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  1. Office of Clinical Policy and Programs

Office of Orphan Products Development

During this public health emergency associated with the COVID-19 pandemic, the Office of Orphan Products Development (OOPD) is providing sponsors with increased flexibility for submission of orphan drug designation requests and related submissions (amendments, annual reports, etc.). During this public health emergency, orphan drug designation, humanitarian use device designation, and rare pediatric disease designation requests and submissions may be submitted electronically by email to the OOPD.

When transmitting information to the Orphan Drug Designation Program via email, please utilize the mailbox orphan@fda.hhs.gov. The use of automated read receipt is recommended to avoid the need to call to verify receipt of the email. Sponsors and others who plan to email information to FDA that is considered to be private, sensitive, proprietary, or commercial confidential are strongly encouraged to send it from an FDA secured email address so the transmission is encrypted. The OOPD will assume that the addresses of emails received or email addresses provided as a point of contact are FDA secure when responding to those email addresses.

Sponsors and others can establish a secure email address link to FDA by sending a request to SecureEmail@fda.hhs.gov. There may be a fee to a commercial enterprise for establishing a digital certificate as part of the set-up process before emails can be sent to FDA encrypted.

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 trials and natural history studies that advance rare disease medical product development. The Pediatric Device Consortia (PDC) Grants Program provides funding to develop nonprofit consortia to facilitate pediatric medical device development.

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