Off-Label Use of Pharmaceuticals and Related Products

Where We Stand:

The Academy of Managed Care Pharmacy (AMCP) supports off‐label use of FDA‐approved pharmaceuticals and related products when medically appropriate, safe, and necessary. AMCP opposes government‐mandated coverage of specific pharmaceuticals and related products, whether for FDA‐approved or off‐label uses, which can fail to consider safe, effective, and lower-cost alternatives. 

Off‐label use means the use of a pharmaceutical or related product for clinical uses other than those stated in the product labeling approved by the U.S. Food and Drug Administration (FDA). An example would be using a drug to treat one form of cancer (e.g., pancreatic) that has FDA approved uses for treating other types of cancer (e.g., ovarian, bladder, breast and lung). 

AMCP believes each pharmaceutical product on the market should be used only in accordance reliable scientific data and generally accepted standards of medical practices in order to best ensure safe and effective patient care. AMCP also recognizes that pharmacotherapy is a rapidly changing field and that FDA‐approved uses for particular drug products do not always keep pace with changing clinical guidelines and medical practices. Therefore, AMCP supports managed care organizations and third-party payors consider the following criteria before deciding whether to provide coverage of FDA‐approved pharmaceuticals or related products for off‐label uses:

• Whether the pharmaceutical product has been proven safe, effective and accepted for the treatment of the specific medical condition for which it has been prescribed according to nationally recognized drug compendia: the current edition of American Hospital Formulary Service ‐ Drug Information® (AHFS‐DI®), Micromedex® DrugDex®, Clinical Pharmacology, Lexi-Drug®, or the National Comprehensive Cancer Network (NCCN) Drug and Biologics Compendium™. 

• Whether the pharmaceutical product is recommended for the particular condition involved and has been proven to be safe and effective for that condition according to nationally recognized evidence-based guidelines, or reproducible formal clinical studies, or reliable scientific data, the results of which have been published in peer‐reviewed evidence‐based medical literature. Randomized, controlled trials are preferred over observational research or case studies. 

AMCP does not believe government should mandate coverage of specific pharmaceuticals or related products. Benefit design, including coverage determination, must be the responsibility of managed care organizations so they may ensure the provision of appropriate services for their members. Coverage determination when used in conjunction with other managed care pharmacy tools, such as formularies, drug utilization review and medical treatment guidelines promote quality and optimal outcomes. Well-developed and quality based managed care plans, as part of developing a sound formulary system, utilize a variety of tools and strategies which incorporate practicing health care professionals and Pharmacy and Therapeutic Committees to valid appropriate evidence based current clinical practice. To ensure the provision of appropriate services for their members, managed care organizations must have authority to determine when to cover pharmaceuticals and related products for off‐label uses, and in making these determinations, should take into account medical appropriateness, fiscal responsibility and principles of compassionate use.
 
See also: 

• AMCP’s position statement on Formularies, Prescription Drug Coverage, Principles of a Sound Drug Formulary, and Government Mandated Pharmacy Benefits. 
• AMCP Where We Stand series: https://www.amcp.org/policy-advocacy/policy-advocacy-focus-areas/where-we-stand-position-statements 

Revised by the AMCP Board of Directors, December 2020 
Revised by the AMCP Board of Directors, December 2008  
Revised by the AMCP Board of Directors, July 1997  
Adopted by the AMCP Board of Directors, February 1994