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 Modernization Plan
On June 29, 2017 , the U.S. Food and Drug Administration unveiled a strategic plan to completely eliminate the agency’s existing orphan designation backlog and ensure continued timely response to all new requests for designation with firm deadlines. The agency’s Orphan Drug Modernization Plan comes a week after FDA Commissioner Scott Gottlieb committed to eliminating the backlog within 90 days and respond to all new applications within 90 days of receipt during his testimony before a Senate subcommittee.
- FDA's Orphan Drug Modernization Plan
- September 12, 2017 Update to Orphan Drug Modernization Plan
- FDA Orphan Drug Products Policy Council Charter (SMG 2010.19)
- Guidance for Industry, Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases
Clinical Superiority Findings
In accordance with section 527(e)(2) of the FD&C Act (21 U.S.C. 360cc(e)(2)), FDA will publish a summary of the clinical superiority findings when a drug is granted exclusive approval or licensure on the basis of a demonstration of clinical superiority here: