Objective: 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.
Study design: 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.
Results: 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.
Conclusions: 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.