Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
ID: 359645Type: Posted
Overview

Buyer

National Institutes of Health (HHS-NIH11)

Eligible Applicants

Others

Funding Category

Income Security and Social Services

Funding Instrument

Grant

Opportunity Category

Discretionary

Cost Sharing or Matching Requirement

Yes

Original Source

https://grants.gov/search-results-detail/359645

Additional Information

http://grants.nih.gov/grants/guide/pa-files/PAR-25-450.html
Timeline
    Description

    The National Institutes of Health (NIH) has announced a funding opportunity titled "Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)" aimed at supporting clinical projects that enhance readiness for clinical trials in rare diseases. This initiative seeks applications that focus on developing and testing biomarkers and clinical outcome measures, as well as defining the characteristics of rare diseases to facilitate the design of future clinical trials. The program is particularly important for addressing the needs of rare diseases affecting fewer than 200,000 individuals in the U.S., with a maximum budget of $275,000 for direct costs over a project period of up to two years. Interested applicants can find more information and submit inquiries via email to grantsinfo@nih.gov, with the application deadline set for July 20, 2028.

    Point(s) of Contact
    grantsinfo@nih.gov
    Files
    Title
    Posted
    PAR-25-450-Full-Announcement.html
    PAR-25-450.html
    The Department of Health and Human Services (HHS) and the National Institutes of Health (NIH) have reissued a Notice of Funding Opportunity (NOFO) titled "Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)." This initiative, PAR-25-450, aims to support clinical projects that address critical needs for clinical trial readiness in rare diseases by facilitating the movement of candidate therapeutics or diagnostics toward clinical trials. The NOFO emphasizes developing and testing rigorous biomarkers and clinical outcome assessment measures, or defining the presentation and course of rare diseases to enable the design of upcoming clinical trials. The maximum project period is 2 years, with a budget of up to $275,000 direct costs. Applications must focus on rare diseases affecting fewer than 200,000 individuals in the U.S. and should not propose clinical trials. The NOFO encourages leveraging existing research resources and partnerships to optimize trial readiness.
    Similar Opportunities
    Forecast for Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) is preparing to publish a Notice of Funding Opportunity (NOFO) for the R21 grant aimed at enhancing clinical trial readiness for rare diseases, disorders, and syndromes. This initiative seeks to support research projects that address critical needs in the field, such as the development of biomarkers and clinical outcome measures, which are essential for facilitating the transition of therapeutics and diagnostics into clinical trials. The opportunity is particularly significant as it aims to improve the success rates of clinical trials for rare diseases, which often face unique challenges. Interested small businesses are encouraged to begin forming collaborations and developing proposals, with the estimated synopsis posting date set for November 14, 2025, and applications expected to be accepted until February 16, 2026. For further inquiries, potential applicants can contact Dr. Alice Chen Grady at alice.chen2@nih.gov or by phone at 301-827-2015.
    Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering a funding opportunity titled "Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)" aimed at supporting preclinical efficacy studies for therapeutic agents targeting rare diseases. This initiative seeks to fund projects that demonstrate the effectiveness of proposed treatments in established rare disease models, along with necessary pharmacodynamic and pharmacokinetic studies, to facilitate further development towards Investigational New Drug (IND) applications or clinical trials. With a total funding budget of up to $1.2 million, the NIH anticipates awarding 3-5 grants, each with a maximum budget of $275,000 over a two-year project period. Interested applicants must submit letters of intent by May 1, 2024, with full applications due by May 3, 2024, and can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries.
    Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) has announced a funding opportunity for preclinical proof of concept studies targeting rare diseases under the R21 Exploratory/Developmental Research Grant mechanism. This initiative aims to support efficacy studies in established preclinical models for therapeutic agents, including small molecules and biologics, with the goal of advancing projects toward Investigational New Drug (IND) applications. The funding is particularly significant as it addresses the unmet medical needs of approximately 30 million individuals in the U.S. affected by rare diseases, enhancing the likelihood of progressing novel therapies to clinical trials. The total funding available is up to $1,200,000 for FY26, with a maximum budget of $275,000 per two-year project. Interested applicants must submit their letters of intent by May 1, 2025, and can find more information by contacting NIH Grants Information at grantsinfo@nih.gov or visiting the opportunity's webpage at https://grants.nih.gov/grants/guide/rfa-files/RFA-TR-25-002.html.
    NIH Exploratory/Developmental Research Project Grant (Parent R21 Clinical Trial Required)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering the Exploratory/Developmental Research Project Grant (Parent R21 Clinical Trial Required) to support innovative research projects in the early stages that can lead to significant advancements in biomedical, behavioral, and clinical research. This funding opportunity requires applicants to propose at least one clinical trial and is aimed at addressing critical gaps in knowledge within the scientific missions of participating NIH Institutes and Centers. Eligible applicants include a diverse range of organizations, such as higher education institutions, nonprofits, and foreign entities, with a total project period limited to two years and a budget of up to $275,000. The application period opens on January 16, 2025, and closes on January 8, 2028; interested parties can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries.
    Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R21 - Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering a funding opportunity titled "Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R21 - Clinical Trial Optional)" aimed at promoting research and development of novel screening methods and therapeutic interventions for conditions identified through newborn screening. This initiative encourages innovative projects that generate preliminary data on both existing and potentially fatal genetic disorders, emphasizing the importance of early detection and intervention. The program is open to a wide range of eligible applicants, including higher education institutions, nonprofits, and governmental bodies, with a total funding ceiling of $275,000 over two years. Interested applicants should note that the submission deadlines begin on January 16, 2025, and can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries.
    Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
    Food and Drug Administration
    The Food and Drug Administration (FDA) is offering a federal grant opportunity titled "Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)." This grant aims to fund clinical trials that evaluate the efficacy and safety of products intended to address unmet needs in rare diseases or conditions, with the goal of increasing the number of approved treatments in this area. The initiative is crucial for enhancing drug development for rare diseases, thereby potentially improving patient outcomes. Interested applicants, including higher education institutions, nonprofits, for-profit organizations, and various levels of government, can reach out to Kimberly Pendleton at Kimberly.Pendleton@fda.hhs.gov or call 240-402-7610 for more information. The opportunity is currently posted for the fiscal year 2025, with no cost-sharing or matching requirements.
    Notice of Intent to Publish a Notice of Funding Opportunity (NOFO) for the Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN)
    National Institutes of Health
    The National Institutes of Health (NIH) is preparing to publish a Notice of Funding Opportunity (NOFO) for the Rare Diseases Clinical Research Consortia (RDCRC) as part of the Rare Diseases Clinical Research Network (RDCRN). This initiative aims to solicit applications for collaborative, multi-site research focused on advancing the diagnosis, management, and treatment of rare diseases, with an emphasis on addressing unmet clinical trial readiness needs. The funding, estimated at $1 million, will support specialized centers through cooperative agreements, with the NOFO expected to be published in winter 2024 and applications due in summer 2024. Interested applicants should prepare to develop meaningful collaborations, as the estimated award date is set for July 1, 2025.
    Notice of Intent to Publish a Funding Opportunity Announcement for the Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN)
    National Institutes of Health
    The National Institutes of Health (NIH) plans to publish a Funding Opportunity Announcement (FOA) for the Rare Diseases Clinical Research Consortia (RDCRC) as part of the Rare Diseases Clinical Research Network (RDCRN). This initiative aims to enhance the diagnosis, management, and treatment of rare diseases through collaborative, multi-site, patient-centric research, with a focus on addressing unmet clinical trial readiness needs. The estimated total program funding for this opportunity is $1,000,000, with the FOA expected to be published in 2024 and applications due later that year. Interested applicants can reach out to Dr. Tiina K. Urv at 301-827-2746 or via email at urvtiin@mail.nih.gov for further information.
    Ancillary Studies to Ongoing Clinical Projects (R21 Clinical Trial Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) is announcing an upcoming funding opportunity titled "Ancillary Studies to Ongoing Clinical Projects (R21 Clinical Trial Not Allowed)," aimed at supporting research that leverages resources from ongoing clinical studies. This initiative encourages applications for ancillary studies that utilize well-characterized patient cohorts, infrastructure, data, and biological samples, while aligning with the mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). With an estimated total program funding of $600,000 and the expectation of awarding two grants, potential applicants are advised to begin developing collaborations and project proposals in advance of the official solicitation, which is expected to be published in early 2026. For further inquiries, interested parties may contact Dr. Heiyoung Park at Heiyoung.Park@nih.gov or by phone at 301-594-5032.
    Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering a funding opportunity titled "Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders" (PAR-25-327), aimed at supporting Investigational New Drug (IND)-enabling studies and preparations for First-in-Human (FIH) clinical trials. This initiative focuses on advancing innovative gene-based therapies, such as oligonucleotides and viral vector approaches, for ultra-rare disorders affecting fewer than 6,000 patients, with an emphasis on projects that demonstrate robust proof of concept data and readiness for clinical trials. Eligible applicants include a wide range of organizations, including higher education institutions and various nonprofit and for-profit entities, with applications requiring detailed project planning and compliance with Good Manufacturing Practices (GMP). The application period opens on January 9, 2025, with a close date of October 8, 2027, and interested parties can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries.