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

This contract opportunity aims to provide funding for clinical trials that evaluate the efficacy and safety of products intended for new indications or labeling changes to address unmet medical needs in rare diseases or conditions. The goal is to support the development of treatments for rare diseases by encouraging collaborative, efficient, and innovative clinical trials. By doing so, the FDA seeks to increase the number of approved therapies available for rare diseases, thereby positively influencing the broader landscape of rare disease drug development. Issued by the Food and Drug Administration under the Department of Health and Human Services, this contract is specifically directed toward clinical studies involving orphan products. The solicitation, titled "Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)," was posted in July 2025. Prospective applicants or interested parties can contact Kimberly Pendleton at the FDA for more information. The funding opportunity underscores a strategic effort to enhance treatment options for rare disease populations through rigorous and well-supported clinical research initiatives.