Orphan Drugs

Pursuant to Section 340B(e) of the Public Health Service Act (PHSA), rural referral centers, sole community hospitals, critical access hospitals, and free-standing cancer hospitals participating in the 340B Program, the term "covered outpatient drug" doesn't 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 aren't 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.

Orphan drug designation list

Stakeholders are encouraged to access the most up-to-date information that is publicly available on the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) database to determine the orphan drug status of covered outpatient drugs.

Covered entities, working with their partners, may need to conduct additional analyses of the drugs provided on the OOPD list to determine the appropriate orphan drugs to exclude from the 340B Program and the appropriate time frame. HRSA encourages 340B stakeholders to work, in good faith, to resolve any potential disputes regarding access to orphan drugs.

The following outlines how to create a list of currently designated orphan drug products from the OOPD database:

  1. Navigate to: FDA OOPD Database
  2. Enter in the Search Results field: All designations
  3. Enter in the Output Format: Download Excel File
  4. Filter the Orphan Designation Status Column to include “Designated” and “Designated/Approved.”

Program integrity

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

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