80 FR 49242 - Disease Natural History Database Development-(U24)
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 158 (August 17, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 158 (August 17, 2015)
| Page Range | 49242-49243 | |
| FR Document | 2015-20130 |
[Federal Register Volume 80, Number 158 (Monday, August 17, 2015)] [Notices] [Pages 49242-49243] From the Federal Register Online [www.thefederalregister.org] [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, [email protected]. Vieda Hubbard, Office of Acquisition and Grants Services, Food and Drug Administration, 5630 Fishers Lane, Rockville, MD 20857, 240-402- 7588, [email protected]. 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
![49242 Federal Register / Vol. 80, No. 158 / Monday, August 17, 2015 / Notices
IV. Electronic Access to obtain detailed requirements, please ‘‘longitudinal’’ information about
Persons with access to the Internet refer to the full FOA located at individual patients is invaluable to the
may obtain the document at either www.grants.gov. Search by Funding design of a drug development program.
http://www.fda.gov/Drugs/ Opportunity Number: RFA–FD–15–038. The rare disease community is in need
GuidanceCompliance SUPPLEMENTARY INFORMATION: of a means of collecting and analyzing
RegulatoryInformation/Guidances/ this knowledge: A natural history
I. Funding Opportunity Description database tool.
default.htm or http://
www.regulations.gov. RFA–FD–15–038
93.103 B. Research Objectives
Dated: August 12, 2015.
A. Background The development of natural history
Leslie Kux,
There are an estimated 7,000 rare databases will directly further FDA’s
Associate Commissioner for Policy. public health mission. We anticipate
[FR Doc. 2015–20230 Filed 8–14–15; 8:45 am] diseases, in total affecting
approximately 30 million Americans. that the successful implementation of a
BILLING CODE 4164–01–P
Most of these are serious conditions natural history database will have
with no approved therapies. Rare profound and far-reaching effects on
diseases constitute an enormous unmet development of therapies for rare
DEPARTMENT OF HEALTH AND diseases. As a basis for solid natural
HUMAN SERVICES medical need.
Drug development for rare diseases, as history knowledge of a disease it may
Food and Drug Administration well as for common diseases, relies on help to make a clinical development
an indepth knowledge of the diseases’ program for a candidate therapy appear
[Docket No. FDA–2015–N–0012] natural histories. Natural history is the feasible, and thus a more attractive area
course of the disease in the absence of to pharmaceutical companies for
Disease Natural History Database devoting a portion of their drug
a clinical intervention (that is, treatment
Development—(U24) discovery resources. This too will lead
under clinical care or study). Natural
AGENCY: Food and Drug Administration, history knowledge makes possible the to greater numbers of therapies for rare
HHS. design of successful and efficient drug diseases.
ACTION: Notice. development programs. This knowledge C. Eligibility Information
has wide-ranging applications at every
SUMMARY: The Food and Drug stage of drug development, for example, Only the following organization is
Administration (FDA) is announcing the insight into the mechanism of disease, eligible to apply: The National
availability of grant funds for the which can inform proof-of-concept Organization for Rare Disorders. NORD
support of Natural History Database studies; development of biomarkers that is uniquely qualified to apply for this
Development. The National can expedite clinical studies at every grant as the only applicant. Natural
Organization for Rare Disorders (NORD) stage of drug development; recognition history studies is an area of unmet need
is developing an Internet-based data and understanding of phenotypes of and there are very few efforts towards
collection tool with promise to further disease that may respond more (or less) building these studies. Those efforts that
the accumulation of natural history data to a therapy; and knowledge of the exist are very limited to specific
for many rare diseases. The goal of this aspects of disease that matter to diseases (e.g., cystic fibrosis, urea cycle
grant is to enable NORD to further patients, with an impact on developing disorders). These individual efforts
develop, refine, and disseminate the drugs that have a meaningful impact on cannot and do not support other patient
database tool. how a patient feels, functions, or groups starting their own studies. Most
DATES: Important dates are as follows: survives. The lack of natural history efforts are largely focused on patient
1. The application due date is knowledge can result in the failure of communication and patient reports
September 4, 2015. drug programs, even for drugs with great through Web-based self-reporting and
2. The anticipated start date is promise. Unfortunately, the natural are not likely to conform to sufficient
September 2015. history of rare diseases is often poorly scientific rigor to be able to support
3. The opening date is July 2015. understood. drug development. Although patient
4. The expiration date is September 5, Impediments to the understanding of registries exist, these are not the same
2015. the natural history of a rare disease thing as natural history studies, and can
ADDRESSES: Submit electronic include the small numbers of patients often be very broad and general and
applications to: http://www.grants.gov. and the sparse dispersal of clinical cannot be customized to the depth and
For more information, see section III of experience even among the chief scope needed to support multiple
the SUPPLEMENTARY INFORMATION section clinical referral centers. The rare disease natural history studies in a diverse
of this notice. community is largely composed of group of rare diseases. The rigor, scope,
FOR FURTHER INFORMATION CONTACT: small, diverse groups including patient and flexibility of NORD’s platform,
James Kaiser, Office of Translational and patient-family support, nonprofit which comes from approximately 15
Sciences, Center for Drug Evaluation disease groups (including umbrella years of working with the rare disease
and Research, Food and Drug groups), academic researchers, and community on these efforts, is unique
Administration, 10903 New Hampshire small- to medium-sized biotechnology and directly suited to the needs of FDA.
Ave., Silver Spring, MD 20993, 301– and pharmaceutical companies. For
II. Award Information/Funds Available
mstockstill on DSK4VPTVN1PROD with NOTICES
796–1237, james.kaiser@fda.hhs.gov. most rare diseases there has been no
Vieda Hubbard, Office of Acquisition mechanism to systematically collect rare A. Award Amount
and Grants Services, Food and Drug disease knowledge. In addition, it has
Administration, 5630 Fishers Lane, become increasingly clear that it is FDA/Center for Drug Evaluation and
Rockville, MD 20857, 240–402–7588, vitally important to collect more Research intends to fund up to
Vieda.Hubbard@fda.hhs.gov. knowledge from living patients over $250,000, for fiscal year 2015 in support
For more information on this funding time, not simply to collect currently of this grant program. It is anticipated
opportunity announcement (FOA) and available information. This that one award will be made, not to
VerDate Sep<11>2014 16:57 Aug 14, 2015 Jkt 235001 PO 00000 Frm 00045 Fmt 4703 Sfmt 4703 E:\FR\FM\17AUN1.SGM 17AUN1](https://thefederalregister.org/doc/80_FR_49400/page/1.svg)
![Federal Register / Vol. 80, No. 158 / Monday, August 17, 2015 / Notices 49243
exceed $250,000 in total costs (direct Science and Innovation (M–CERSI) is a first-come, first-served basis. The costs
plus indirect). announcing a public workshop entitled of registration for the different
‘‘Scientific Inquiry Into How Mobile categories of attendees are as follows:
B. Length of Support
Health and Social Data Sources May
The maximum project period is 1 Inform Medical Product Safety and Category Cost
year. Efficacy.’’ The purpose of the public
workshop is to discuss important Industry Representatives .................... $50
III. Electronic Application,
scientific questions about using two of Charitable Nonprofit/Academic ........... 50
Registration, and Submission
the most ubiquitous and fastest growing Government/Students ......................... 0
Only electronic applications will be data sources, mobile health data and
accepted. To submit an electronic social computing data, focusing Persons interested in attending this
application in response to this FOA, especially on the implications for public workshop must register online at
applicants should first review the full product safety. http://go.umd.edu/
announcement located at
DATES: The public workshop will be mobilesocialanalytics by September 1,
www.grants.gov. Search by Funding
held on September 11, 2015, from 12:30 2015. To register, please refer to:
Opportunity Number: RFA–FD–15–038.
p.m. to 5:30 p.m. http://www.rhsmith.umd.edu/centers-
For all electronically submitted
ADDRESSES: The public workshop will excellence/center-health-information-
applications, the following steps are
required. be held at the Adele H. Stamp Student decision-systems/events/m-cersi-
• Step 1: Obtain a Dun and Bradstreet Union, University of Maryland, 1021A workshop. Early registration is
(DUNS) Number Adele H. Stamp Student Union, College recommended because space is limited.
• Step 2: Register With System for Park, MD. For additional travel and Those without Internet access should
Award Management (SAM) hotel information, please refer to: contact Ann Anonsen to register (see
• Step 3: Obtain Username & http://go.umd.edu/ FOR FURTHER INFORMATION CONTACT
Password mobilesocialanalytics. regarding registration).
• Step 4: Authorized Organization Submit electronic comments to
http://www.regulations.gov. Submit Attendees are responsible for their
Representative (AOR) Authorization
• Step 5: Track AOR Status written comments to the Division of own hotel accommodations. If you need
• Step 6: Register With Electronic Dockets Management (HFA–305), Food special accommodations due to a
Research Administration (eRA) and Drug Administration, 5630 Fishers disability, please contact Ann Anonsen,
Commons Lane, Rm. 1061, Rockville, MD 20852. at 301–405–0285 or email: aanonsen@
Steps 1 through 5, in detail, can be All comments should be identified with umd.edu.
found at http://www07.grants.gov/ the docket number found in brackets in
III. Comments
applicants/organization_ the heading of this document.
registration.jsp. Step 6, in detail, can be FOR FURTHER INFORMATION CONTACT: Regardless of attendance at the public
found at https://commons.era.nih.gov/ Regarding this notice: Leslie D. workshop, interested persons may
commons/registration/ Wheelock, Food and Drug submit either electronic comments to
registrationInstructions.jsp. After you Administration, Office of the http://www.regulations.gov or written
have followed these steps, submit Commissioner, 10903 New Hampshire comments to the Division of Dockets
electronic applications to http:// Ave., Bldg. 1, Rm. 4345, Silver Spring, Management (see ADDRESSES). The
www.grants.gov. MD 20993, 301–796–8450, FAX: 301– deadline for submitting comments
Dated: August 10, 2015. 847–8106; email: leslie.wheelock@ related to this public workshop is
Leslie Kux, fda.hhs.gov. August 28, 2015. It is only necessary to
Regarding registration: Ann Anonsen,
Associate Commissioner for Policy. send one set of comments. Identify
University of Maryland, Fischell Dept.
[FR Doc. 2015–20130 Filed 8–14–15; 8:45 am] comments with the docket number
of Bioengineering, 2207 Jeong H. Kim
BILLING CODE 4164–01–P found in brackets in the heading of this
Bldg., College Park, MD 20742, 301–
405–0285, FAX: 301–405–9953, email: document. Received comments may be
aanonsen@umd.edu. seen in the Division of Dockets
DEPARTMENT OF HEALTH AND Management between 9 a.m. and 4 p.m.,
HUMAN SERVICES SUPPLEMENTARY INFORMATION:
Monday through Friday, and will be
I. Background posted to the docket at http://
Food and Drug Administration
Mobile health and social computing www.regulations.gov.
[Docket No. FDA–2015–N–2735] data sources create unique and hitherto Dated: August 10, 2015.
unavailable opportunities, but there are Leslie Kux,
Scientific Inquiry Into How Mobile
important questions that need to be
Health and Social Data Sources May Associate Commissioner for Policy.
answered. This workshop will bring
Inform Medical Product Safety and [FR Doc. 2015–20129 Filed 8–14–15; 8:45 am]
together thought leaders from industry,
Efficacy; Public Workshop; Request
academia, and the regulatory BILLING CODE 4164–01–P
for Comments
communities to reflect on the
AGENCY: Food and Drug Administration, opportunities and challenges that these
mstockstill on DSK4VPTVN1PROD with NOTICES
HHS. new data sources create.
ACTION: Notice of public workshop; II. Attendance and Registration
request for comments.
There is a registration fee to attend
SUMMARY: The Food and Drug this public workshop. The registration
Administration (FDA), in collaboration fee is charged to help defray the costs
with the University of Maryland’s for facilities, materials, and food. Seats
Center of Excellence in Regulatory are limited, and registration will be on
VerDate Sep<11>2014 16:57 Aug 14, 2015 Jkt 235001 PO 00000 Frm 00046 Fmt 4703 Sfmt 9990 E:\FR\FM\17AUN1.SGM 17AUN1](https://thefederalregister.org/doc/80_FR_49400/page/2.svg)
Document Created: 2015-12-15 11:06:50
Document Modified: 2015-12-15 11:06:50
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| Dates | Important dates are as follows: | |
| 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, [email protected] | |
| FR Citation | 80 FR 49242 |
2025 Federal Register | Disclaimer | Privacy Policy
USC | CFR | eCFR