Off-Label Use of Pediatric Orthopaedic Devices: Important Issues for the Future

Abstract

Although off-label use of drugs and medical devices is a common practice in the United States, it comes with its own set of medicolegal and ethical issues. In pediatric orthopaedics, the widespread use of off-label medical devices stems jointly from the physician’s obligation to serve the patient’s best interest and the financial, political, and procedural impediments resulting from the current legislative language. Although a number of policy changes intended to streamline the device approval process by the U.S. Food and Drug Administration (FDA) have been passed in the past decade, more can be done to hasten these mechanisms while maintaining focus on the safety and the efficacy intended by FDA policy. Through a discussion of the dynamics of medical device regulation in the U.S., we aim to shed light on the practical, legal, and ethical challenges facing stakeholders, and present a framework to facilitate the FDA approval process for pediatric orthopaedic devices. The FDA defines the uses for which drugs and devices can be marketed by their manufacturers, while the common practice of off-label, or physician-directed, use refers to use of these products for non-FDA-approved indications. A simple definition of FDA approval and its relationship to off-label use belies the complex issues of this common practice, which involves a precarious balance between medical practice, federal policy, and ethical responsibility. The long and invariably expensive process of gaining FDA approval requires that a manufacturer show clinical evidence of safety, efficacy, and clinical benefit to the FDA’s Center for Drug Evaluation and Research, which then gives a product its “label.” This FDA policy has come under intense scrutiny in the past decade, and has sparked debate as to how we might remove obstacles to pediatric device development for pediatric patients in need. A discussion of the practical ramifications and the ethical issues …