The development of natural history databases will directly further 
FDA's public health mission. We anticipate that the successful 
implementation of a natural history database will have profound and 
far-reaching effects on development of therapies for rare diseases. As 
a basis for solid natural history knowledge of a disease it may help to 
make a clinical development program for a candidate therapy appear 
feasible, and thus a more attractive area to pharmaceutical companies 
for devoting a portion of their drug discovery resources. This too will 
lead to greater numbers of therapies for rare diseases.

C. Eligibility Information

    Only the following organization is eligible to apply: The National 
Organization for Rare Disorders. NORD is uniquely qualified to apply 
for this grant as the only applicant. Natural history studies is an 
area of unmet need and there are very few efforts towards building 
these studies. Those efforts that exist are very limited to specific 
diseases (e.g., cystic fibrosis, urea cycle disorders). These 
individual efforts cannot and do not support other patient groups 
starting their own studies. Most efforts are largely focused on patient 
communication and patient reports through Web-based self-reporting and 
are not likely to conform to sufficient scientific rigor to be able to 
support drug development. Although patient registries exist, these are 
not the same thing as natural history studies, and can often be very 
broad and general and cannot be customized to the depth and scope 
needed to support multiple natural history studies in a diverse group 
of rare diseases. The rigor, scope, and flexibility of NORD's platform, 
which comes from approximately 15 years of working with the rare 
disease community on these efforts, is unique and directly suited to 
the needs of FDA.

II. Award Information/Funds Available

A. Award Amount

    FDA/Center for Drug Evaluation and Research intends to fund up to 
$250,000, for fiscal year 2015 in support of this grant program. It is 
anticipated that one award will be made, not to

[[Page 49243]]

exceed $250,000 in total costs (direct plus indirect).

B. Length of Support

    The maximum project period is 1 year.

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 www.grants.gov. Search by 
Funding Opportunity Number: RFA-FD-15-038. 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)
     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: August 10, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-20130 Filed 8-14-15; 8:45 am]
 BILLING CODE 4164-01-P