Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional) | PAR 24 206

Frequently Asked Questions (FAQs)

What is the RDCRC funding opportunity (PAR-24-206)?

PAR-24-206 is a National Institutes of Health (NIH) funding opportunity to support the formation or continuation of Rare Diseases Clinical Research Consortia (RDCRCs) within the Rare Diseases Clinical Research Network (RDCRN). It uses a cooperative agreement mechanism (U54) to fund multi-site, highly collaborative, patient-centered research programs focused on rare diseases.

What is the overall goal of this program?

The goal is to speed progress in how rare diseases are identified, diagnosed, managed, and treated. The program aims to support coordinated efforts that connect clinical research, translational science, and (optionally) clinical trials to generate evidence that improves care and prepares the field for effective therapeutic testing.

What does NIH mean by a "multi-site research consortium" in this context?

In this announcement, a consortium is a coordinated group of institutions and disciplines that work together across multiple clinical sites. The expectation is that the group will build and execute a shared research agenda around rare conditions, harmonize methods and data collection where appropriate, and collaborate actively to accelerate progress beyond what a single site could achieve.

What is the RDCRN and how do RDCRCs relate to it?

The Rare Diseases Clinical Research Network (RDCRN) is the larger network framework. RDCRCs are the multi-site consortia funded within that network. This opportunity supports either the formation of new RDCRCs or the continuation (renewal) of existing RDCRCs as part of the RDCRN.

What types of activities does NIH expect RDCRCs to support?

RDCRCs are expected to address rare disease research challenges through coordinated approaches such as shared protocols across sites, standardized data collection, natural history and longitudinal studies, patient registries or cohort-building efforts, and translational pipelines that move discoveries toward interventions.

Is the program focused on clinical trials?

The program emphasizes clinical trial readiness and may include clinical trials as an optional component. A central theme is preparing the field for therapeutic testing by improving cohort readiness, characterization, infrastructure, and operational capability needed for rigorous trials.

What does "clinical trial readiness" mean in this NOFO?

Based on the information provided, clinical trial readiness refers to building the foundation that makes trials feasible and rigorous in rare diseases. This includes developing coordinated, multi-site approaches; improving cohort identification and characterization; using standardized data collection; and generating evidence (for example through natural history and longitudinal studies) that supports future or optional therapeutic testing.

Why is early and timely identification of rare disease patients emphasized?

The announcement highlights that many rare disease patients face long diagnostic delays and fragmented care. Since rare disease cohorts are often small and geographically dispersed, improving early and timely identification helps build well-characterized cohorts and supports both improved care and readiness for future clinical studies and trials.

What makes rare disease trials and studies especially challenging, according to the announcement?

The announcement notes that trials often struggle because patient cohorts are small, geographically dispersed, and not consistently characterized. RDCRCs are expected to address these issues through coordinated, standardized, and collaborative approaches across sites.

What does it mean that this is a U54 cooperative agreement?

A U54 cooperative agreement is a funding mechanism used when NIH expects substantial involvement in oversight and coordination. For RDCRCs, this reflects the need for harmonized methods, shared goals, and active collaboration across funded groups within a network-based program.

How involved will NIH be if an award is made?

The opportunity states that awardees should expect substantial NIH involvement in oversight and coordination, which is typical for cooperative agreements supporting network-based programs that require harmonized methods and active collaboration.

Who is eligible to apply as an applicant organization?

Eligible applicants include a wide range of U.S.-based public and private entities, including state/county/city/township/special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; nonprofits with or without 501(c)(3) status (other than institutions of higher education); for-profit organizations (other than small businesses); small businesses; and other organizations that meet NIH eligibility rules.

Are minority-serving institutions and community-based organizations eligible?

Yes. The NOFO explicitly highlights additional eligible applicant categories including Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), and faith-based or community-based organizations.

Are tribal governments eligible to apply?

Yes. Native American tribal governments that are federally recognized are eligible. The NOFO also notes eligibility for Indian/Native American tribal governments that are not federally recognized in its "other eligible applicants" language.

Are U.S. territories or possessions eligible to apply?

Yes. The NOFO includes U.S. territories or possessions among the explicitly highlighted eligible applicant categories.

Can a foreign (non-U.S.) organization apply as the primary applicant?

No. Non-domestic (non-U.S.) entities (foreign organizations) are not eligible to apply as the primary applicant for this opportunity.

Can a U.S. applicant include international participation in the project?

Yes, in limited form. While foreign organizations cannot be the primary applicant, foreign components are allowed as defined by the NIH Grants Policy Statement when scientifically justified and appropriately structured under NIH policy.

Are non-domestic components of U.S. organizations eligible to apply?

No. The information provided states that non-domestic components of U.S. organizations are not eligible to apply.

Why might a project want a foreign component in rare disease research?

The opportunity notes that rare disease patient populations and specialized expertise may be distributed globally. A foreign component can be relevant when scientifically justified, but the administrative lead must still be a domestic eligible organization.

Is this opportunity intended for new consortia, existing consortia, or both?

Both. The NOFO invites new and renewal applications, meaning existing consortia can seek continued support while new groups can propose to join and expand the network's overall coverage of rare diseases.

What is the award ceiling shown for this opportunity?

The award ceiling shown is $1,000,000.

What is the original closing date listed?

The original closing date listed is 2024-08-19.

When was this opportunity created?

The creation date listed is 2024-04-02.

What is the funding instrument for PAR-24-206?

This is a discretionary funding opportunity using a cooperative agreement mechanism, specifically a U54.

What does it mean that the NOFO references multiple CFDA numbers?

The NOFO references multiple CFDA numbers (93.121, 93.172, 93.173, 93.233, 93.350, 93.837, 93.838, 93.839, 93.840, 93.846, 93.847, 93.853, 93.855, 93.865, 93.866), reflecting that RDCRN efforts can involve multiple NIH institutes and centers and span health and related areas.

What kinds of organizations are a strong fit for this program?

The program is designed for organizations that can demonstrate real consortium capacity: coordinating multiple clinical sites, engaging patients and advocacy communities, conducting high-quality clinical and translational studies, and producing shared resources and evidence that move rare disease care forward.

What kinds of outcomes is NIH looking for from funded RDCRCs?

Based on the description provided, NIH is looking for outcomes such as shorter paths to diagnosis, well-characterized cohorts, coordinated and standardized research approaches across sites, and infrastructure and readiness (including endpoints and operational capability) needed to launch or support rigorous clinical trials when therapies are ready to be tested.

Does the program require patient-centered approaches?

Yes. The opportunity emphasizes patient-centered programs and highlights the importance of engaging patients and advocacy communities as part of highly collaborative consortium efforts.