National Center for Advancing Translational Sciences – Rare Diseases
Program Funding
Annual program obligations reported to SAM.gov.
Program Objective
The National Center for Advancing Translational Sciences' (NCATS') mission is to turn research observations into health solutions through translational science. We work to develop or enhance the development, testing, and implementation of diagnostics and therapeutics for a wide range of diseases and conditions. Key approaches include understanding what’s similar across diseases to spur multiple treatments at a time, developing models that better predict a person’s reaction to treatment, enhancing clinical trials so results more accurately reflect the patient population, and leveraging real-world data and data science approaches to address public health needs. Facilitating these approaches are our robust partnerships with other government agencies, including other NIH institutes, centers, and offices; industry; academia; nonprofit organizations; and patients, patient advocates, and other communities. NCATS' vision is to bring more treatments for all people more quickly.
Eligibility
Eligible Applicants
- U.S. Federal Government
- U.S. State Government
- U.S. Territory Government
- Department/Agency of U.S. State
- Department/Agency of U.S. Territorial Gov
- Interstate Organization
- Federally Recognized Tribal Government
- Tribal Government (other)
- Tribally Designated Housing Authority
- Municipality/Township Government
- County Government
- School District Government
- School District
- Local Government Consortium
- Public Housing Authority
- Other Special District Government
- Local
- State
- Territorial
- Tribal
- Foreign Government
- Foreign Nonprofit Organization
- Foreign Not-for-Profit Organization
- Foreign For-Profit Organization
- International Organization
- Nonprofit Organization
- Not-for-Profit Organization
- For-Profit Organization
How to Apply
Award Procedure
The initial review of applications from eligible institutions is conducted by committees comprised of authorities in various fields of biomedical research and science education, as appropriate. Each application is given a peer evaluation for merit. Recommendations for award are forwarded to the NCATS Advisory Council for the second level of review and recommendation for award.
Decision Timeline
- Approval: > 180 Days
- Renewal interval: > 180 Days
- Appeal: From 15 to 30 days
Program details & compliance
Description
The Division of Rare Diseases Research Innovation (DRDRI) facilitates and coordinates many rare disease programs at NCATS, as well as NIH-wide activities involving rare diseases research. Major efforts include the Rare Diseases Clinical Research Network and gene-targeted therapy development efforts, such as the Bespoke Gene Therapy Consortium and Platform Vector Gene Therapy Pilot Project. DRDRI also develops and manages rare disease information resources for patients and patient advocacy organizations. The Advanced Therapeutics and Research Branch supports novel and innovative research efforts related to innovation in clinical trial design of treatments and therapies for rare disease patients. It also supports novel and innovative research efforts related to innovation in clinical trials of gene-targeted therapies. The Collaborative Research, Informatics and Special Programs Branch coordinates NIH-wide activities involving research into combating and treating the broad array of rare diseases and maintains a centralized informational database on rare diseases. It also collaborates within and outside NCATS in assessing the burden of rare diseases on the healthcare system.
Mission Categories
Primary: Research and Development
Other categories:
General Health and MedicalHealth EducationMedical EducationCapacity Building/Cybersecurity
Use of Funds
Allowed Uses
NCATS is all about getting more treatments to all patients more quickly. There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. A novel drug, device or intervention can take 14 years and $2 billion to develop, with a failure rate exceeding 95%. NCATS is directly addressing this problem by discovering new technologies and other approaches that could greatly accelerate the process of developing and deploying solutions that can be used by all translational researchers. NCATS supports a network of research consortia, each targeted to several related rare diseases. NCATS also supports an extensive resource of information for the public on rare diseases. NCATS supports expansion of the target landscape for therapeutic development by supporting pilot studies on druggable proteins that have largely been neglected by the research community, but are associated with rare diseases. It also supports strategies that will enable targeting biologic entities that cannot be modulated with traditional drug development of biologic strategies. In cases where a potential therapeutic has been identified for a rare disease, and a model system for testing the treatment has already been established, NCATS will support proof of concept studies to advance development of the candidate treatment.
Required Documentation
The required credentials of the applicant are described in the relevant Notice of Funding Opportunity.
Reporting & Compliance
Applicable 2 CFR 200 Subparts
- Subpart B — General Provisions
- Subpart C — Pre-Federal Award Requirements
- Subpart D — Post-Federal Award Requirements
- Subpart E — Cost Principles
- Subpart F — Audit Requirements