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 firstname.lastname@example.org. 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) was created to advance the development of orphan products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. Rare diseases, as generally defined in the US Orphan Drug Act (ODA), are diseases or conditions with a prevalence of fewer than 200,000 persons in the US. Approximately 30 million Americans are affected by 7,000 known rare diseases but only a few hundred of these rare diseases have approved treatments. The Orphan Products Grants Program is an incentive program that has been supporting clinical trial research since 1983 and has funded clinical trials that have facilitated the marketing approval of 70 products. Lack of natural history data presents a significant unmet need for rare disease product development. To help address this unmet need the program has also committed funding to natural history studies since 2016.
Learn about the background and impact of the Orphan Products Grants Program, including past and currently supported Clinical Trials and Natural History Studies.
See request for applications (RFAs) for grants currently available through OOPD, steps to submit, and resources for applicants and current grantees.
See recent press release, publications, and historical information.
Find contact information for various questions relating to the program.