Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)

Background
This Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare diseases research by enabling efficient and effective movement of candidate therapeutics or diagnostics towards clinical trials, and to increase their likelihood of success through development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials.

The NIH supports translational and clinical 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. Most are serious or life-threatening, leading to significant morbidity and mortality, and most affect children. Despite advances in our understanding of the causes and mechanisms of many rare diseases, effective treatments are available for fewer than 5%. To address this significant public health concern, the NIH investment into discovery research has contributed to unprecedented opportunities to translate scientific advances into better treatments.

Grant Details
Attaining effective therapies for rare diseases is challenging due to their low prevalence resulting in fewer patients, clinicians, researchers, and resources compared to common diseases. This leads to gaps in our understanding of a rare disease's natural history, and a dearth of suitable biomarkers or clinical outcome measures, or of other components needed to design, conduct, and interpret rigorous clinical trials.

The initiative aims to support studies that address these gaps in understanding of disease natural history and appropriate outcome measures. To optimize clinical trial readiness, there needs to be sufficient understanding of the rare disease to permit design, conduct, and interpretation of rigorous clinical trials. This NOFO describes a specialized type of clinical research that is intended to provide data necessary for the design of future clinical trials. Clinical trial readiness is the state of having validated clinical research tools and knowledge of disease natural history necessary for the design of efficient clinical trials.

Eligibility Requirements
Eligible applicants include higher education institutions, nonprofits with 501(c)(3) IRS status (other than institutions of higher education), for-profit organizations (including small businesses), state governments, county governments, city or township governments, special district governments, Indian/Native American tribal governments (federally recognized), independent school districts, public housing authorities/Indian housing authorities, Native American tribal organizations (other than federally recognized tribal governments), faith-based or community-based organizations, regional organizations. Foreign institutions are not eligible to apply.

Period of Performance
The project period is limited to 2 years.

Grant Value
Applications can request budgets that are up to $275,000 direct costs for the 2-year project period of the award. No more than $200,000 direct costs may be requested in any single year.