RDCRN Applicant Information

The Rare Diseases Clinical Research Network (RDCRN) was established by The Rare Diseases Act of 2002 (Public Law 107-280), which directed the National Institutes of Health (NIH) to support "Rare Disease Regional Centers of Excellence" for clinical research, career enhancement, and demonstration of diagnostic, prevention, control and treatment methods for rare diseases. The RDCRN has been continually funded through competitive grant cycles every five years since 2003.

Open Funding Opportunity

See the open funding opportunity for the RDCRN Program.

All applicants are strongly encouraged to contact NIH institute, center and office (ICO) staff to discuss how their proposed work aligns with the goals of this notice of funding opportunity (NOFO) and the RDCRN.

Prior RDCRC Notice of Funding Opportunity

• RFA-RR-03-008
• RFA-OD-08-001
• RFA-TR-13-002
• RFA-TR-18-020

Resources

If you are applying for the PAR-24-206 Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional), you may find the resources below helpful.

Technical Assistance Webinar:

• Attend the upcoming technical assistance webinar for potential applicants. NIH staff will answer questions about this and the companion NOFO. The time, date and participant log in information for the webinar will be announced in an NIH Guide Notice.
• Download slides (coming soon).
• View recording (coming soon). 

Questions and Answers

What is the purpose of this Notice of Funding Opportunity?

The objective of this Notice of Funding Opportunity (NOFO) is to invite new and renewal applications for the Rare Diseases Clinical Research Consortia (RDCRC) that comprise the Rare Diseases Clinical Research Network (RDCRN). The RDCRCs are intended to advance and improve diagnosis, management, and treatment of numerous, diverse rare diseases through highly collaborative, multi-site, patient-centric, translational and clinical research. Special emphasis will be placed on the early and timely identification of individuals with rare diseases and clinical trial readiness.

This is a reissue of RFA-TR-18-020. What are the major changes?

• In the required Rare Disease Status attachment (under Other Attachments), all applicants must justify that the diseases/conditions being studied are rare in the United States.
  • The Rare Disease Status attachment may be no more than 3 pages in length and must include all targeted diseases/conditions.
  • In an "Other Attachment" entitled "Rare Disease Status", all applicants must include a justification that the diseases/conditions being studied are rare in the U.S.
  • This section may include one or more references confirming that the prevalence of the diseases/conditions that are the primary focus of the research application is 200,000 or fewer patients in the U.S., as defined by The Rare Diseases Act of 2002 (Public Law 107-280). If the diseases/conditions have been granted orphan status by the U.S. Food and Drug Administration, provide this information in the justification.
  • If it is a rare variant or subset of a more common condition, provide a justification for including this variant in the RDCRC. Describe the scientific basis for separating biomarker/clinical outcome assessment validation for this rare variant or subset from that of the common condition.
  • Applications missing a Justification for Rare Disease Status may be deemed incomplete and not sent forward for review.
• Applications must include at least two and no more than four Clinical Research Projects.
• It is imperative that RDCRC applicants carefully review the Data Management and Coordinating Center (DMCC) NOFO (RFA-TR-24-021) to fully understand the resources and services that will be provided to the network participants by the DMCC. Applicants should ensure that planned activities involve coordination with the DMCC and do not replicate efforts.
• Advancing rare disease research by freely sharing high-value data is a critical goal of the program. All RDCRC award recipients are required to work collaboratively within the RDCRN DMCC cloud environment and to ultimately share their data within the RDCRN Data Repository. Informed Consent for every protocol should include language that allows broad sharing of the data while protecting the confidentiality of and minimizing the risk for re-identification of the participant.

What qualifies as a rare disease for the RDCRN?

In this NOFO, a rare disease is defined as a condition affecting fewer than 200,000 people in the U.S., as defined by the Rare Diseases Act of 2002 (Public Law 107-280).

How many rare diseases must be represented in the application?

Each RDCRC application must indicate at least three different rare diseases that may share, but are not limited to, common pathways/mechanisms of action/organ system, and may be defined as:

• Conditions — a particular state of being that limits/restricts something else.
• Disorders — abnormal physical or mental conditions or ailments.
• Syndromes — a group of symptoms that occur together, or a condition characterized by a set of associated symptoms.
• Diseases — a disorder of structure or function that affects a specific location and is not simply a result of physical injury.

What is “clinical trial readiness"?

For this NOFO, clinical trial readiness is the state of having validated clinical research tools and sufficient knowledge of disease natural history to design efficient clinical trials. Validated clinical research tools can include biomarkers or clinical outcome assessment measures that are fit-for-purpose within a defined context of use relevant to the clinical trials. Knowledge of disease natural history necessary for clinical trial design can include, but is not limited to, characteristics for stratification or determining inclusion and exclusion criteria, the stage of disease progression that may be responsive to treatment, and data needed for determining sample size through power calculations.

Who needs to have an eRA account?

Applicant organizations must be registered in eRA Commons and should start the registration process at least six weeks before the grant application submission deadline to allow plenty of time to address unforeseen issues. All Senior/Key Personnel, as defined in the NIH Grants Policy Statement (NIH GPS 1.2), and Other Significant Contributors (OSCs) must be listed on the R&R Senior/Key Person Profile Form and must also be registered in the NIH eRA system (see NOT-OD-24-042, Create and Manage an eRA Commons Account webpage, and the Application Guide). Note: All Errors must be corrected before an application can be successfully submitted to the agency.

Where can I find the RDCRN Data Sharing Template Document (Section V1.3 Data Management and Sharing)?

All RDCRN Data Sharing resources can be found on the RDCRN Data Sharing Resources webpage. The Principles of Data Sharing Checklist outlines the basic elements that need to be incorporated in the informed consent language, the contractual language established between the consortium administrative core and its sites, and the consortium policies that govern data sharing, use, and publication within the consortium and with external users. The goal of the RDCRN Guidance for the Development of Data Sharing Policies by Individual Consortia Document is to offer non-binding guidance to consortia on general principles for data sharing and key factors to consider when developing data sharing policies.

How many awards will be made?

The number of awards is contingent upon NIH appropriations and the submission of a sufficient number of meritorious applications that align with the specific research interests and priorities of the participating NIH ICO.

Does each clinical project need a 12-page component or are they bundled together?

Each separate clinical trial component (2-4) is allowed a 12-page research plan. See the NIH Grants & Funding Page Limits. The U54 mechanism falls under the category of "all other activity codes" and requires following NOFO instructions.