Orphan Drug Regulations 21 CFR Part 316

[Docket No. FDA–2011–N–0583]

Federal Register / Vol. 78, No. 113 / Wednesday, June 12, 2013

RIN 0910–AG72

AGENCY: Food and Drug Administration, HHS.

ACTION: Final Rule

SUMMARY: The Food and Drug Administration (FDA) is issuing final regulations amending the 1992 Orphan Drug Regulations issued to implement the Orphan Drug Act. These amendments are intended to clarify regulatory provisions and make minor improvements to address issues that have arisen since those regulations were issued.

Download “Orphan Drug Final Rule” 2013-13930.pdf – Downloaded 846 times – 294 KB

Draft Guidance for Industry, Researchers, Patient Groups, and Food and Drug Administration Staff 

Meetings with the Office of Orphan Products Development


This guidance document is being distributed for comment purposes only.

Document issued on: April 9, 2014

This guidance provides recommendations to industry, researchers, patient groups, and other stakeholders (collectively referred to in this guidance as “stakeholders”) interested in requesting a meeting, including a teleconference, with the Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD) on issues related to orphan-drug designation requests, humanitarian use device (HUD) designation requests, rare pediatric disease designation requests, funding opportunities through the Orphan Products Grants Program and the Pediatric Device Consortia Grants Program, and orphan product patient related topics of concern. It is also intended to assist OOPD staff in addressing such meeting requests. This guidance describes procedures for requesting, preparing, scheduling, conducting, and documenting such meetings.

Download “Meetings with the Office of Orphan Products Development” UCM392593.pdf – Downloaded 409 times – 92 KB

Designating an Orphan Product: Drugs and Biological Products

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.

Searchable Databases

Searchable database for Orphan Designated and or Approved Products. Note list will be updated monthly.