1. Horizon Blue Cross Blue Shield of New Jersey considers "off-label" use of a prescription drug medically necessary when the following criteria are met:

A. The drug has been approved by the United States Food and Drug Administration (FDA) for use but the specific treatment for which it is being prescribed is not indicated in the labelling approved by the FDA; and

B. The appropriateness of the specific treatment for which the drug is being prescribed is recognized or supported in one of the following established reference compendia:
• the American Medical Association (AMA) Drug Evaluations (see informational note below);
• the American Hospital Formulary Service (AHFS) Drug Information; or
• the United States Pharmacopoeia Drug Information (USP DI) (see informational note below).

[INFORMATIONAL NOTE:
• AMA Drug Evaluations combined with USP DI:
  In 1994, the United States Pharmacopoeia (USP) signed an agreement with the AMA to combine the information in the AMA's Drug Evaluations database with the USP DI database to develop a single product that contains drug and therapeutic information.
• USPDI changed its name to DrugPoints:
  In July 2007, Thomson Corporation announced that the combined AMA USP DI had been replaced by Thomson Healthcare's enhanced DrugPoints. DrugPoints provides summary information on dosing, drug interactions, adverse effects, pregnancy warnings, indications, cautions, therapeutic classes, and brand information.
  
• Truven Micromedex (formerly known as Thompson Micromedex DrugDex®)
  More detailed information and research summaries can be found in Truven Micromedex . It includes evidence-based, fully referenced information, and provides strength of evidence, recommendation and efficacy ratings for all therapeutic uses.]

• National Comprehensive Cancer Network (NCCN) Drugs & Biologics Compendium
• Clinical Pharmacology

The off-label uses which are experimental or investigational are not covered. The clinical evidence or study design should be reviewed to determine whether it supports a finding that the use is not experimental or investigational, considering the following:
• the prevalence and life history of the disease when evaluating the adequacy of the number of subjects and response rate;
• the effect on the patient's well-being and other responses to therapy that indicate effectiveness, i.e., an objective decrease in the size of the tumor or a reduction in symptoms related to the tumor;
• whether the experimental design in light of the drugs and conditions under investigation is appropriate to address the investigative question;
• that observational surveys, case report registers and case reports with clinical follow up and documentation may be a basis for supportive clinical evidence for determining accepted uses of the drugs;
• that individual case reports are generally considered uncontrolled and anecdotal information that does not provide adequate supportive clinical evidence for determining accepted uses of the drugs;
• opinion of outside consultants expert in the specific area in question.

[Please note that Horizon Blue Cross Blue Shield of New Jersey has agreed to participate in a voluntary state-wide program to provide coverage for routine costs associated with all phases of approved cancer clinical trials in New Jersey.]

Medicare Advantage Products differs from the Horizon BCBSA Policy. Medicare Advantage Products follows the below CMS guidance regarding Off-Label Use of Prescription Drugs:

Local Coverage Article: Approved Drugs and Biologicals; Includes Cancer Chemotherapeutic Agents (A53049). Available to be accessed at Novitas Solutions, Inc., Medical Policy Search page: https://www.novitas-solutions.com/webcenter/portal/MedicareJL/LcdSearch?_afrLoop=90769712476969#!%40%40%3F_afrLoop%3D90769712476969%26centerWidth%3D100%2525%26leftWidth%3D0%2525%26rightWidth%3D0%2525%26showFooter%3Dfalse%26showHeader%3Dfalse%26_adf.ctrl-state%3D63y7eftob_46.

Title XVIII of the Social Security Act, Section 1861(t)(1) Drugs and Biologicals. Available at: Social Security Administration search page:https://www.ssa.gov/OP_Home/ssact/title18/1861.htm#act-1861-r.

Medicaid Coverage

For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf

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Horizon BCBSNJ Medical Policy Development Process:

This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.

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Index:
Off-Label Use of Prescription Drugs
Prescription Drugs, Off-Label Use

References:
1. New Jersey State Mandate (P.L. 1993, c. 321) approved December 23, 1993 and effective June 21, 1994 requiring health service corporations, HMOs. hospital service corporations, and medical service corporations to provide coverage for drugs which have been approved by the FDA in cases where the drugs are prescribed for treatments other than those stated in the labelling approved by FDA ("off-label" use).

2. Medicare Part B Answer Book. Coverage for off-label chemotherapy drugs. April 2001.

3. National Comprehensive Cancer Network (NCCN) Drugs and Biologics Compendium. Available at: http://www.nccn.org/professionals/drug_compendium/content/contents.asp

4. CMS Manual System (Pub 100-02 Medicare Benefit Policy): Compendia as Authoritative Sources for Use in the Determination of a "Medically Accepted Indication" of Drugs and Biologicals used Off-Label in an Anti-Cancer Chemotherapeutic Regimen. Transmittal 96, Oct. 24, 2008. Available at: http://www.cms.hhs.gov/Transmittals/downloads/R96BP.pdf


Codes:
(The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)

CPT*