The U.S. Food and Drug Administration approved a medical device that supports the weakened heart of children with heart failure to help keep them alive until a donor for a heart transplant can be found. The mechanical pulsatile cardiac assist device is called the EXCOR Pediatric System, made by a German company, Berlin Heart. The device comes in graduated sizes to fit children from newborns to teens. “This is a step forward, it is the first FDA-approved pulsatile mechanical circulatory support device specifically designed for children.” – Susan Cummins MD Chief Pediatric Medical Officer FDA Center for Devices and Radiological Health “Previous adult heart assist devices were too large to be used in critically ill children to keep them alive while they wait to get a new heart,” she stated. The device consists of one or two external pneumatic (driven by air) blood pumps, multiple tubes to connect the blood pumps to heart chambers and the great arteries, and the driving unit. Heart failure in children is much less common than in adults. Heart transplantation offers effective relief from symptoms. However, far fewer pediatric sized donor hearts are available for transplantation than for adults, limiting the use of heart transplantation in children and prolonging the waiting period until transplant can occur. In infants, the median
waiting time for a donor heart is 119 days. Overall a reported 12-17 percent of children and 23 percent of infants die while on the wait list for a heart transplant. In the primary U.S. study group of 48 patients, the use of the device was found to improve survival to transplant in patients when compared with the use of extracorporeal membrane oxygenation which is the current standard of care, although not FDA approved. Stroke, which can cause serious brain deficits, is a risk of the EXCOR Pediatric System. The EXCOR was designated as a Humanitarian Use Device (HUD) by the Office of Orphan Products Development at the FDA. This designation is for medical devices intended to benefit patients in the treatment or diagnosis of a disease or condition that affects fewer than 4,000 individuals in the United States annually. The device was approved under a Humanitarian Device Exemption (HDE), a type of marketing application that is similar to a premarket approval application in that the level of safety required for approval is the same. The FDA approval of an HDE authorizes an applicant to market the device subject to certain use restrictions. After the passing of the Pediatric Medical Device Safety and Improvement Act of 2007, HUDs intended and labeled for use in a pediatric population are permitted to be marketed for profit.