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  1. Office of Clinical Policy and Programs

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.

For more information on any of OOPD programs, visit Developing Products for Rare Diseases and Conditions 

OOPD provides incentives for sponsors to develop products for rare diseases. The program has successfully enabled the development and marketing of drugs, biologics, and medical devices for rare diseases since 1983.

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 medical devices that are intended to benefit patients in the treatment or diagnosing a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year as eligible for the Humanitarian Device Exemption.

The Rare Pediatric Disease Priority Review Voucher Program says that a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

The OOPD administers 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. The newest grant program, the Orphan Products Natural History Grants Program will support studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics.