CurrentMass General Brigham Health PlanPolicy N/A

Off-Label Non-FDA Approved Indication

Policy governing prior authorization for off-label (non-FDA) medication uses under the pharmacy and medical benefits for MassHealth enrollees covered by Mass General Brigham Health Plan.

Policy Summary

PayerMass General Brigham Health Plan

PolicyOff-Label Non-FDA Approved Indication

Policy CodePolicy N/A

Change TypeAdded prior therapy requirement

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionSubmit prior authorization with documentation of evidence source and, for non-oncology uses, documented failure/intolerance/contraindication to 3 standard therapies.

SourceLink

POLICY UPDATE CHANGES

Requirement for documented failure, intolerance, or contraindication to 3 standard/conventional therapies for 'all other indications'.

Obesity/weight management was removed as an excluded category in a prior review (01/01/2024).

12 monthsApproval duration

3Prior therapy failures

MassHealthPlan applicability

Prior AuthProgram type

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Anti-cancer chemotherapeutic regimen

Covered when ANY ONE of the following is met for anti-cancer chemotherapeutic regimens:

Oncology evidence sources: Diagnosis/use supported by AHFS DI; OR supported in NCCN Drugs and Biologics Compendium with Category 1, 2A, or 2B; OR supported in DRUGDEX Uses/Non-FDA Uses with Strength Class I, IIa, or IIb; OR supported as an indication in Clinical Pharmacology; OR off-label use is supported in one of the listed peer-reviewed journals (e.g., Journal of Clinical Oncology; New England Journal of Medicine; Lancet; Blood; Journal of the National Cancer Institute; Journal of the National Comprehensive Cancer Network; among others); OR supported in Wolters Kluwer LexiDrugs as Evidence Level A with a Strong recommendation.

List of accepted peer-reviewed journals and compendia provided in policy.

All other indications

Covered when ALL of the following conditions are met for all other indications:

Evidence requirement: ONE of: use supported in AHFS DI; OR supported in DRUGDEX Uses/Non-FDA Uses with Strength IIb or better; OR supported by clinical research in two articles from major peer-reviewed medical journals demonstrating the proposed off-label use is generally safe and effective unless there is clear and convincing contradictory evidence.

Exclusion filters: Product is NOT used to treat excluded categories: cosmetic/hair growth, cough and cold, fertility, or sexual dysfunction; AND product is NOT defined as a DESI drug or as experimental/investigational.

Obesity/weight management exclusion was removed per prior review.

Prior therapy requirement: Documented history of failure, intolerance, or contraindication to 3 standard, conventional therapies to treat or manage the disease or condition, where available.3 therapies

Requirement added in 06/11/25 review; effective 07/01/2025.

Products used to treat the following categories are excluded from coverage under the "All other indications" pathway: cosmetic/hair growth, cough and cold, fertility, and sexual dysfunction.

Products that are defined as DESI (less than effective) drugs or that are classified as experimental and investigational are excluded from coverage under the "All other indications" criteria.

Requirements and Provider Actions

Prior Authorization

Prior Authorization and Approval Duration

Prior authorization is required for off-label (non‑FDA) medication uses. Approvals may be granted for up to 12 months; prior authorization must be obtained before claim submission to avoid denials.

Documentation Required

Oncology Off‑Label Evidence Sources

For oncology off‑label uses, authorization requires documentation that the requested use is supported by ONE of the following authoritative sources: AHFS Drug Information; the NCCN Drugs & Biologics Compendium with a Category of Evidence and Consensus of 1, 2A, or 2B; DRUGDEX Uses/Non‑FDA Uses with a Strength of Recommendation of Class I, IIa, or IIb; Clinical Pharmacology; Lexicomp (Wolters Kluwer) with Evidence Level A and a 'Strong' recommendation; or support in one of the listed peer‑reviewed medical journals.

AHFS Drug Information
NCCN Drugs & Biologics Compendium (Category 1, 2A, or 2B)
DRUGDEX Uses/Non‑FDA Uses (Class I, IIa, or IIb)
Clinical Pharmacology
Wolters Kluwer Lexicomp (Evidence Level A, 'Strong')
Support in one of the listed peer‑reviewed journals (e.g., Journal of Clinical Oncology, New England Journal of Medicine, Lancet, Blood, JAMA, etc.)

Denial Risk

Insufficient Prior Therapy

For non‑oncology off‑label uses ('All other indications'), requests must include documentation of failure, intolerance, or contraindication to three (3) standard, conventional therapies for the condition, where such alternatives are available. Requests without this documentation are at high risk for denial.

Documented failure, intolerance, or contraindication to 3 standard/conventional therapies (when available)
If alternatives are not available or appropriate, provide clinical rationale and supporting evidence

Documentation Required

Required Supporting Documentation

Provide supporting documentation with the prior authorization request that includes: the off‑label diagnosis and clinical justification; the specific evidence source(s) relied upon (cite compendia, DRUGDEX/Lexicomp/NCCN entries, or the peer‑reviewed articles); and, for non‑oncology indications, detailed documentation of prior therapies tried and the reason each failed, was not tolerated, or is contraindicated.

Off‑label diagnosis and clinical rationale
Cite the evidence source used (e.g., AHFS, NCCN with category, DRUGDEX class, Clinical Pharmacology, Lexicomp Evidence Level A, or specific peer‑reviewed articles)
For non‑oncology indications: medication names, doses, dates of therapy, and reason for failure/intolerance/contraindication for at least 3 standard therapies

Initial Authorization Requirements

Initial therapy

Initial authorization requirements for non-oncology off-label uses

Initial non-oncology: Documentation of evidence per accepted compendia/journals (AHFS DI; DRUGDEX Strength IIb or better; or two supporting peer-reviewed articles) AND documented history of failure, intolerance, or contraindication to 3 standard, conventional therapies.3 therapies

Applies to initial authorization requests for non-oncology off-label indications.

Definitions and Scope

Oncology off-label use defined

Accepted compendia — AHFS DIDiagnosis/use supported as a use in American Hospital Formulary Service Drug Information (AHFS DI)

Accepted compendia — NCCNDiagnosis/use supported in the NCCN Drugs and Biologics Compendium with Category 1, 2A, or 2B

Accepted compendia — DRUGDEXDiagnosis/use supported in DRUGDEX Uses/Non‑FDA Uses with Strength of Recommendation Class I, IIa, or IIb

Accepted reference — Clinical PharmacologyDiagnosis supported as an indication in Clinical Pharmacology

Peer‑reviewed journal support (examples)Off‑label use supported in one of the listed peer‑reviewed medical journals (e.g., Annals of Oncology; Blood; British Journal of Cancer; American Journal of Medicine; Annals of Internal Medicine)

Non‑oncology off‑label use defined

Evidence requirementONE of: AHFS DI support; OR DRUGDEX Uses/Non‑FDA Uses with Strength IIb or better; OR clinical research in two articles from major peer‑reviewed medical journals demonstrating safety and effectiveness (unless convincingly contradicted)

Excluded use categoriesProduct is NOT used for cosmetic/hair growth; cough and cold; fertility; or sexual dysfunction

Excluded product typesProduct is NOT a DESI (Less than effective/DESI) drug and NOT an experimental or investigational drug

Prior therapy requirementDocumented history of failure, intolerance, or contraindication to 3 standard, conventional therapies to treat or manage the disease/condition, where available

Background

This policy establishes the evidentiary standards and prior authorization process for permitting off‑label (non‑FDA) medication use for MassHealth enrollees covered by Mass General Brigham Health Plan. It differentiates two pathways: one for anti‑cancer chemotherapeutic regimens (authorized when supported by specified oncology compendia or peer‑reviewed oncology journals) and a separate pathway for all other indications that requires compendia/journal support plus a documented history of failure, intolerance, or contraindication to 3 standard, conventional therapies. Authorization decisions follow these standards and approvals may be granted for the durations specified in the policy.

OpenPayer

Off-Label Non-FDA Approved Indication

Coverage Criteria for Off‑Label Uses

Requirements and Provider Actions

Initial Authorization Requirements

Definitions and Scope

Background