Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)

The purpose of this opportunity 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. Through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development. The key aspects of this opportunity include: • Funding for 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 • Increased number of approved treatments for rare diseases • Broad and positive impact on rare disease drug development • Collaborative, efficient, and/or innovative clinical trials • Eligibility for various types of organizations, including higher education institutions, nonprofits, for-profit organizations, and local governments