The notice of funding opportunity (NOFO) Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54, Clinical Trial Optional) aims to invite new and renewal applications for the individual RDCRCs that comprise the Rare Diseases Clinical Research Network, which since 2002 has supported a broad range of clinical research, including clinical trial readiness, natural history studies, identification of biomarkers and outcome measures, and early-phase clinical trials.
Each RDCRC will advance and improve diagnosis, management, and treatment of numerous, diverse rare diseases through highly collaborative, multi-site, patient-centric, translational, and clinical research. In this context, a rare disease is defined by law as a condition affecting fewer than 200,000 individuals in the United States. Special emphasis will be placed on the early and timely identification of individuals with rare diseases and clinical trial readiness.
RDCRC Elements
Each RDCRC application must indicate at least three different rare diseases that may share common pathways or mechanisms of action or 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.
Each RDCRC is required to have one natural history or longitudinal study. Each research study must be conducted at multiple sites; however, pilot studies may be conducted at a single site.
Additionally, each RDCRC must form partnerships with patient advocacy groups.
RDCRC Structure
The structural requirements of an RDCRC under this NOFO are as follows:
- Administrative Core—responsible for activities such as administrative support for RDCRC, management and sharing of data and biospecimens, and coordination of patient advocacy groups participation.
- Pilot/Feasibility Governance Core—established to enable future innovative single- or multi-site pilot studies aimed at advancing the diagnosis, clinical trial readiness, management, and/or treatment of rare diseases. Pilot projects that extend RDCRC research collaborations beyond the RDCRN are allowed. Pilot projects may be awarded to institutions that are not already RDCRC members; however, execution of new subawards may be required.
- Career Enhancement Core—to provide support for career enhancement-related activities and support the education of diverse candidates in rare diseases research.
- Clinical Research Projects (two to four)—one of the projects must be longitudinal in nature with the intent of understanding the clinical course of the disease and helping inform future clinical trials (e.g., natural history studies).
Also, note that RDCRCs will receive resources and services from the RDCRN’s Data Management and Coordinating Center, which will be established through the companion NOFO Single Source for the Continuation of the Data Management and Coordinating Center (DMCC) for Rare Diseases Clinical Research Network (RDCRN) (U2C, Clinical Trial Not Allowed). Applicants should ensure all planned activities involve coordination with the DMCC as not to replicate efforts.
NIAID Research Interests and Priorities
As a participating institute, NIAID is especially interested in research activities on rare diseases classified into four areas: infectious diseases, primary immunodeficiency diseases, autoimmune diseases, and allergic diseases.
- Infectious diseases include diseases caused by bacteria, parasites, viruses, fungi, and other pathogens. Research on rare infectious diseases is aimed at delineating mechanisms of disease pathogenesis and developing more effective diagnostic, treatment, and prevention strategies.
- Primary immunodeficiency diseases are hereditary disorders caused by intrinsic defects in the cells of the immune system and are characterized by unusual susceptibility to infection. NIAID research is focused on the identification of gene defects and immunologic abnormalities that lead to defective function, and the development of new approaches for the diagnosis and treatment of primary immunodeficiency disease, including gene transfer as an effective and curative therapy.
- Autoimmune diseases are diseases in which the immune system mistakenly attacks and damages the body's own cells and tissues. NIAID research is focused on the identification of mechanisms of pathogenesis and the development of new approaches to prevention and treatment.
- Allergies are inappropriate or exaggerated reactions of the immune system to substances that cause no symptoms in the majority of people.
For applications that propose clinical trials, NIAID strongly encourages applicants to contact NIAID staff early in the planning stage for assistance meeting NIAID clinical trial requirements.
Make Sure Your Application Is Responsive
The following types of studies are not responsive to this NOFO. We will consider applications proposing such studies nonresponsive, withdraw them from review, and not consider them for funding.
- Single site clinical studies
- Phase III clinical trials as part of Clinical Research Projects
- Fewer than three rare diseases included
- Absence of at least one longitudinal study
- Proposing less than two or more than four Clinical Research Projects
- Absence of patient advocacy group involvement
- Basic sciences studies
- Applications that propose any type of animal studies within the RDCRC. The use of in vitro models must be relevant to clinical endpoints (i.e., testing drugs, validating biomarkers versus more basic research)
Refer to the NOFO for additional details.
Other Application Elements
Your application must include milestones. Describe how measurable outcomes will be collected using rigorous and transparent experimental approaches. Future year support is contingent on satisfactory achievement of performance milestones. If milestones are not achieved fully, NIH may request development of a remedial plan and more frequent monitoring of progress.
Applicants may request up to $1 million in annual direct costs. All costs must be well justified in accordance with the activity proposed.
Applicants may request up to 5 years of support, but the actual needs of the research should determine the length of the project period.
The deadline to apply is August 13, 2024.
Have Questions?
Staff from the National Center for Advancing Translational Sciences have assembled a helpful set of Questions and Answers about this initiative.
You can also watch the webinar recording Technical Assistance Webinars for PAR-24-206: “RDCRC for the RDCRN (U54, Clinical Trial Optional)”.
For questions specific to NIAID’s areas of research interest, reach out to our scientific/research contact Dr. Ruth Florese at ruth.florese@niaid.nih.gov or 301-761-6284.