NEW - Preclinical Proof of Concept Studies for Rare Diseases
RFA-TR-23-016: Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
This notice of funding opportunity (NOFO) provides funding to conduct efficacy studies in an established rare disease preclinical model to demonstrate that a proposed therapeutic agent warrants further development. In addition to preclinical efficacy, accompanying pharmacodynamic and pharmacokinetic studies would be supported. Therapeutic agents include small molecules, biologics or biotechnology-derived products. The goal of this NOFO is to spur therapeutic development for rare diseases by advancing projects to the point where they would attract subsequent investment supporting full Investigational New Drug (IND) application development or progression to clinical trials in the case of repurposing or repositioning.
The NIH supports research on a broad range of diseases that are defined as rare; that is, diseases affecting fewer than 200,000 individuals in the United States. While individually, each rare disease affects a limited number of individuals, when viewed collectively, rare diseases represent a significant unmet medical need. Approximately 30 million people in the United States are living with one of the estimated 10,000 rare diseases; many of which affect children and are serious or life-threatening. Despite advances in our understanding of the mechanisms of many rare diseases, and large investments by pharmaceutical and biotech industries, effective treatments are available for fewer than 5% of diseases. This NOFO solicits applications that propose to conduct efficacy studies in an established rare disease preclinical model to demonstrate proof of concept that a proposed therapeutic agent warrants further development. In addition to preclinical efficacy, accompanying pharmacodynamic (PD) and pharmacokinetic (PK) studies would be supported.