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    v1.0.28
    OpenPayerPolicy UpdatesPharmacy & Specialty DrugsBlue Cross Blue Shield Kansas off-label drug Cover…
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    Currentblue cross blue shield - kansasPolicy N/A

    Off-Label, Orphan, and Expanded Access Drug Use — Coverage Criteria

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    Policy governing coverage criteria for off-label use of FDA-approved drugs, orphan drugs, and expanded access (compassionate use) investigational drugs for BCBS Kansas members and administered providers.

    Policy Summary
    Payerblue cross blue shield - kansas
    PolicyOff-Label, Orphan, and Expanded Access Drug Use — Coverage Criteria
    Policy CodePolicy N/A
    Change TypeNo material changes
    Effective DateDec 7, 2015
    Last ReviewNov 20, 2024
    Next Review DateN/A
    Key ActionWhen requesting coverage for an off-label cancer drug use, provide documentation supporting the proposed use if requested by the insurer.
    SourceLink
    POLICY UPDATE CHANGES

    No material clinical or coverage changes in this revision.

    state-mandatedState mandate for cancer drugs
    FDA approval requiredOff-label non-cancer requirement

    OpenPayer is powered by Trek Health's payer performance platform. Trek continuously ingests, validates, and normalizes Transparency in Coverage data alongside payer policies and other commercial payer data to create a structured payer intelligence foundation. OpenPayer uses this foundation to deliver personalized search results, dynamically generated policy pages, and tailored policy monitoring based on each user's payers, specialties, billing codes, and areas of interest. The same intelligence powers broader payer performance workflows, including reimbursement benchmarking, contract evaluation, payer negotiations, and financial decision-making.

    Orphan + approval
    Orphan drug requirement
    ExperimentalExpanded access stance

    Coverage Criteria

    Coverage criteria

    Overall policy: Coverage depends on drug type and supporting evidence/approval status; see subnodes.
    Off-label cancer drug use: Per Kansas statute, off-label use of an FDA-approved prescription drug for cancer treatment is covered when the use is recognized in a standard reference compendium or in substantially accepted peer-reviewed medical literature; the prescribing physician shall submit documentation supporting the proposed off-label use if requested by the insurer.

    Supported by Kansas Statute 40-2,168.

    Off-label non-cancer drug use (medically necessary): Considered medically necessary when ONE of the following is met:

    Condition 1 for non-cancer off-label use

    • a.i: The drug is approved by the U.S. Food and Drug Administration.
    • a.ii: The prescribed use is supported by compendia (NCCN level 1/2A/2B, AHFS, DrugDex, Clinical Pharmacology) or the prescriber has submitted additional documentation supporting the requested use.
    Condition 2 for non-cancer off-label use: Sufficient evidence from published studies in major peer-reviewed journals that support the proposed use for the specific condition as safe and effective (accepted study designs include, but are not limited to, randomized double-blind, placebo-controlled clinical trials).
    Orphan drug use: Medically necessary only when the product has FDA orphan drug designation AND has received FDA marketing approval; orphan designation alone without marketing approval is considered not medically necessary.
    Expanded access (Compassionate Use): Expanded access (e.g., single-patient IND approved for compassionate use) is considered experimental/investigational but may be covered if the Research Urgent or Off-Label Drug use requirements are met and coverage is allowed by the member's contract benefits.

    Refer to member contract benefits to determine coverage for an individual member.

    Experimental or investigational chemotherapy drugs that do not have FDA marketing approval are not covered. Per the policy, such agents should be denied as experimental/investigational when used for cancer treatment.

    A product that has only received FDA orphan drug designation but does not have FDA marketing approval is considered not medically necessary and is not covered. Coverage requires both orphan designation and marketing approval.

    Provider Actions & Requirements

    Prior Authorization

    Prior authorization: evidence required for off‑label non‑cancer use

    Off‑label non‑cancer drug use requires either: (a) the drug is FDA‑approved and the requested indication is supported by a recognized compendium (NCCN level 1/2A/2B, AHFS, DrugDex, Clinical Pharmacology) or the prescriber submits additional documentation supporting the requested therapeutic use; OR (b) sufficient evidence from published peer‑reviewed studies (for example randomized, double‑blind, placebo‑controlled trials) demonstrates the proposed use is safe and effective. Prior authorization will require submission of the supporting compendia citations or published evidence as specified above.

    • Compendia support: NCCN Compendium (levels 1, 2A, 2B), AHFS, DrugDex, Clinical Pharmacology.
    • Acceptable peer‑reviewed evidence includes randomized, double‑blind, placebo‑controlled clinical trials.
    Note

    Provider action for expanded access / investigational requests

    If a single‑patient expanded access (compassionate use) or other investigational request is submitted, the provider must follow the policy's Research Urgent / Off‑Label documentation requirements and verify member contract benefits; submit any FDA compassionate use approval and requested supporting documentation as part of authorization.

    Background

    Off-label drug use refers to prescribing an FDA-approved drug for an indication that is not included in the approved labeling. For cancer drugs, Kansas statute requires coverage when the off-label use is recognized in standard reference compendia or in substantially accepted peer-reviewed medical literature, and the insurer may request supporting documentation from the prescribing physician. For non-cancer off-label uses, coverage is limited to situations where either the drug is FDA-approved and the indication is supported by accepted compendia or the prescriber provides documentation, or there is sufficient evidence from peer-reviewed published studies demonstrating safety and effectiveness. Orphan-designated products and investigational expanded-access uses have separate requirements and do not automatically meet coverage criteria.

    Definitions

    Off-label drug use
    DefinitionUse of an FDA-approved drug for an indication not included in the approved labeling (also called 'unlabeled' use).
    ContextFDA approves drugs for specific indications; uses outside those indications are termed off-label even when widely used and supported by literature.
    Documentation noteMany off-label uses are effective and well documented in the literature; coverage may require supporting evidence per policy requirements.
    Orphan drug
    DefinitionA product intended to treat a rare disease (e.g., affecting fewer than 200,000 Americans) that may receive FDA orphan designation.
    Regulatory pathwayOrphan designation can be obtained prior to submission of a marketing application; safety and efficacy must be established through clinical studies for marketing approval.
    Policy Summary
    Payerblue cross blue shield - kansas
    PolicyOff-Label, Orphan, and Expanded Access Drug Use — Coverage Criteria
    Policy CodePolicy N/A
    Change TypeNo material changes
    Effective DateDec 7, 2015
    Last ReviewNov 20, 2024
    Next Review DateN/A
    Key ActionWhen requesting coverage for an off-label cancer drug use, provide documentation supporting the proposed use if requested by the insurer.
    SourceLink
    On This Page
    Coverage CriteriaProvider Actions & RequirementsBackgroundDefinitions
    • Expanded access requests are considered experimental/investigational but may be covered if Research Urgent or Off‑Label Drug use requirements are met.
    • Confirm coverage by referring to the member's contract benefits at time of service.
    Documentation Required

    Documentation required for off‑label cancer use (physician to provide if requested)

    For off‑label cancer drug use, the prescribing physician must submit documentation supporting the proposed off‑label use or uses if requested by the insurer, consistent with Kansas statute.

    • Documentation should demonstrate the use is recognized in a standard reference compendium or is supported by substantially accepted peer‑reviewed medical literature, per the statute cited in policy.
    Denial Risk

    Denial triggers: investigational chemotherapy and orphan‑only products

    Requests for chemotherapy or other drugs that are investigational (not FDA‑approved) will be denied as experimental/investigational; likewise, products with only orphan drug designation but without FDA marketing approval are considered not medically necessary and will be denied.

    • Experimental/investigational chemotherapy drugs that are not FDA approved: deny as experimental/investigational.
    • Orphan designation alone without FDA marketing approval is considered not medically necessary.
    Coverage implicationOrphan designation alone does not equal marketing approval; FDA marketing approval is required for the product to be considered medically necessary under this policy.
    Expanded access / compassionate use
    DefinitionUse of an investigational new drug (IND) outside a clinical trial for patients with serious or life‑threatening conditions who are ineligible for the trial (also called expanded access or compassionate use).
    Eligibility conditionsAvailable when patients may benefit, therapy can be given safely outside the trial, no alternative therapy is available, and the drug developer agrees to provide access.
    Program typesIncludes expanded access programs (EAPs) and single‑patient INDs (emergency or compassionate use requests submitted by a physician).