Office of Orphan Products Development
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare disease and to further advance scientific development of such promising medical products. The office also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.
OOPD provides incentives for sponsors to develop products for rare diseases. The program has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983. In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983. The Orphan Grants Program has been used to bring more than 45 products to marketing approval. The Humanitarian Use Device Program has been the first step in approval of more than 50 Humanitarian Device Exemption approvals.
The Orphan Drug Designation program
provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
The Humanitarian Use Device (HUD) program
designates a device that is intended to benefit patients by treating or diagnosing a disease or condition that affects fewer than 4,000 individuals in the United States per year as per 21 CFR 814.3(n).
The OOPD administers two extramural grants programs. The Orphan Products Grants Program
provides funding for clinical research that tests the safety and efficacy of drugs, biologics, medical devices and medical foods in rare diseases or conditions. The Pediatric Device Consortia (PDC) Grants Program
provides funding to develop nonprofit consortia to facilitate pediatric medical device development.
For more information on any of our programs, click here
to visit the programs’ web pages.
Director for the Office of Orphan Products Development:
Gayatri R. Rao, M.D., J.D., Director for the Office of Orphan Products Development (OOPD) received her M.D. from the University of Medicine & Dentistry of New Jersey - New Jersey Medical School. Upon graduation from medical school, she went on to earn a joint J.D. from the University of Pennsylvania Law School, where she concentrated on healthcare and FDA related issues, and a masters in bioethics from the University of Pennsylvania School of Medicine. Following law school, she worked for an international law firm, in Washington, D.C., focusing primarily on food and drug law and other healthcare related matters, including matters related to orphan products. She then joined the FDA’s Office of the Chief Counsel where she provided advice on a wide range of issues related to medical devices, combination products, clinical trials, and human subject protection. She has brought her unique medical, legal/regulatory, and bioethical background to help move the OOPD rare disease mission forward.