21 September 2023

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – FY 2025 to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement 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. Additionally, 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.

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)
Receipt Dates: October 24, 2023; October 22, 2024

FOA Number: RFA-FD-23-001

• Purpose: 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.

• Focus: Efficiency, innovation, impact, collaboration, leveraging patient input, infrastructure and financial resources

• Efficiency: Encourage efficient and innovative clinical trials in all phases of product development through adaptive and seamless trial designs, basket, umbrella and platform trials studying multiple rare diseases/products and the use of data modeling and simulations. Allow additional total costs up to $250,000 per year for studies using efficient and innovative designs.