Off-Label Use of Chemotherapeutic Drugs and Biologicals for Non-Cancer
Indications

This article addresses the off-labeled use of chemotherapeutic drugs and
biologicals for non-cancer indications.

**Definitions:  
**  
Off-label (unlabeled) drug use is defined as a non-FDA approved indication
that is not listed on the drug's official label/prescribing information. Off-
label use is further defined as giving the drug in a way that deviates
significantly from the labeled prescribing information for a particular
indication. This includes but is not necessarily limited to, dosage, route of
administration, duration, and frequency of administration, and population to
whom the drug would be administered.

An indication is defined as a diagnosis, illness, injury, syndrome, condition,
or other clinical parameter for which a drug may be given.

**Guidance:**

For the off-label (unlabeled) use of drugs and biologicals for indications
that are not part of an anti-cancer chemotherapeutic regime, NGS will consider
coverage on case by case basis per CMS internet only manual (IOM), 100-02,
Chapter 15, 50.4.2 Unlabeled Use of Drugs. Inclusion of these drugs and
biologicals in a compendium does not guarantee coverage, but the compendium
recommendation will be considered as part of the review.

The following may be submitted to support the coverage with the claim:

  * Submission of compendium reference if available
  * Full text support articles for use of the agent for the indication
  * Societal recommendations

The medical records must document the rationale for the off-label use of the
agent.

If a cancer chemotherapy drug/biologic use is identified as not indicated by
the Centers for Medicare and Medicaid Services (CMS) or the FDA or if NGS
determines that peer-reviewed medical literature does not support the off-
label usage, the drug/biologic is not covered.

**Not Otherwise Classified (NOC) Drug Billing:  
**  
List the appropriate J or Q HCPCS code to indicate the drug being billed. When
a specific HCPCS code does not exist, list the appropriate NOC code J3490,
J3590, or J9999 with the drug name, and dose in the remarks field of the claim
Item 19 of the CMS-1500 form for claims billed to the Part B MAC.

For Part A, claims with C9399 are suspended, and remarks should be placed on
the claim in field locator 80 for the UB04 claim form or SV202-7 for 837I
electronic claim.

Facilities under the hospital OPPS may use code C9399, billed as one unit, for
drugs that do not have an assigned HCPCS code or the assigned HCPCS code is
not yet effective.

**Note:** Claims billed to the Part B MAC with C9399 will be rejected.

Claims billed with a NOC drug code are suspended and the following information
should be provided on the claim in the following fields:

  * Field locator 80 for paper claims /line SV202-7 for electronic claims for Part A.
  * Box 19 for paper claims/loop 2400 or SV101-7 for Part B.

Claims billed with a NOC drug code need to include the following information
in the fields listed above:

  * Name of the drug
  * Dose administered (mg, cc, etc.)
  * Route of administration (IV, IM, SC, PO, etc.)
  * NDC number if available
  * The invoice price (for new drugs if the wholesale acquisition cost (WAC) is unavailable, or for compounded drugs).

The JW modifier is required for wastage in single-dose vials; this modifier
does not apply to multi-dose vials per CMS Internet-Only Manual, Pub 100-04,
Chapter 17, Section 40 Discarded Drugs and Biologicals (
<https://www.cms.gov/Regulations-and-
Guidance/Guidance/Manuals/downloads/clm104c17.pdf>).