Off-Label Use of Drugs and Biologicals for Anti-Cancer Chemotherapeutic
Regimen

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

**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:**

The general utilization of any cancer chemotherapy drug or biological will be
covered for a use that is not FDA approved (unlabeled) if used for anti-
neoplastic conditions, and if the use is listed as acceptable in one of the
Medicare-approved drug compendia and the use is not listed as "not indicated"
or unfavorably evaluated in any of the compendia per the Centers for Medicare
and Medicaid Services (CMS) Internet-Only Manual (IOM), Pub 100-02, Chapter
15, 50.4.5 Off Label Use of Anti-Cancer Drugs and Biologicals.

NGS may also identify off-label uses for anti-cancer drugs and biologicals as
part of an anti-cancer chemotherapeutic regimen that are not listed as
supported/recommended in medically accepted compendia (NCCN, Micromedex Drug
DEX, Lexi-Drugs, AHFS or Clinical Pharmacology). Coverage will be determined
for off-label use if supported by clinical research under the conditions
identified in the CMS IOM, Pub 100-02, Chapter 15, Section 50.4.5 Off-Label
Use of Anti-Cancer Drugs and Biologicals. For a review of medications under
this process, please submit full articles, not abstracts, for consideration.

Consistent with CMS IOM, Pub 100-02, Chapter 15, Section 50.4.5 D, if a cancer
chemotherapy drug/biologic use is identified as not indicated by CMS or the
FDA, or the use is specifically identified as not indicated in one or more of
the compendia, 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, if
it does not have an assigned HCPCS or the assigned HCPCS 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_ ).