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  5. Frequently Asked Questions (FAQ) About Designating an Orphan Product
  1. Designating an Orphan Product: Drugs and Biological Products

Frequently Asked Questions (FAQ) About Designating an Orphan Product

Frequently Asked Questions

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.

Designation is given to a drug/biologic for the treatment, diagnosis or prevention of a rare disease or condition, not to proposed drug indication or how a sponsor may wish to study a drug.


Additional Resources:

 

For any other questions not listed here, please email orphan@fda.hhs.gov