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U.S. Department of Health and Human Services

For Industry

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Orphan Drug Regulations: Regulatory History


On June 12, 2013, the Food and Drug Administration (FDA) issued a final rule (78 FR 35117) amending the 1992 Orphan Drug Regulations, which implement the Orphan Drug Act (ODA, Relevant Excerpts: Public Law 97-414, as amended).  This final rule is effective as of August 12, 2013 and is codified at 21 CFR part 316.  

These amendments are intended to clarify certain regulatory language and propose areas of minor improvement regarding orphan-drug designation and orphan-drug exclusivity.  FDA believes these revisions clarify, streamline, and improve the orphan-drug designation process. 

These amendments are fully consistent with the Orphan Drug Act (ODA, Relevant Excerpts: Public Law 97-414) and continue to provide incentives for the development of potentially promising orphan drugs that may not otherwise be developed and approved, including drugs that are potentially safer or more effective than already approved drugs.


To view the Orphan Drug Final Rule of June 12, 2013, click here.

To view the Orphan Drug Proposed Rule of October 19, 2011, click here.


To view the Orphan Drug Final Rule of December 29, 1992, click here .

To view the Orphan Drug Proposed Rule of January 29, 1991, click here.


If you have questions about the 2013 Final Rule and this regulatory history, please contact:

 Ms. Erica K. McNeilly, 301-796-8679 erica.mcneilly@fda.hhs.gov