Off-label Use Of Medical Devices Research Articles

PEDT-9 A study of NovoTTF-100A to expand the regulatory indication for childhood glioblastoma through a pediatric clinical trial based on the Advanced Medical Care system

Background: Tumor-treating fields (TTF) are alternating electric fields applied continuously to the brain by attaching 2-pair arrays on the scalp. Although TTF therapy has demonstrated efficacy against supratentorial glioblastoma (GBM) in adults, its safety and efficacy in children have not been confirmed. In Japan, off-label use of medical devices is almost impossible because the national health insurance system does not cover the cost of off-label use of drugs and medical devices. Therefore, TTF therapy cannot be applied to the treatment of pediatric GBM.[Objectives] The investigator-initiated clinical trial aims to expand regulatory approval of TTF therapy for pediatric GBM treatment based on safety and exploratory efficacy data. Methods: Patients aging between 5 and 17 years with histopathological diagnosis of GBM (either newly diagnosed or first-recurrence), which located in the supratentorial region would be included. All the patients will receive TTF therapy for 28 days per course for up to 26 courses until the end-of-therapy criteria are met. The primary endpoint is the adverse event rate with causality. The secondary endpoints include various time-to-event measures and QoL. In total ten patients will be enrolled. Current Status: Discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) led to a tentative consensus that the accumulated data on the efficacy of NovoTTF-100A for adult GBM may be extrapolatable to pediatric GBM if the trial is able to demonstrate efficacy equivalent to that found in previous, adult studies. On the other hand, the combination of the pediatric safety data gathered in this trial and the findings of international studies, including clinical trials and post-marketing surveillance studies, may expedite approval of the device for pediatric GBM treatment. The trial started patient enrollment in April, 2021 with the supervision of the Advanced Medical Care administration system and is currently awaiting the first eligible patient.

Ethical and legal problems arising from off-label use of medical devices and some solution recommendations

The off-label use of medical devices is becoming increasingly widespread. Though the off-label use of drugs has been controlled with the establishment of legal and ethical rules, similar regulations have not been implemented for medical devices. Legal gaps in this field, and the broad initiative granted to physicians have led to the emergence of problems in practice, and physicians are vulnerable to potential criminal sanctions that may emerge as a consequence of these off-label applications. Since the off-label use of medical devices cannot rely on scientifically proven, evidence-based information, evaluation of the risks creates critically important ethical problems that may have serious deleterious effects, despite the good intent of a physician tying to be helpful. During the process of obtaining informed consent from the patient for the off-label use of a device, the physician has crucial responsibilities. The ethical responsibilities of physicians particularly increase in vulnerable patient groups that should be protected from harm. In addition to the physician, the family members who make decisions on behalf of the patient also assume some responsibilities. The development of ethical norms and guidelines enforced by legal regulations concerning this issue, an effective role for ethics committees in the decision-making process, and collaboration between patients, scientists, nongovernmental organizations, medical device companies, specialists in the field of medical ethics, and legal authorities will be important in solving this problem.

Evolving Medical-Legal Issues in Breast Imaging

Because breast imaging practice is associated with a high number of medical malpractice lawsuits, understanding basic medical-legal parameters becomes even more important as new technology and conditions of practice evolve. Incorporating new supplementary breast screening in strategizing a response to the importance of breast density, recognizing the purpose and ramifications of issuing diagnostic imaging reports, understanding the role of expert witnesses in assisting standard of care determinations and employing reasonable applications for off-label use of medical devices exemplify the relevance of integrating defensible practice patterns in a changing medical environment. This article addresses these issues and provides a contextual discussion so that physicians and facilities are educated in their selection of a wide variety of approaches that can be applied to clinical use.

Outcomes Associated with the Off-label Use of Medical Devices in Congenital Heart Disease at a Single Institute.

Background and ObjectivesWhile the off-label use of implantable medical devices for treating congenital heart disease is not uncommon, the present conditions and outcomes of their off-label use have rarely been described. Therefore, this study evaluated the prevalence and results of using implantable devices off-label to treat congenital heart disease at a single institute.Subjects and MethodsThis was a retrospective study based on the medical records of catheter-based interventions for congenital heart disease performed from July 1, 1995 to June 1, 2015. The inclusion criterion was the off-label use of an implantable device. Patient demographic data, procedural success, and follow-up status regarding late complications were investigated, and the results of the off-label use of each device were compared.ResultsOff-label use occurred in 144 of 1730 interventions with device implantation, accounting for 8.3% of the interventions. The median patient age and mean body weight were 51.0 months and 16.3 kg, respectively. Immediate and late failures were found in 9 cases, and 3 cases of mortality were not directly related to the devices used. The overall success rate was 93.8%. There were no long-term complications of the off-label use of occlusion devices. No procedural failures resulted from stent implantation, but one case of stent malposition and two cases of stent fracture were identified after procedure completion.ConclusionIn general, the off-label use of implantable devices for treating congenital heart disease is safe and effective.

Off-Label Use of Medical Devices in Children.

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.

TCTAP A-071 Cardiac Intervention with Off-Label Use of Medical Devices in Congenital Heart Disease

TCTAP A-071 Cardiac Intervention with Off-Label Use of Medical Devices in Congenital Heart Disease

The Ethics of Refusing a Request for Off-Label Deep Brain Stimulation Currently in Clinical Trial

Physicians must ensure that patients understand that the principal aim of research is to increase generalizable knowledge. However, patients may hope for individual benefit as a secondary goal of participation. Highly motivated patients may request treatment off label if trial enrollment has ended, leading to ethical dilemmas for clinicians. This case uses early deep brain stimulation (DBS) as an example for exploring the ethically relevant questions related to the off-label use of medical devices.

A surgeon’s perspective regarding the regulatory, compliance, and legal issues involved with physician-modified devices

Physician-modified endovascular devices are becoming commonplace in a modern climate where innovation outpaces regulated technological advancement. Off-label use of medical devices occurs on a daily basis throughout many institutions across the United States and when performed by physicians, is both legal and unregulated. The purpose of this invited commentary is to review the regulatory, compliance, and legal issues regarding the practice of medical device modification.

Off-label use of medical devices in pediatric interventional cardiology: prerogative or necessity?

The history of medicine is characterized by innovation, through the application of the latest in biomedical science and technology to the bedside. In many ways, using approved medical devices for off-label indications exemplifies this spirit of innovation. Yet there are clear drawbacks to this practice, which include the safety to the patients themselves, the entrepreneurial nature of industry and educational support for clinicians. The issue of off-label use of devices is especially prescient among pediatric cardiac populations in which the majority of patients who undergo interventional catheterization procedures are treated with a device for an indication outside of its labeled uses. The scope of this practice has recently been documented and defined, helping to bring attention to the need for pediatric-specific devices. There remains a critical need to enhance cardiac device review and approval processes to include pediatric applications.

Off-Label Use of Medical Devices in Radiology: Regulatory Standards and Recent Developments

The US Food and Drug Administration (FDA) enjoys a broad legal mandate to ensure that medical products are safe and effective for their intended uses, authority that extends to the medical devices that make modern radiology possible. Under FDA's regulatory framework, medical devices are cleared or approved for specific indications for use that are described in the labeling provided with the devices. This article outlines this framework as well as the regulatory and legal implications of using medical devices in a manner inconsistent with their FDA-cleared or FDA-approved labeling.

Pediatric Interventional Cardiology in the United States is Dependent on the Off-label Use of Medical Devices

A substantial unmet medical device need exists in pediatric care. As a result, the off-label use of approved devices is routine in pediatric interventional cardiology, but the extent and nature of this practice has not been previously described. The purpose of this study, therefore, is to evaluate the prevalence and nature of off-label cardiac device use in an active pediatric interventional program in the United States. This study is a retrospective review of all interventional cardiac procedures performed at our institution from July 1, 2005 to June 30, 2008. Diagnostic (noninterventional) catheterizations, myocardial biopsies, invasive electrophysiology studies, and studies involving investigational devices were excluded. Interventions performed were compared with the manufacturer's labeled indications for each device. During this 3-year period, 473 patients (median age 4.1 years) underwent 595 transcatheter interventions. An approved device was utilized for an off-label application in 63% of patients, and in 50% of all interventions performed. The most frequent off-label procedures were stent implantations (99% off-label), balloon dilations (78% off-label), and coil embolizations (29% off-label). In contrast, the off-label use of septal and ductal occluders was relatively uncommon. In our routine (noninvestigational) practice of pediatric interventional cardiology, 63% of patients underwent procedures utilizing medical devices for off-label indications. These data underscore the need to enhance cardiac device review and approval processes in the United States to include pediatric applications.

The “art” of medicine and the “smokescreen” of the randomized trial off‐label use of vascular devices

Once a device is approved for sale in the United States by the Food and Drug Administration (FDA), it can legally be used by doctors to treat any condition a physician determines is medically appropriate. Based on postmarket published data and physician procedural experience, this may even become the standard of care when an alternative device either does not exist or is inferior in performance, even before FDA approval. This right of physicians to practice medicine without FDA approval is Federal law. The off-label use of medical devices for the treatment of peripheral vascular disease has recently become the latest target by groups with interests that have little to do with patient care. This interference has begun to negatively impact the latitude necessary for physicians to best treat their patients.

Off-label use of medical devices: What every radiologist should know

Off-label use of medical devices: What every radiologist should know