Medical Policies - Prescription Drugs
Off-Label Use of Drugs Without a Medical Policy
*CAREFULLY CHECK STATE REGULATIONS AND/OR THE MEMBER CONTRACT*
Medical policies are a set of written guidelines that support current standards of practice. They are based on current peer-reviewed scientific literature. A requested therapy must be proven effective for the relevant diagnosis or procedure. For drug therapy, the proposed dose, frequency and duration of therapy must be consistent with recommendations in at least one authoritative source. This medical policy is supported by FDA-approved labeling and nationally recognized authoritative references. These references include, but are not limited to: MCG care guidelines, DrugDex (IIb level of evidence or higher), NCCN Guidelines (IIb level of evidence or higher), NCCN Compendia (IIb level of evidence or higher), professional society guidelines, and CMS coverage policy.
Please check the appropriate state mandate for laws governing off-label use of prescription drugs within individual plans that may supersede this medical policy when applicable.
Off-label use of U.S. Food and Drug Administration (FDA) approved drugs as prescribed by a physician to treat chronic, disabling, or life-threatening illnesses may be considered medically necessary when approved by the FDA for at least one indication, AND one of the following:
• Is recognized in one of the following prescription drug reference compendium for treatment of the indication for which the drug is prescribed
1. Thompson Micromedex Drug Dex Compendium (Drug Dex);
2. American Hospital Formulary Service Drug Information (AHFS DI);
3. National Comprehensive Cancer Network's Drugs and Biologics Compendium;
4. The United States Pharmacopoeia-Drug Information; OR
• Is supported by qualified clinical research that appears in peer-reviewed scientific literature specific for treatment of the indication for which the drug is prescribed.
NOTE #1: Qualified Peer-reviewed literature means a published scientific study including a trial that is large, multi-centered and prospective, double blinded and randomized, OR high-quality research with applicable controls in circumstances where a large, randomized, double-blind trial was not feasible or currently unavailable. Qualified peer-reviewed medical literature does not include publications or supplements to publications sponsored to a significant extent by a pharmaceutical company or an issuer of a health benefit plan.
A published scientific study is further defined as a journal article or other publication in which original manuscripts are published only after they have been critically reviewed by unbiased independent experts in the same field for scientific accuracy, validity, and reliability, and have been determined by the International Committee of Medical Journal Editors to have met the Uniform Requirements for Manuscripts submitted to biomedical journals.
NOTE #2: 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.
Off-label use of a FDA approved drug that does not meet the medically necessary coverage criteria as addressed in this medical policy or any specific HCSC drug medical policy, is considered experimental, investigational and/or unproven.
NOTE #4: If there is an HCSC medical policy regarding a specific drug, such medical policy supersedes use of this Off-Label Use policy.
Use of an orphan drug may be considered medically necessary when used to treat rare diseases and disorders as defined by the Orphan Drug Act of the U.S. Food and Drug Administration.
NOTE #5: 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 seven years following FDA approval. The Orphan Drug Act was signed into law on January 4, 1983."
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.
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.
Unapproved 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 shown to be safe and effective by the FDA after the review of adequate and controlled clinical trials that have documented their uses.
A compendium is a comprehensive listing of FDA-approved drugs and biologics. In some cases, compendia specialize in a particular subset of drugs, such as those used for anti-cancer treatment. Compendia include a summary of how each drug works in the body, as well as information for health care practitioners about proper dosing and whether the drug is recommended or endorsed for use in treating a specific disease. A compendium may be used as one of several tools to determine whether a drug should be covered.
In certain instances, scientific evidence may support using a drug to treat a disease even if the drugs U.S. Food and Drug Administration (FDA) approved label does not include those clinical conditions. In these circumstances, the compendia or scientific peer-reviewed literature specific for the indication in question may recommend uses beyond those included in the FDA approved labels. Medical policies on specific drugs are reviewed for consideration of change when valid new scientific literature emerges.
As of January 12, 2016, a search of peer reviewed literature identified no new clinical trial publications or any additional information that would change the coverage position of this medical policy.
This medical policy was updated with a literature review through January 2018. A search of peer reviewed literature identified no new clinical trial publications or any additional information that would change the coverage position of this medical policy.
Each benefit plan, summary plan description or contract defines which services are covered, which services are excluded, and which services are subject to dollar caps or other limitations, conditions or exclusions. Members and their providers have the responsibility for consulting the member's benefit plan, summary plan description or contract to determine if there are any exclusions or other benefit limitations applicable to this service or supply. If there is a discrepancy between a Medical Policy and a member's benefit plan, summary plan description or contract, the benefit plan, summary plan description or contract will govern.
Disclaimer for coding information on Medical Policies
Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each policy. They may not be all-inclusive.
The presence or absence of procedure, service, supply, device or diagnosis codes in a Medical Policy document has no relevance for determination of benefit coverage for members or reimbursement for providers. Only the written coverage position in a medical policy should be used for such determinations.
Benefit coverage determinations based on written Medical Policy coverage positions must include review of the member’s benefit contract or Summary Plan Description (SPD) for defined coverage vs. non-coverage, benefit exclusions, and benefit limitations such as dollar or duration caps.
The following codes may be applicable to this Medical policy and may not be all inclusive.
Dependent on drug
ICD-9 Diagnosis Codes
Refer to the ICD-9-CM manual
ICD-9 Procedure Codes
Refer to the ICD-9-CM manual
ICD-10 Diagnosis Codes
Refer to the ICD-10-CM manual
ICD-10 Procedure Codes
Refer to the ICD-10-CM manual
The information contained in this section is for informational purposes only. HCSC makes no representation as to the accuracy of this information. It is not to be used for claims adjudication for HCSC Plans.
The Centers for Medicare and Medicaid Services (CMS) does not have a national Medicare coverage position. Coverage may be subject to local carrier discretion.
A national coverage position for Medicare may have been developed since this medical policy document was written. See Medicare's National Coverage at <http://www.cms.hhs.gov>.
1. 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. Available at: <www.legis.state.tx> (last accessed February 2018).
2. Illinois Mandated Benefits; Cancer Off-Label Drugs 215 ILCS 5/370 or, 215 ILCS 125/4-6.3. Last accessed September 2014
3. New Mexico Mandated Benefits, Prescription Drugs 13:10. 13:12. Last accessed September 2014
4. Oklahoma Statutes Citationized Title 63. Public Health and Safety Chapter 1 - Oklahoma Public Health Code Article 26 - Kidney Health Planning Act of Oklahoma Section 1-2604 - Coverage for Prescription Drugs for Cancer Treatment or Study of Oncology. Last accessed September 2014.
5. AMA House of delegates Health and Ethics Policies, H-120.988 Patient Access to Treatments Prescribed by Their Physicians. Available at: <www.ama-assn.org>.
6. Dresser, R., At Law: The Curious case of Off-Label Use. The Hastings Center Report. 6/7/2007.
7. Off-Label Drug Use. Chicago, Illinois: Blue Cross Blue Shield Association Medical Policy Reference Manual (Archived 2011 December) Prescription Drug: 5.01.01.
|11/1/2018||Document updated with literature review. Document updated with literature review. The following statement was added to coverage. "Off-Label use of a FDA approved drug that does not meet the medically necessary coverage criteria as addressed in this medical policy or any specific HCSC drug medical policy, is considered experimental, investigational and/or unproven". In addition, the United States Pharmacopoeia-Drug Information compendium added to the listing of accepted compendia. Some wording in coverage modified for clarification.|
|3/1/2017||Reviewed. No changes.|
|3/1/2016||Document updated with literature review. Coverage unchanged.|
|7/1/2015||Reviewed. No changes.|
|12/1/2014||Document updated with literature review. Coverage unchanged.|
|8/1/2012||Document updated with literature review. Coverage statements modified to add current compendium.|
|9/15/2007||Revised/Updated Entire Document|
|2/27/2004||Revised/Updated Entire Document|
|6/1/1996||Revised/Updated Entire Document|
|3/1/1996||Revised/Updated Entire Document|
|4/1/1994||New medical policy|
|Title:||Effective Date:||End Date:|
|Off-Label Use of Drugs Without a Medical Policy||03-01-2017||10-31-2018|
|Off-Label Use of Drugs Without a Medical Policy||03-01-2016||02-28-2017|
|Off-Label Use of U.S. Food and Drug Administration (FDA) Approved Drugs||07-01-2015||02-29-2016|
|Off-Label Use of FDA Approved Drugs||12-01-2014||06-30-2015|
|Off-Label Use of U.S. Food and Drug Administration (FDA) Approved Drugs||08-01-2012||11-30-2014|
|Off-Label Use of FDA Approved Drugs||09-15-2007||07-31-2012|
|Off-Label Use of FDA Approved Drugs||02-27-2004||09-14-2007|
|Off Label Use of FDA Approved Drug||05-01-1996||02-26-2004|