Off‑Label Use of Medications and Other Interventions
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Defines when off-label use of FDA‑approved drugs, biologics, devices, or other interventions may be considered medically necessary and the evidence sources Capital BlueCross will accept; applies to members and providers covered by applicable Capital Blue Cross programs.
No material clinical or coverage changes in this revision.
Off‑Label Use Coverage Criteria
Off‑Label Use — Medical Necessity Criteria
Off‑label use may be considered medically necessary when ALL of the following are met:
If the FDA or the evidence sources listed in this policy determine the use is contraindicated or not indicated/not recommended for specific conditions or patient groups, that use is considered investigational and excluded from coverage.
If the FDA has determined a drug, biologic, or device to be contraindicated for a proposed use, that use is excluded from coverage and is considered investigational. The policy also treats uses as investigational when the evidence sources listed in this policy (e.g., accepted standards of practice, national specialty society guidelines, peer‑reviewed literature, DrugDex/AHFS, NCCN, Clinical Pharmacology, Lexi‑Drugs rated Use: Off‑Label Evidence Level A, ICER, or Blue Cross Blue Shield Association Evidence Street) deem the use to be not indicated or not recommended for specific conditions or defined patient groups.
Products that have not received FDA approval for any indication are considered investigational for all uses and are excluded from coverage under this policy.
Off‑label uses that lack support from at least one of the policy’s accepted evidence sources — or that are specifically contraindicated by the FDA or by the listed compendia/guidance sources — are considered not medically necessary/investigational and will not be covered.
Procedure and Code Guidance
| No codes listed |
Provider Requirements and Authorization
Investigational or Contraindicated Uses
Investigational or contraindicated uses: Requests for uses that the FDA has determined to be contraindicated, or uses that the compendia/sources listed in this policy deem not indicated or not recommended for specific conditions or defined patient groups, will be considered investigational and are not medically necessary.
- Includes any request for a drug, biologic, or device that has not received FDA approval for any indication
- Includes off-label uses specifically contraindicated by the FDA or judged not indicated by the authoritative sources referenced in this policy
Required Supporting Documentation
Required supporting documentation: Provider must document FDA approval status and supply supporting evidence from accepted sources to justify off-label use.
- Documentation of the FDA-approved indication(s) and statement of the proposed off-label indication
- One or more supportive references from accepted sources (examples listed in policy): national specialty society practice guidelines, peer-reviewed medical literature, or recognized compendia (e.g., Thomson Micromedex DrugDex, AHFS-DI, NCCN Drugs and Biologics Compendium, Clinical Pharmacology, Lexi‑Drugs rated Evidence Level A, ICER, BCBS Association Evidence Street)
- If applicable, documentation that no FDA contraindication exists for the proposed use
- When expert medical opinion is used, include the consultant's credentials and rationale
Stage IV Cancer Step Therapy Cross-Reference
Stage IV cancer step therapy cross-reference: For patients with late-stage metastatic disease (Stage IV), follow the Capital BlueCross policy MP 2.373 for additional guidance on step therapy and treatment requirements.
- See MP 2.373: Step Therapy Treatment in Cancer, Including Treatments for Stage Four, Advanced Metastatic Cancer, and Severe Related Health Conditions
Background
FDA approvals are indication‑specific; using a drug, biologic, or device outside its labeled indications is considered off‑label use. While many off‑label uses may be supported by evidence, this policy requires the product to be FDA‑approved and the proposed off‑label use to be supported by at least one accepted evidence source (for example, accepted standards of medical practice, expert opinion identified by Capital, national specialty society practice guidelines, peer‑reviewed articles, DrugDex/AHFS, NCCN, Clinical Pharmacology, Lexi‑Drugs rated Evidence Level A, ICER, or Evidence Street) and to have no specific contraindications for the member in order to be considered medically necessary.
Key Definitions and Compendia
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