Despite widespread therapeutic needs, the majority of medical and surgical devices used in children do not have approval or clearance from the Food and Drug Administration (FDA) for use in pediatric populations. The clinical need for devices to diagnose and treat diseases or conditions occurring in children has led to the widespread and necessary practice in pediatric medicine and surgery of using approved devices for "off-label" or "physician-directed" applications that are not included in FDA-approved labeling. This practice is common and often appropriate, even with the highest-risk (class III) devices. The legal and regulatory framework used by the FDA for devices is complex, and economic or market barriers to medical and surgical device development for children are significant. Given the need for pediatric medical and surgical devices and the challenges to pediatric device development, off-label use is a necessary and appropriate part of care. In addition, because of the relatively uncommon nature of pediatric conditions, FDA clearance or approval often requires other regulatory pathways (eg, Humanitarian Device Exemption), which can cause confusion among pediatricians and payers about whether a specific use, even of an approved device, is considered experimental. This policy statement describes the appropriateness of off-label use of devices in children; the use of devices approved or cleared through the FDA regulatory processes, including through the Humanitarian Device Exemption; and the important need to increase pediatric device labeling information for all devices and especially those that pose the highest risk to children.

https://pediatrics.aappublications.org/content/pediatrics/early/2016/12/22/peds.2016-3439.full.pdf

Off-Label Use of Medical Devices in Children

Pediatric medical device development by surgeons via capstone engineering design programs

Surgeons frequently use orthopedic devices in an off-label manner. It is the responsibility of the surgeon to be familiar with the product and to use it based on scientific and clinical rationale. However, such practices are poorly publicized, and there remains paucity in the current literature surrounding the off-label use of orthopedic devices. Furthermore, the off-label use of devices is fraught with difficulty, including limitations in investigational studies and insurance reimbursement or coverage of use. Surgeons should be aware of the common off-label use in orthopedics. This review provides an overview of the off-label use in orthopedics and the obstacles associated with it; this review also offers clinical examples of its implementation.

Off-Label Use in Orthopaedic Surgery.

Abstract Introduction The use of magnetic resonance imaging (MRI) with a magnetic intramedullary lengthening nail in place is contraindicated per the manufacturer due to the concern of implant activation and migration. A prior in vitro study did not confirm these complications only noting that a 3.0 T MRI weakened the internal magnet. Therefore, a retrospective analysis of patients who underwent an MRI with a magnetic nail in place was performed to determine if any adverse effects occurred in the clinical setting. Materials and methods A retrospective review of all patients who underwent an MRI with a magnetic lengthening nail in place was performed. The time spent being imaged in the MRI, number of times the patient entered the MRI suite, and the images obtained were recorded. Radiographs were performed before and after the MRI to determine if any hardware complications occurred. The patients were monitored for any adverse symptoms while they were in the suite. Results A total of 12 patients with 13 nails were identified. Two patients underwent imaging with a 3.0 T MRI while the remaining 10 underwent imaging with a 1.5 T MRI. Each patient entered the MRI suite 2.1 times and spent an average of 84.7 min being imaged in the MRI (range 21–494). No patients noted any adverse symptoms related to the nail while in the suite and no hardware complications were identified. Conclusion MRI appears to be safe with a magnetic nail in place and did not result in any complications. Given the manufacturer’s recommendations, informed consent should be obtained prior to an MRI being performed and a 3.0 T MRI should be avoided when possible if further activation of the nail is required. 

Clinical compatibility of magnetic resonance imaging with magnetic intramedullary nails: a feasibility study.

Background: Despite a tremendous increase in the number of orthopaedic devices authorized by the U.S. Food and Drug Administration (FDA), novel devices designed specifically for the pediatric population remain sparse. Surgeons frequently repurpose adult implants for “off-label” use in pediatric patients, with both legal and technical ramifications. This study seeks to objectively quantify and characterize the nature of pediatric device innovation over time. Methods: The FDA employs 4 pathways for assessing safety and effectiveness of novel devices prior to authorization. Perceived device risk and novelty determine the pathway. Orthopaedic devices were identified from the FDA’s online database. All devices approved since inception via the Humanitarian Device Exemption, Pre-Market Approval, and De Novo regulatory pathways were included and grouped as “highly innovative.” Because of their number and the rapidity of their development, the evaluation of 510(k) devices was limited to those cleared from January 1, 2018, to December 31, 2022. Such 510(k) devices make up ∼97% of devices and by definition are less risky and less novel. Approval statements were assessed for pediatric indications within the approved labeling. As a secondary analysis, the impact of company size on developing a product with a pediatric indication was analyzed. Results: Of the 1,925 devices cleared via the 510(k) pathway, 9 (0.5%) were designed exclusively for pediatrics and 160 (8.3%) included pediatric indications. Five of the 9 pediatrics-only devices were for spine and 4 were for trauma indications. Of the 97 highly innovative devices, only 2 (2%) were exclusively pediatric and another 2 (2%) included pediatric indications. The 2 pediatrics-only devices were for the spine. Large and medium-sized companies were 1.9 times and 1.6 times more likely to bring to market a device with pediatric indications than a small company, respectively. Conclusions: Innovation for pediatric orthopaedic devices lags substantially behind that for adult orthopaedic devices. These findings are consistent with clinical experience and the common practice of modifying adult implants for “off-label” use in pediatric patients. Despite long-standing efforts to stimulate innovation for this vulnerable population, our results suggest little progress. 

Pediatric Device Innovation

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 …

Off-label use of pediatric orthopaedic devices: important issues for the future.

Handle everyday research tasks with reliable, citation-backed results

Your personal Research Agent to handle research tasks with citation-backed results

Popular Tasks used by Researchers

How can I help with your research?

Meet SciSpace

Get more enhanced response by uploading the PDFs you want me to reference.

No relevant PDFs in your library

SciSpace is the AI research assistant for academics. Run systematic literature reviews on 280M+ papers, and write papers with cited sources. Trusted by 1M+ students, PhDs & researchers.

SciSpace | AI for Research

Analyze PDFs

Code & Manuscripts

Funding & Grants

Literature & Patents

Medical & Clinical Data

Systematic Review

Visualize & Present

Web & Data

Build a Google Scholar-like website for your research.

Build a website

Create charts and images for your research

Create a Chart

Write a paper for submission to a journal

Draft a manuscript

Patent Search

Design eye-catching scientific posters in minutes.

Scientific Poster Generation

Systematic Literature Review

One task is running at the moment. Your messages will be shown right after.

Drag and drop or click here to browse

Loved by <highlight>1 million+</highlight> researchers

Extract a list of specific topics and their sources from unstructured text

Topics

Compare and analyze relevant papers that matches with your search

Papers

Get insights from PDFs and bookmarked papers from your library

My library

Recent searches

Try searching for:

Catch AI-generated content in scholarly and non-scholarly content

{ai} Detector

Ai Writer

Get PDF Summaries, highlighted text explanations 

Chat with PDF

Effortlessly create in-text citations and bibliographies in APA and 2,500 other formats

Citation generator

Get explanations, summaries, and answers on academic papers

Ease up your research workflow with {scispace}'s cohort of exciting AI tools

Elevate your academic writing skills and convey your ideas the way you want

Paraphraser

Explore our range of reading and writing tools

Your file is being prepared and should be ready in a few minutes. If it's a large file, it might take a bit longer. You can close this window, and we'll email you the file when it's done.

You have reached a maximum limit of <strong>{limit}</strong> columns in the table. Remove at least <strong>1</strong> column to add or create another one.

Posna

Author Tools

Chat about Author

Papers

Off-label use of pediatric orthopaedic devices: important issues for the future.