On October 14, 2015, the U.S. District Court for the District of Columbia vacated HRSA’s Orphan Drug Interpretive Rule. Access the court ruling.
This means that for rural referral centers, sole community hospitals, critical access hospitals, and free-standing cancer hospitals participating in the 340B Program, the term "covered outpatient drug" does not include a drug designated by the Secretary under section 526 of the Federal Food, Drug, and Cosmetic Act for a rare disease or condition. Therefore, manufacturers are not required to provide these covered entities orphan drugs under the 340B Program. A manufacturer may, at its sole discretion, offer discounts on orphan drugs to these hospitals.
The following Orphan Drug Designation List was updated and developed using the methodology referenced below and should be used to govern the quarter April 1 - June 30, 2016 (see About the Orphan Drug List).
Orphan Drug List Governing October 1 - December 31, 2015 (XLS- 288 KB)
Archived Orphan Drug List Governing January 1 – March 31, 2015 (PDF - 1.74 MB)
Archived Orphan Drug List Governing July 1 - September 30, 2014 (PDF - 1,596 KB)
Archived Orphan Drug List Governing April 1 - June 30, 2014 (PDF - 823 KB)
The list is developed by HRSA and updated quarterly. It is based upon specific fields captured from the list of orphan drug designations provided by the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD). The list posted should be the source used by 340B stakeholders to ensure compliance with the orphan drug exclusion. Covered entities may need to conduct additional analyses of the drugs provided on this list to determine the appropriate orphan drugs to exclude from the 340B Program.
HRSA recognizes that orphan drug designation sponsors listed on the FDA orphan drug list may not be the current manufacturer for an orphan drug. The sponsor listed reflects the latest information reported by the sponsor to the FDA OOPD. HRSA encourages 340B stakeholders to work, in good faith, to resolve any potential disputes that may result from the use of this list. HRSA will continue to improve the list that is posted on a quarterly basis to ensure covered entities have the information they need to comply with the orphan drug exclusion.
HRSA uses the following methodology to develop the list:
340B Drug Pricing Program covered entities must ensure program integrity and maintain accurate records documenting compliance with all 340B Program requirements.
Covered entities are subject to audit by manufacturers or the federal government. Failure to comply may make the 340B covered entity liable to manufacturers for refunds of discounts obtained.
Learn more: Program Integrity