In July 2014, HRSA issued an interpretive rule pertaining to the statutory requirement for inclusion of drugs with orphan drug designations in the 340B drug pricing program. The interpretive rule outlines the agency’s interpretation that the 340B statute requires manufacturers to offer entities newly eligible for 340B under the Affordable Care Act 340B pricing on drugs that have received an orphan designation when those drugs are used for their non-orphan indication.
HRSA posts updated Orphan Drug Designation Lists, found below, as well as the Orphan Selection File, found on the 340B database, in order to assist all 340B stakeholders in complying with HRSA's policy. 340B hospitals subject to the orphan drug exclusion (critical access hospitals, free-standing cancer hospitals, sole community hospitals and rural referral centers) are responsible for ensuring that any orphan drugs purchased through the 340B Program are not transferred, prescribed, sold, or otherwise used for the rare condition or disease for which the orphan drugs are designated under section 526 of the Federal Food, Drug, and Cosmetic Act.
HRSA also makes available a list of manufacturers that are not offering 340B pricing for their designated orphan products (when used for their non-orphan indications) to newly eligible covered entities.
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, 2015 (see "About the Orphan Drug List").
Orphan Drug List Governing April 1 - June 30, 2015 (PDF - 1 MB)
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:
The 340B program database allows drug manufacturers and wholesalers to identify affected hospitals that will purchase orphan drugs under the 340B Program and will maintain auditable records to demonstrate compliance with the orphan drug exclusion (Orphan Drug Participation = Yes), or that cannot or do not wish to maintain auditable records regarding compliance with the orphan drug exclusion and will purchase all orphan drugs outside of the 340B Program regardless of the indication for which the drug is used (Orphan Drug Participation = No).
The database takes a snapshot of affected hospitals’ elections at 12:01am ET on the 15th day of the month prior to the start of each calendar quarter, irrespective of weekends or holidays. Covered entities may request changes to their election at any time but changes take effect quarterly and only then if approved by OPA before the time of the snapshot.
To access current or past quarters’ files, follow the ‘Download Orphan Drug Selection File’ link from the database home page.
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