Off-label and Administrative Criteria

Off-label and Administrative Criteria

Diagnosis considered for coverage:

• Food and Drug Administration (FDA)-approved indication
• Off-label non-FDA approved indication

Coverage Criteria:

For drugs without criteria approved by the WHA P&T Committee:

• Prescribed medication is being used for a Food and Drug Administration (FDA) approved indication, AND
• All components of the FDA approved indication are met (e.g., concomitant use, previous therapy requirements, age limitations, testing requirements, etc.), AND
• Patient meets one of the following:
  • Dose requested does not exceed FDA approved maximum dose for the indication, OR
  • Dose requested is appropriate for the patient’s age or weight

For diagnosis of off-label non-FDA approved indication(s):

• The drug has been approved as safe and effective by the FDA for at least one indication, AND
• Preferred drugs for the condition have failed to achieve therapeutic goals, are contraindicated, or caused unacceptable side effects, AND
• The drug is prescribed by a participating (PAR) provider for a life-threatening condition, or a chronic and seriously debilitating condition, for which no other drug or therapy exists, AND
• Patient meets one of the following: 
  • Diagnosis is supported as a use in American Hospital Formulary Service Drug Information (AHFS DI), OR
  • Diagnosis is supported as a use in the National Comprehensive Cancer Network (NCCN) Drugs and Biologics Compendium with a Category of Evidence and Consensus of 1, 2A, or 2B, OR
  • Diagnosis is supported in the FDA Uses/Non-FDA Uses section in DRUGDEX Evaluation with a Strength of Recommendation rating of Class I, Class IIa, or Class IIb, OR
  • Diagnosis is supported as an indication in Clinical Pharmacology, OR
  • The use is supported by clinical research in two articles from major peer reviewed medical journals that present data supporting the proposed off-label use as generally safe and effective unless there is clear and convincing contradictory evidence presented in a major peer-reviewed medical journal

Coverage Duration: 

• Initial: 1 year
• Reauthorization: 1 year

Additional Information: 

• Examples of peer-reviewed medical literature:
  • American Journal of Medicine 
  • Annals of Internal Medicine 
  • Annals of Oncology 
  • Annals of Surgical Oncology 
  • Biology of Blood and Marrow Transplantation 
  • Blood 
  • Bone Marrow Transplantation 
  • British Journal of Cancer 
  • British Journal of Hematology 
  • British Medical Journal 
  • Cancer 
  • Clinical Cancer Research 
  • Drugs 
  • European Journal of Cancer (formerly the European Journal of Cancer and Clinical Oncology) 
  • Gynecologic Oncology 
  • International Journal of Radiation, Oncology, Biology, and Physics 
  • The Journal of the American Medical Association 
  • Journal of Clinical Oncology 
  • Journal of the National Cancer Institute 
  • Journal of the National Comprehensive Cancer Network (NCCN) 
  • Journal of Urology 
  • Lancet 
  • Lancet Oncology 
  • Leukemia 
  • The New England Journal of Medicine 
  • Radiation Oncology

Policy Updates:

• 5/17/2022 – New policy approved by P&T

References:

• Center for Medicaid & Medicare Services. Medicare Prescription Drug Benefit Manual. Chapter 6 – Part D Drugs and Formulary Requirements. Section 10.6. Available at: https://www.cms.gov/Medicare/Prescription-Drug-Coverage/PrescriptionDrugCovContra/Downloads/Part-D-Benefits-Manual-Chapter-6.pdf. Accessed September 9, 2020. 
• Center for Medicaid & Medicare Services. Medicare Benefit Policy Manual. Chapter 15 - Covered Medical and Other Health Services. Section 50.4.5. Available at: https://www.cms.gov/Regulations-and-Guidance/Guidance/Manuals/downloads/bp102c15.pdf. Accessed September 9, 2020. 
• National Comprehensive Cancer Network Categories of Evidence and Consensus. Available at: https://www.nccn.org/professionals/physician_gls/categories_of_consensus.aspx. Accessed September 9, 2020. 
• Center for Medicaid & Medicare Services. Medicare Benefit Policy Manual. Wolters Kluwer Clinical Drug Information Lexi-Drugs Compendium Revision Request - CAG-00443O. Available at: https://www.cms.gov/medicare-coverage-database/details/medicare-coverage-document-details.aspx?MCDId=31#decision. Accessed September 9, 2020. 
• Wolters Kluwer Clinical Drug Information’s Request for CMS evaluation of Lexi-Drugs as a compendium for use in the determination of medically-accepted indications of drugs/biologicals used off-label in anti-cancer chemotherapeutic regimens. Available at: https://www.cms.gov/Medicare/Coverage/CoverageGenInfo/downloads/covdoc31.pdf. Accessed September 9, 2020. 
• Micromedex Healthcare Series. Recommendation, Evidence, and Efficacy Ratings. https://www.micromedexsolutions.com/micromedex2/librarian/ssl/true/CS/6E0ED9/ND_PR/evidencexpert/ND_P/evidencexpert/DUPLICATIONSHIELDSYNC/8B9F5B/ND_PG/evidencexpert/ND_B/evidencexpert/ND_AppProduct/evidencexpert/ND_T/evidencexpert/PFActionId/evidencexpert.IntermediateToDocumentLink?docId=3198&contentSetId=50. Accessed September 9, 2020.
• Center for Medicaid & Medicare Services. Medicare Coverage Document. Thomson Micromedex DrugDex ® Compendium Revision Request - CAG-00391. Available at: https://www.cms.gov/medicare-coverage-database/view/medicare-coverage-document.aspx?MCDId=16. Accessed October 4, 2021.