REIMBURSEMENT FOR UNLABELED USES OF FDA-APPROVED DRUG PRODUCTS

To support third-party reimbursement for FDA-approved drug products appropriately prescribed for unlabeled uses.

Rationale

Off-label use of drug products includes any use of a drug product for a diagnosis, combination with other medications, dosage, frequency, route of administration, place in therapy, or patient population that is not specifically approved by the U.S. Food and Drug Administration (FDA) and contained in the drug labeling. Drug products’ labeling often fails to represent the most current therapeutic information, because making changes to FDA labeling is a time-consuming and expensive process and medical knowledge is constantly expanding based on evolving evidence.

Once a drug is FDA-approved for a specific indication, it can legally be used for any indication, and off-label prescribing is common. Researchers have conservatively estimated that it accounts for 10-20% of all prescriptions, but the practice is much more common in specific patient populations (e.g., children, geriatric patients, and patients with life-threatening or terminal conditions).

In many clinical situations, off-label use represents a therapeutic approach that has been extensively studied, is supported by the medical literature, and is most appropriate for the patient. Failure to recognize these circumstances or, more importantly, regarding such uses as unapproved or experimental, may restrict patient access to effective drug therapies. Some degree of flexibility must be maintained to optimize patient outcomes and allow for individualized care. While the ultimate responsibility for the safety and efficacy of off-label use resides with the prescriber, hospital and health-system pharmacy and therapeutics committees often develop policies and procedures for managing off-label medication use, with the goal of providing access to the most appropriate, effective treatment for each patient. Although a distinction must be made between evidence-based and inappropriate off-label use, the clinical judgment of healthcare practitioners and experts, as reflected in peer-reviewed publications, clinical practice guidelines, and approved compendia, provides better guidance than FDA labeling alone.