the Orthopaedic forum Off-Label Use of Pediatric Orthopaedic Devices: Important Issues for the Future Evan D. Sheha, BS, Qusai Hammouri, MD, Brian D. Snyder, MD, PhD, Robert M. Campbell Jr., MD, Michael G. Vitale, MD, MPH, and the POSNA/SRS Task Force for Pediatric Medical Devices Although off-label use of drugs and medical devices is a com- mon 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 pro- cess 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 in- volves a precarious balance between medical practice, federal policy, and ethical responsibility. The long and invariably expen- sive process of gaining FDA approval requires that a manufac- turer 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 ram- ifications and the ethical issues surrounding off-label use that now face stakeholders in the arena of pediatric medical devices will be vital to the identification of future challenges and the development of practical solutions. An Overview of FDA Policy FDA labeling restricts the promotion of a product by manufac- turers for uses beyond the approved indications. However, this law does not interfere with a practitioner’s authority to use any legally marketed device for any medical condition or disease that justifies its use. As a result, off-label use has become a common practice that is often supported by clinical evidence and profes- sional guidelines; the economic and practical barriers to adding Disclosure: None of the authors received payments or services, either directly or indirectly (i.e., via his or her institution), from a third party in support of any aspect of this work. One or more of the authors, or his or her institution, has had a financial relationship, in the thirty-six months prior to submission of this work, with an entity in the biomedical arena that could be perceived to influence or have the potential to influence what is written in this work. No author has had any other relationships, or has engaged in any other activities, that could be perceived to influence or have the potential to influence what is written in this work. The complete Disclosures of Potential Conflicts of Interest submitted by authors are always provided with the online version of the article. Peer Review: This article was reviewed by the Editor-in-Chief and one Deputy Editor, and it underwent blinded review by two or more outside experts. The Deputy Editor reviewed each revision of the article, and it underwent a final review by the Editor-in-Chief prior to publication. Final corrections and clarifications occurred during one or more exchanges between the author(s) and copyeditors. e21(1) COPYRIGHT Ó 2014 BY THE J OURNAL OF BONE AND J OINT SURGERY,I NCORPORATED J Bone Joint Surg Am. 2014;96:e21(1-6) d http://dx.doi.org/10.2106/JBJS.M.00288