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  1. Developing Products for Rare Diseases & Conditions

Designating an Orphan Product: Drugs and Biological Products

New! Online Portal for Submission of Orphan Drug Designation Requests

The Office of Orphan Products Development (OOPD) announces the availability of an online Portal for submission of new orphan drug designation requests.

What are the benefits of submitting requests via the CDER NextGen Portal?

  • Ease of use: Requesting via the Portal is easy, and all information regarding your requests is stored in one place.
  • Automated confirmation: You can log in, initiate a request and receive an automated confirmation of your transaction.
  • Historical record: Requestor can view all transactions and check the status of any submission at any time.

How to Gain Access?

  • New Users - To register for an account with the CDER NextGen Portal, navigate to https://edm.fda.gov and follow the signup instructions.
  • Existing Portal Users - The ability to submit an Orphan Drug Designation was added to your account automatically – click on “Orphan Drugs” when you are ready to submit a request.

Need Support?

In order to get assistance with the CDER NextGen Portal, please reference the user guides and FAQ's. For additional support, please contact the CDER Platform Support Team at [email protected].

Note: If you submitted a request via portal, do not re-submit via e-mail.

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 [email protected]. 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 [email protected]. 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 Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes “orphan status”). For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA’s implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

A sponsor seeking orphan designation for a drug must submit a request for designation to OOPD with the information required in 21 CFR 316.20 and 316.21. Each designation request must stand on its own merit. Sponsors requesting designation of the same drug for the same rare disease or condition as a previously designated product must submit their own data and information in support of their designation request. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.

Orphan Drug Designations and Approvals

https://www.accessdata.fda.gov/scripts/opdlisting/oopd/

 



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