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        Medical products for rare diseases and conditions

Designating an Orphan Product: Drugs and Biological Products

                        
                Orphan Drug Designation Request Form
          Orphan Drug Act - Relevant Excerpts
          Frequently Asked Questions (FAQ) About Designating an Orphan Product
          Orphan Drug Designation: Disease Considerations
          Clinical Superiority Findings
          Instructions for Searchable Designation Database
          Public Identification of Orphan Drug Designation
          Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases; FDA Report to Congress
          Contact Us Home
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          Medical products for rare diseases and conditions
          Designating an Orphan Product: Drugs and Biological ProductsMedical products for rare diseases and conditions Designating an Orphan Product: Drugs and Biological Products
  	
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    PrintSupporting 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 approvalSponsors 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 orphan@fda.hhs.gov
	By mailing the required information to:
	Office of Orphan Products Development
	Attention: Orphan Drug Designation Program
	Food and Drug Administration
	WO32-5295
	10903 New Hampshire Avenue
	Silver Spring, MD 20993-0002Portal for submitting orphan drug designation requests

New users of the CDER NextGen Portal must register for an account. See the frequently asked questions for more information or contact edmsupport@fda.hhs.gov 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

                                                                                  Content current as of:
      07/08/2022
                  
                   
Regulated Product(s)
		 DrugsLaw(s) & Regulation(s)
			  Orphan Drug ActDesignating an Orphan Product: Drugs and Biological Products                
                          

                        Orphan Drug Designation Request Form
          Orphan Drug Act - Relevant Excerpts
          Frequently Asked Questions (FAQ) About Designating an Orphan Product
          Orphan Drug Designation: Disease Considerations
          Clinical Superiority Findings
          Instructions for Searchable Designation Database
          Public Identification of Orphan Drug Designation
          Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases; FDA Report to Congress
          Contact Us
 
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