WHA

High Dollar/Claim Dollar

Indications for Prior Authorization

Criteria

A drug (non-anti-cancer chemotherapeutic regimen) used for an off-label indication or FDA approved indication

**May not apply to all benefit plans.

Administrative

Length of Approval: 12 months, if no PA is on file. Approval duration is granted for length of current PA on file (if existing PA is on file).

  • One of the following:
    • Requested drug is being used for a Food and Drug Administration (FDA)-approved indication
      • OR
    • One of the following:
      • Diagnosis is supported as a use in American Hospital Formulary Service Drug Information (AHFS DI) [1]
        • OR
      • Diagnosis is supported in the FDA Uses/Non-FDA Uses section in DRUGDEX Evaluation with a Strength of Recommendation rating of IIb or better (see DRUGDEX Strength of Recommendation table in Background section) [1]
        • 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 or uses as generally safe and effective unless there is clear and convincing contradictory evidence presented in a major peer-reviewed medical journal**
    AND
  • One of the following:
    • The dosage quantity/duration of the medication is reasonably safe and effective based on information contained in the FDA approved labeling, peer-reviewed medical literature, or accepted standards of medical practice
      • OR
    • The dosage/quantity/duration of the medication is reasonably safe and effective based on one of the following compendia:
      • American Hospital Formulary Service (AHFS) Compendium
      • Thomson Reuters (Healthcare) Micromedex/DrugDex (not Drug Points) Compendium
A drug or biological in an anti-cancer chemotherapeutic regimen

**May not apply to all benefit plans.

Administrative

Length of Approval: 12 months, if no PA is on file. Approval duration is granted for length of current PA on file (if existing PA is on file).

  • One of the following:
    • Requested drug is being used for a Food and Drug Administration (FDA)-approved indication
      • OR
    • One of the following:
      • Diagnosis is supported as a use in American Hospital Formulary Service Drug Information (AHFS DI) [2]
        • OR
      • Diagnosis is supported in the FDA Uses/Non-FDA Uses section in DRUGDEX Evaluation with a Strength of Recommendation rating of IIb or better (see DRUGDEX Strength of Recommendation table in Background section) [2]
        • 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 (see NCCN Categories of Evidence and Consensus table in Background section) [2, B]
        • OR
      • Diagnosis is supported as an indication in Clinical Pharmacology [2]
        • OR
      • Off-label use is supported in one of the published, peer-reviewed medical literature listed below: [2, C]
        • 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
        OR
      • Diagnosis is supported as a use in Wolters Kluwer Lexi-Drugs rated as "Evidence Level A" with a "Strong" recommendation. (see Lexi-Drugs Strength of Recommendation table in Background section) [2, 4, 5]
    AND
  • One of the following:
    • The dosage quantity/duration of the medication is reasonably safe and effective based on information contained in the FDA approved labeling, peer-reviewed medical literature, or accepted standards of medical practice
      • OR
    • The dosage/quantity/duration of the medication is reasonably safe and effective based on one of the following compendia:
      • American Hospital Formulary Service (AHFS) Compendium
      • Thomson Reuters (Healthcare) Micromedex/DrugDex (not Drug Points) Compendium
      • Elsevier Gold Standard’s Clinical Pharmacology Compendium
      • National Comprehensive Cancer Network Drugs and Biologics Compendium

  • Guideline ID
    GL-149294

  • Formulary
    EHB

  • Effective date
    Sep 1, 2024

  • P&T approval date
    Mar 26, 2017

P & T Revisions

2024-07-01, 2023-09-21, 2022-10-14, 2022-01-06, 2021-10-26, 2021-09-28, 2021-05-20, 2020-10-11, 2019-10-04

  1. 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 July 1, 2024.
  2. 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 July 1, 2024.
  3. 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, 2021.
  4. 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 July 1, 2024.
  5. 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 July 1, 2024.
  6. 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 July 1, 2024.
  1. OptumRx has high dollar criteria for clients who opt for such a program to help manage prescription costs. If the prescription cost exceeds the claim or high dollar threshold, then an administrative PA will be required. The pharmacist will review the prescription to see if it is in-line with FDA approved labeling or well supported by the approved compendia or a peer-reviewed medical journal.
  2. NCCN Categories of Evidence and Consensus. Category 1: The recommendation is based on high-level evidence (i.e., high-powered randomized clinical trials or meta-analyses), and the NCCN Guideline Panel has reached uniform consensus that the recommendation is indicated. In this context, uniform means near unanimous positive support with some possible neutral positions. Category 2A: The recommendation is based on lower level evidence, but despite the absence of higher level studies, there is uniform consensus that the recommendation is appropriate. Lower level evidence is interpreted broadly, and runs the gamut from phase II to large cohort studies to case series to individual practitioner experience. Importantly, in many instances, the retrospective studies are derived from clinical experience of treating large numbers of patients at a member institution, so NCCN Guideline Panel Members have first-hand knowledge of the data. Inevitably, some recommendations must address clinical situations for which limited or no data exist. In these instances the congruence of experience-based judgments provides an informed if not confirmed direction for optimizing patient care. These recommendations carry the implicit recognition that they may be superseded as higher level evidence becomes available or as outcomes-based information becomes more prevalent. Category 2B: The recommendation is based on lower level evidence, and there is nonuniform consensus that the recommendation should be made. In these instances, because the evidence is not conclusive, institutions take different approaches to the management of a particular clinical scenario. This nonuniform consensus does not represent a major disagreement, rather it recognizes that given imperfect information, institutions may adopt different approaches. A Category 2B designation should signal to the user that more than one approach can be inferred from the existing data. Category 3: Including the recommendation has engendered a major disagreement among the NCCN Guideline Panel Members. The level of evidence is not pertinent in this category, because experts can disagree about the significance of high level trials. Several circumstances can cause major disagreements. For example, if substantial data exist about two interventions but they have never been directly compared in a randomized trial, adherents to one set of data may not accept the interpretation of the other side's results. Another situation resulting in a Category 3 designation is when experts disagree about how trial data can be generalized. An example of this is the recommendation for internal mammary node radiation in postmastectomy radiation therapy. One side believed that because the randomized studies included this modality, it must be included in the recommendation. The other side believed, based on the documented additional morbidity and the role of internal mammary radiation therapy in other studies, that this was not necessary. A Category 3 designation alerts users to a major interpretation issue in the data and directs them to the manuscript for an explanation of the controversy. [3]
  3. Abstracts (including meeting abstracts) are excluded from consideration. When evaluating peer-reviewed medical literature, the following (among other things) should be considered: 1) Whether the clinical characteristics of the beneficiary and the cancer are adequately represented in the published evidence 2) Whether the administered chemotherapy regimen is adequately represented in the published evidence. 3) Whether the reported study outcomes represent clinically meaningful outcomes experienced by patients. 4) Whether the study is appropriate to address the clinical question. The following should be considered: a) Whether the experimental design, in light of the drugs and conditions under investigation, is appropriate to address the investigative question. (For example, in some clinical studies, it may be unnecessary or not feasible to use randomization, double blind trials, placebos, or crossover.); b) That non-randomized clinical trials with a significant number of subjects may be a basis for supportive clinical evidence for determining accepted uses of drugs; and c) That case reports are generally considered uncontrolled and anecdotal information and do not provide adequate supportive clinical evidence for determining accepted uses of drugs. [2]
  • 2024-07-01: 2024 UM Annual Review. Minor updates without changing clinical intent.
  • 2023-09-21: 2023 Annual Review. No changes to criteria.
  • 2022-10-14: 2022 Annual Review
  • 2022-01-06: Update guideline
  • 2021-10-26: Updated references section.
  • 2021-09-28: Addition of EHB formulary to guideline, no changes to criteria
  • 2021-05-20: Addition of EHB formulary to guideline, no changes to criteria
  • 2020-10-11: Annual Review - Addition of Annals of Internal Medicine as one of the references to support off-label anti-cancer use; background and reference updates.
  • 2019-10-04: Annual Review. Updated Background/References.