Determination of Approved and Accepted Off-label Drug Indications

In the absence of a drug-specific National Coverage Determination (NCD), Local Coverage Determination or other Medicare instruction(s), a provider may use the following guidance to determine drug coverage under regular Parts A and B of Medicare (please see separate instructions for coverage under the Part D Prescription Drug Program or under the contractor websites for Durable Medical Equipment, Prosthetics, Orthotics and Supplies (DMEPOS) program).

CMS has established five drug compendia as authoritative sources on drug benefit category for cancer drugs. Noridian uses the evidentiary levels of efficacy discussed in these compendia to determine whether a drug may be covered for a given indication. The CMS approved authoritative compendia are listed in the CMS Internet Only Manual (IOM), Publication 100-02, Medicare Benefit Policy Manual, Chapter 15, Section 50.4.5. The approved compendia are as follows in no particular order.

  • American Hospital Formulary Service-Drug Information (AHFS-DI)
  • National Comprehensive Cancer Network (NCCN) Drugs and Biologics Compendium
  • Micromedex DrugDex
  • Clinical Pharmacology
  • Lexi-Drugs

For all drugs, including anti-cancer drugs, and in the absence of any statutory, CMS, or Noridian exclusion of any drug or specific drug indication, Noridian recognizes an indication to be medically accepted if the indication is both:

  • FDA-approved for the drug at the FDA published dose and frequency; or
  • Listed in one or more of the approved compendia with an appropriate level of evidence of efficacy (see below); or
  • Recognized, following Medicare's review of the peer reviewed literature, as an appropriate treatment: (The requestor is expected to provide significant peer reviewed full articles to the Contractor for review)

AND

  • The use is not listed as unsupported, not indicated, not recommended (or equivalent terms) in any of the compendia. Any such listing precludes reimbursement for the drug

"Medically accepted" definitions by compendium:

  1. NCCN: The level of evidence for the indication is Category 1 or 2A. (If a provider chooses to use NCCN level 2B in support of a chemotherapeutic drug used for an off-label indication, Noridian expects that the provider will make available to Noridian significant peer reviewed phase II or phase III studies demonstrating such support.)
  2. DrugDex: The level of evidence for the indication is a Class I, Class IIa, or Class IIb.
  3. AHFS-DI or Clinical Pharmacology: The narrative text is supportive.
  4. Lexi-Drugs: The indication is listed as "Use: Off-Label" and rated as "Evidence Level  A."

Not "medically accepted" by a compendium:

  1. NCCN: The level of evidence for the indication is Category 3 in NCCN.
  2. DrugDex: The level of evidence for the indication is Class III in DrugDex.
  3. AHFS-DI or Clinical Pharmacology: The narrative text is "not supportive" (or equivalent term).
  4. Lexi-Drugs: Indication is listed as "Use: Unsupported."

The complete absence of narrative text on a use is considered neither supportive nor non-supportive.

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Last Updated Dec 14 , 2022