OFF-LABEL USE of FDA APPROVED DRUGS RX503.001 Bluereview POSTED DATE: 11/17/2003 EFFECTIVE DATE: 2/27/2004 ______

OFF-LABEL USE OF FDA APPROVED DRUGS RX503.001 BlueReview POSTED DATE: 11/17/2003 EFFECTIVE DATE: 2/27/2004 ______

COVERAGE:

Off-Label use of FDA approved drugs as prescribed by a physician to treat chronic, disabling, or life-threatening illnesses may be covered when meeting the criteria outlined below:

· Has been approved by the FDA for at least one indication

· Is in one of the standard reference compendia for the off-label indication,

Standard reference compendia means: 1. The United States Pharmacopoeia Drug Information; 2. The American Hospital formulary service Drug Information

· Is supported by clinical research that appears in peer-reviewed literature specific for the indication in question.

Peer-reviewed literature means a published scientific study including a trial that is preferably large, multi-centered and prospective, double blinded and randomized.

Peer-reviewed medical literature does not include publications or research studies that are sponsored to a significant extent by a pharmaceutical company or financially interested parties.

Coverage of a drug that meets the coverage criteria shall also include medically necessary services associated with the administration of the drug.

Off-Label use of FDA approved drugs is considered non covered when the FDA has determined its use to be contraindicated for treatment of the condition for which coverage is requested.

Exception: If there is an HCSC medical policy or position statement regarding a specific drug that provides a coverage position for off- label use(s) of that drug, such medical policy or position statement supersedes use of this Off-Label Use policy.

Note: Medical policies or position statements on specific drugs are reviewed for consideration of change when valid new scientific literature emerges.

Use of an orphan drug is considered medically necessary when used to treat rare diseases and disorders as defined by the Orphan Drug Act of Blue Cross and Blue Shield of Texas, a Division of Health Care Service Corporation, a Mutual Legal Reserve Company* Southwest Texas HMO, Inc.* d/b/a HMO BlueÒ Texas * Independent Licensees of the Blue Cross and Blue Shield Association

OFF-LABEL USE OF FDA APPROVED DRUGS RX503.001 BlueReview POSTED DATE: 11/17/2003 EFFECTIVE DATE: 2/27/2004 ______the U.S. Food and Drug Administration.

An Orphan Drug is defined in the 1984 amendments of the Orphan Drug Act as “a drug intended to treat a condition affecting fewer than 200,000 persons in the United States or will not recover development cost, plus a reasonable profit, within 7 years following FDA approval. The Orphan Drug Act was signed into law on January 4,1983.” ______DESCRIPTION:

Off-label or “unlabeled” drug use is the use of a drug approved by the U.S. Food and Drug Administration (FDA) for other uses or in treatment regimens or patient populations that are not included in approved labeling.

Unproved or unlabeled uses of drugs include a variety of situations ranging from completely unstudied to thoroughly investigated drug uses where the FDA has not been asked for approval, whereas approved uses of drugs have been proved to be safe and effective by the FDA after the review of adequate and controlled clinical trials that have documented their uses. ______RATIONALE:

The FDA approves drugs for specific indications that are included in the drug’s labeling. When a drug is used for an indication other than those specifically included in the labeling, it is referred to as an off-label use. Many off-label uses are effective, well-documented in the literature, and widely used.

Texas legislative mandate requires coverage of Off-Label use of FDA approved drugs when meeting the criteria as outlined in the coverage section of this policy.

Illinois department of Insurance has a mandate on Cancer Off-Label Drugs that requires “If a policy provides prescription drug benefits, it must also provide benefits for any drug that has been prescribed for the treatment of a type of cancer, even if the drug has not been approved for that specific cancer by the FDA. The drug must be approved by the FDA and must be recognized for treatment of the specific cancer for which it has been prescribed by an established reference compendium”. This applies to group insurance policies and individual and group HMO contracts. ______PRICING:

None

Blue Cross and Blue Shield of Texas, a Division of Health Care Service Corporation, a Mutual Legal Reserve Company* Southwest Texas HMO, Inc.* d/b/a HMO BlueÒ Texas * Independent Licensees of the Blue Cross and Blue Shield Association

OFF-LABEL USE OF FDA APPROVED DRUGS RX503.001 BlueReview POSTED DATE: 11/17/2003 EFFECTIVE DATE: 2/27/2004 ______

______REFERENCES:

· Legislature of the State of Texas. Section 1. Subchapter E, Chapter 21, Insurance Code. Article 21.53H "Coverage for Off-Label Uses of FDA Approved Drugs" · “Off-Label Drug Use." BCBSA Consortium Health Plan Medical Policy Reference Manual (2002 July 12) Prescription Drug: 5.01.01 · Illinois Mandated Benefits, Cancer Off-Label Drugs 215 ILCS 5/37Or, 215 ILCS 125/4-6.3 ______DISCLAIMER:

State and federal law, as well as contract language, including definitions and specific inclusions/exclusions, takes precedence over Medical Policy and must be considered first in determining coverage. The member’s contract benefits in effect on the date that services are rendered must be used. Any benefits are subject to the payment of premiums for the date on which services are rendered. Medical technology is constantly evolving, and we reserve the right to review and update Medical Policy periodically. HMO Blue Texas physicians who are contracted/affiliated with a capitated IPA/medical group must contact the IPA/medical group for information regarding HMO claims/reimbursement information and other general polices and procedures.