Funding Opportunities for Rare Diseases at FDA

To help support product development, FDA funds research.  Some of these research opportunities are relevant to rare disease product development. Please note that not all opportunities are currently available.  A list of current and open FDA rare disease funding opportunities can be found on the NIH Grants and Funding page. A list of programs that are relevant to rare disease research is located below:

• Office of Orphan Products Development (OOPD)
  OOPD has several funding opportunities that help promote the development of drugs, devices, biologics, and medical foods for patients with rare diseases and special populations.
  • The Clinical Trials Grants Program funds clinical trials 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 program encourages innovative and efficient clinical trial methods such as adaptive and seamless trial designs, modeling and simulations, and basket and umbrella trials. 
  • The Natural History Studies Grant Program supports efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs.  These studies can help at every stage of product development, such as identifying the patient population, identifying or developing clinical outcome assessments and biomarkers, and when appropriate, serving as external controls. This program is intended to fund well-designed, protocol-driven natural history studies with high quality and interpretable data elements that address knowledge gaps, support clinical trials and advance rare disease medical product development.
  • The Rare Neurodegenerative Disease Grant Program awards grants and contracts to cover costs of research and development of interventions intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases in adults and children. FDA continues to develop grant and contract opportunities to develop tools, methods, and processes to characterize the natural history of rare neurodegenerative diseases, identify molecular targets for these diseases and to increase efficiency and productivity of clinical development of therapies.
  • The Pediatric Device Consortia Grants Program provides funding to develop nonprofit consortia to facilitate pediatric medical device development. These nonprofit consortia provide a platform of experienced regulatory, business planning, and device development services such as intellectual property advising, prototyping, engineering, laboratory and animal testing, and grant-writing, to foster advancement of medical devices for pediatric patients. In addition to key advising services, the consortia also provide critical funding to innovators to directly advance these projects.
• Office of New Drugs
  • Drug Development Tools (DDT) Research Grants Program supports research activities that enable progress in drug development tool qualification.  Drug Development Tools (DDTs) are methods, materials, or measures that have the potential to facilitate drug development such as biomarkers, clinical outcome assessments (COAs), animal models, and other innovative tools under the Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program.
• Oncology Center of Excellence (OCE)
  • The OCE provides research funding through various extramural programs. They recently announced the Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers opportunity to fund research to develop approaches to support drug development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers. The program focuses on supporting studies to improve therapeutic development in ultra-rare cancers with high unmet medical need where there is little economic incentive for commercial entities to conduct the research.
• Office of Regulatory Science and Innovation
  • The FDA Broad Agency Announcement (BAA) solicits for advanced research and development proposals to support regulatory science and innovation. Please see FDA BAA Questions & Answers and last year’s BAA Day website for additional information on the BAA program.
  • The Centers of Excellence in Regulatory Science and Innovation (CERSI) Program fosters robust and innovative approaches to advance regulatory science, through collaborative interactions with FDA scientific experts and funding offices.  FDA currently has cooperative agreements with the University of Maryland,  University of California at San Francisco (UCSF) in a joint effort with Stanford University (UCSF-Stanford), Johns Hopkins University, Yale University in joint effort with Mayo Clinic, and Triangle CERSI (a joint effort of Duke University and University of North Carolina at Chapel Hill, North Carolina Central University, and North Carolina State University)
• Office of Minority Health and Health Equity (OMHHE)
  • OMHHE’s Enhance Equity Initiative Funding Opportunities enable OMHHE to advance minority health and health equity focused research by engaging across a broad range of stakeholders, including academia, minority-serving institutions, government agencies, and non-profit organizations.
• Digital Health Technologies for Drug Development
  • The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) support research to address topics related to the use of digital health technologies (DHTs) for remote data acquisition in clinical investigations for drug development. The goal of the current funding opportunity is to explore the role of DHTs (e.g., actigraphy, photography, environmental sensors) in the evaluation of new drugs. The objectives of these projects are to advance DHTs for clinical drug development, expand the ability to capture early manifestations of chronic diseases, determine outcomes in populations with unmet medical needs and enhance convenience for trial participants by allowing for remote data acquisition in clinical investigations.
• Oak Ridge Institute for Science and Education (ORISE)
  • The ORISE Research Fellowship program at the U.S. Food and Drug Administration (FDA) is an educational and training program designed to provide college students, recent graduates, postdocs, and university faculty opportunities to connect with the unique resources of the FDA.