Healthy Skepticism Library item: 7743

Warning: This library includes all items relevant to health product marketing that we are aware of regardless of quality. Often we do not agree with all or part of the contents.

Publication type: Journal Article

Aggarwal K, Cowley EP.
Beyond approved labeling: The USPDI medication information development process.
ASHP Midyear Clinical Meeting 2002;

Abstract:

Prescribers frequently use prescription medications for purposes they consider medically appropriate, even though the Food and Drug Administration (FDA) has not specifically approved them for that particular use. The FDA has long recognized medication use outside the approved labeling within the context of the practice of medicine, however, the FDA also recognizes that off-label use may pose problems. Because this information is not available from product labels, prescribers must obtain data about off-label uses using various sources of supportive evidence from medical literature, textbooks, and symposia. The USP DI Drug Information for the Health Care Professional (USP DI) is a federally recognized source for consensus-based off-label drug use information. Decisions about drug information to be included in the drug monographs are made by the USP Council of Experts information division Expert Committees. These Committees consist of elected volunteers, who work under publicly devised and published rules and procedures that include conflict of interest provisions, ensuring unbiased drug information for use in pharmaceutical care. The unlabeled use information included in USP DI must be supported by “significant evidence of efficacy” defined as a body of medical knowledge sufficient to produce expert consensus that the use of a product in a particular manner constitutes reasonable medical care and current prescribing practice. In this report, we will examine the USP process for generation of an off label use, specifically referring to a recent example of an off-label use developed for darbepoetin alfa. Learning Objectives: 1. Explain the potential advantages and disadvantages of using medications outside approved labeling in the practice of medicine and pharmaceutical care. 2. Outline the USP process by which an off-label use is identified, created, and reviewed. 3. Describe the three classifications given to off label indications in the USP DI. Self-assessment questions: 1. Which of the following is considered an acceptable reference for evidence based medicine? a) Primary clinical trials b) Case reports c) Only A d) Both A and B 2. Reimbursement is available for indications that are not approved by the FDA. a) True b) false 3. Darbepoetin alfa was approved as an off label indication by the USP Expert Committee for treatment of chemotherapy-induced anemia. a) True b) False Answers 1. D; 2. A; 3. A