CurrentBlue Cross Blue Shield - Rhode IslandPolicy N/A

off_label_drug_use_policy

Defines BCBSRI coverage for off-label uses of prescription drugs per Rhode Island General Law Chapter 27-55, including required recognition by standard compendia or medical literature for cancer or disabling or life‑threatening chronic disease; exclusions for unapproved, contraindicated, or experimental drugs; and related dispute/advisory processes.

Policy Summary

PayerBlue Cross Blue Shield - Rhode Island

Policyoff_label_drug_use_policy

Policy CodePolicy N/A

Change Typeclarified

Effective DateJul 12, 1994

Next Review Date

Key ActionPrescribing physician must submit documentation supporting the proposed off-label use if requested by the insurer, referencing standard compendia or at least two peer‑reviewed journal articles as defined by statute.

SourceLink

POLICY UPDATE CHANGES

Policy documents coverage per Rhode Island General Law Chapter 27-55 and states service is covered for all BCBSRI members.

1State statute governing policy

3Primary statutory exclusions

2Medical literature article requirement

Coverage Summary

Rhode Island law (§27-55) mandates coverage of off-label uses of prescription drugs for the treatment of cancer or a disabling or life‑threatening chronic disease when the drug is recognized for that indication in one of the standard reference compendia or in the medical literature. The law also requires that the prescribing physician submit documentation supporting the proposed off‑label use if requested by the insurer. Scope: BCBSRI implements this statute to define coverage for off‑label uses of prescription drugs for the specified conditions, including recognition requirements by compendia or medical literature, exclusions for unapproved/contraindicated/experimental drugs, and processes for advisory review and dispute referral.

Key thresholds & statute reference

StatuteRhode Island General Laws Title 27 Chapter 27-55

Medical literature requirementAt least two articles from major peer‑reviewed medical journals presenting data supporting the proposed off‑label use as generally safe and effective (unless clear and convincing contradictory evidence exists)

Primary statutory exclusions3 (not fully FDA‑licensed; FDA‑determined contraindicated use; experimental drug not approved for any indication)

Effective / Last review datesEffective: 1994-07-12; Last review: 2021-09-15

Medical Necessity Criteria

Coverage for off-label drug use (Section 27-55-2)

Covered when ALL of the following are met:

ALL of the following

Drug is used for treatment of cancer or a disabling or life-threatening chronic disease.
Drug is approved/licensed by the FDA for at least one indication (definition section).
ALL of the following
- ONE of
  - Recognized in one of the standard reference compendia (United States Pharmacopoeia drug information; American Medical Association drug evaluations; American Hospital Formulary Service drug information).
  - Supported by medical literature (published scientific studies in at least two articles from major peer-reviewed medical journals presenting data supporting the proposed off-label use as generally safe and effective unless clear and convincing contradictory evidence exists).At least two peer-reviewed articles
Prescribing physician must submit documentation supporting the proposed off-label use if requested by the insurer.

Exclusions (coverage NOT required if ANY apply)

Drug has not been fully licensed or approved by the FDA.
Use of the drug has been determined by the FDA to be contraindicated.
Drug is experimental and not approved for any indication by the FDA.

Associated services

Coverage includes associated services when drug coverage is required:

ALL of the following

Coverage of a drug that serves as the primary treatment required by this chapter also includes medically necessary services associated with administration of the drug.

Definitions & Evidence

Drug: Any substance prescribed by a licensed health‑care provider intended for use in the diagnosis, mitigation, treatment or prevention of disease that is taken orally, injected, applied to the skin, or otherwise assimilated by the body; the term includes only substances that are approved by the FDA for at least one indication.

Medical literature: Published scientific studies in at least two (2) articles from major peer‑reviewed medical journals that present data supporting the proposed off‑label use as generally safe and effective unless clear and convincing contradictory evidence is presented in a major peer‑reviewed medical journal.

Standard reference compendia: The statute defines this as one of: (i) United States Pharmacopoeia drug information, (ii) American Medical Association drug evaluations, or (iii) American Hospital Formulary Service drug information.

Evidence items

Statute citationRhode Island General Laws Title 27 Chapter 27-55

CMS referenceMedicare Benefit Policy Manual Chapter 15, Ch. 50 Sec. 50.4.5

Rhode Island General Laws establish the off‑label coverage requirement (Section 27-55-2) and set out associated provisions. The statute mandates coverage for off‑label uses for cancer or disabling or life‑threatening chronic disease when recognized in a standard compendium or the medical literature, requires physician documentation on request, and includes coverage for medically necessary services associated with administration when the drug is the primary treatment.

The law (Section 27-55-3) requires the director of the Department of Health to appoint an advisory panel of seven medical experts to make recommendations on medical appropriateness when a dispute about payment for a particular off‑label use is referred to the director. Parties seeking review of a denial must refer the dispute in writing to the director within thirty (30) days of the denial.

The statute also specifies exclusions where coverage is not required: drugs not fully licensed or approved by the FDA, uses the FDA has determined to be contraindicated, and experimental drugs not approved for any indication.

Provider Actions

Documentation Required

Physician documentation for off-label use

Prescribing physicians must submit documentation supporting the proposed off-label use when requested by the insurer. The documentation should reference one of the specified standard reference compendia (United States Pharmacopoeia drug information; American Medical Association drug evaluations; American Hospital Formulary Service drug information) or provide medical literature consisting of at least two articles from major peer‑reviewed medical journals that present data supporting the proposed off‑label use as generally safe and effective.

Reference a standard compendium OR at least two peer‑reviewed journal articles.
Peer‑reviewed articles must meet the statute's definition (major peer‑reviewed medical journals).

Denial Risk

Denial conditions (exclusions that may lead to claim denial)

Claims may be denied (no coverage required) if any of the following apply: the drug has not been fully licensed or approved by the FDA; the FDA has determined the use to be contraindicated; or the drug is experimental and not approved for any indication by the FDA.

Not FDA‑approved for any indication (fully licensed/approved).
FDA‑determined contraindicated use.
Experimental drug not approved for any indication.

Note

Dispute referral to state health director

If coverage is denied, parties may refer the dispute in writing to the Director of the Rhode Island Department of Health within 30 days of the denial. The Director may convene an advisory panel of seven medical experts to make recommendations on whether the particular off‑label use is medically appropriate.

Written referral to the Director must be submitted within 30 days of the denial.
An advisory panel of seven licensed Rhode Island physicians may be convened to advise on medical appropriateness.

Revision History

2021-11provider_updateLatest

Provider Updates listed through November 2021; no clinical policy changes indicated in document.

2021-09-15last_review

Policy last updated (document last reviewed).

1994-07-12effective_date

Policy effective date per document header.

2017-01-01statute_effective_change

Rhode Island statutory provisions (Sections 27-55-1 and 27-55-2) cited in the policy marked effective January 1, 2017, defining coverage requirements and definitions for off-label drug use.