Orphan Drugs and Humanitarian Use Device (HUD)
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Covers medical necessity criteria and coverage considerations for FDA-designated orphan drugs and Humanitarian Use Devices when no other Capital BlueCross medical policy applies; affects providers submitting requests for these products under applicable Capital BlueCross benefit programs.
No material clinical or coverage changes in this revision.
Coverage Criteria
inv-01: Orphan Drug — Covered when ALL of the following are met:
Orphan Drug — Covered when ALL of the following are met:
All conditions required
inv-02: Humanitarian Use Device (HUD) — Covered when ALL of the following are met:
Humanitarian Use Device (HUD) — Covered when ALL of the following are met:
All conditions required
All other uses of a drug designated as an orphan product or a device designated as a Humanitarian Use Device (HUD) that do not meet the specific coverage criteria listed above are considered investigational.
Uses that are not consistent with FDA‑approved indications or that are not supported by peer‑reviewed published medical literature or other appropriately recognized resources (for example, NCCN, AHFS, Micromedex, ICER) are considered investigational and not medically necessary under this policy.
Initial Therapy Criteria
inv-15: Orphan drug initial coverage requirements
Orphan drug initial coverage requirements
See coverage criteria above
Coding and Definitions
| No codes listed |
Provider Actions and Requirements
Prior Authorization Required
Prior authorization is required when coverage is requested for an orphan drug or a Humanitarian Use Device (HUD). Requests must document the drug or device's FDA designation/approval and demonstrate that the proposed use is consistent with FDA-approved indications and supporting clinical evidence.
- Applies to requests for orphan drugs and HUDs
- Include FDA designation/approval documentation
Triggers for Denial
Requests may be denied if the orphan drug or HUD lacks FDA designation or marketing approval, if use is inconsistent with FDA‑approved indications, or if supporting clinical evidence and authoritative references (e.g., peer‑reviewed literature, NCCN, AHFS, Micromedex, ICER) are not provided.
- No FDA orphan designation or HDE/marketing approval
- Use inconsistent with FDA‑approved indications
- Insufficient supporting clinical evidence or authoritative references
Required Documentation
For HUDs, include documentation of Institutional Review Board (IRB) oversight/approval and evidence that use is consistent with FDA‑approved indications. For orphan drugs include FDA Orphan Drug designation and FDA marketing approval documentation. In all cases submit supporting clinical evidence and/or citations to recognized resources (e.g., peer‑reviewed literature, NCCN, AHFS, Micromedex, ICER).
- HUD: IRB oversight/approval documentation and HDE details
- Orphan drug: FDA Orphan Drug designation listing and FDA marketing approval
- Supporting references: peer‑reviewed literature or recognized drug/device resources
Step Therapy Cross‑Reference
Refer to the related step therapy policy for cancer treatment sequences. This policy does not enumerate step therapy sequences itself; see MP 2.373 Step Therapy Treatment in Cancer for applicable step requirements.
- Cross-reference: MP 2.373 for cancer treatment step therapy requirements
Definitions
Site of Care
Use HUDs only at IRB‑oversight facilities
HUDs must be used only at facilities that have Institutional Review Board (IRB) oversight and approval; include confirmation of the facility’s IRB oversight in the request.
Background
Orphan drugs are intended to prevent, diagnose, or treat diseases or conditions that affect a relatively small population. By FDA definition, an orphan drug targets a disease or condition that affects fewer than 200,000 persons in the United States or a condition where recovery of development costs is unlikely. These products may receive orphan designation from the FDA Office of Orphan Products Development to encourage development for rare diseases; coverage under this policy requires the drug to also have FDA marketing approval and meet the other criteria specified in the policy.
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