Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)

The purpose of this Notice of Funding Opportunity (NOFO) is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. The goal of this NOFO is to support clinical trials of orphan products in all phases of product development for rare diseases with unmet medical needs. Key aspects of the project include: • Clinical trials should evaluate safety and/or efficacy of medical products in support of a new indication or a change in labeling. • The use of shared, established infrastructure and resources and collaborative efforts between stakeholders in industry, academia, and patient organizations are highly encouraged. • Patients living with a rare disease, or their caregivers, are encouraged to contribute to important considerations in product development. • Innovative and efficient trial approaches, such as seamless and adaptive trial designs, are allowed for additional funding with justification. The project duration can be up to 4 years, with a maximum budget of $650,000 per year, and potentially up to $900,000 per year with additional funding for innovative approaches. Some key points to consider are: • Eligible organizations include higher education institutions, nonprofits, for-profit organizations, local governments, and others. • Required registrations include System for Award Management (SAM), eRA Commons, and others. • The project requires data and safety monitoring procedures in place to safeguard the well-being of study participants and to ensure scientific integrity.