Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant orphan-drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including:
- Tax credits for qualified clinical trials
- Exemption from user fees
- Potential seven years of market exclusivity after approval
Sponsors seeking orphan drug designation for a drug must submit a request for designation to the agency. 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 to support their designation request. Orphan drug designation is a separate process from seeking approval or licensing. Drugs for rare diseases go through the same rigorous scientific review process as any other drug for approval or licensing.
See the orphan drug designations and approvals database for more information.
How to submit orphan drug designation requests
Sponsors may submit orphan drug designation requests one of three ways:
- Through the CDER NextGen portal
- By emailing the required information to email@example.com
- By mailing the required information to:
Office of Orphan Products Development
Attention: Orphan Drug [or Rare Pediatric Disease] Designation Program
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
New users of the CDER NextGen Portal must register for an account. See the frequently asked questions for more information or contact firstname.lastname@example.org with other questions. Users who submitted a request through the CDER NextGen portal do not need to submit by email. For designation requests submitted by email, the agency recommends using automated read receipt to verify receipt of the email.
Sponsors and others who plan to email information to FDA that is private, sensitive, proprietary or commercial confidential are strongly encouraged to send it from an FDA-secured email address so the transmission is encrypted. The agency will assume the addresses of emails received or email addresses provided as a point of contact are 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 before encrypted emails can be sent to FDA.
Resources for you
- Webinar: Recommended Tips for Creating an Orphan Drug Designation Application (slides and script)
- Orphan Drug Final Rule of June 12, 2013
- Orphan Drug Final Rule of December 29, 1992
- 21 Code Federal Regulations Part 316 - Orphan Drugs
- Public Law 97-414
- Summary of H.R. 5238 - 97th Congress (1981-1982) Orphan Drug Act