by Julie E. Kurzrok
On December 16, FDA approved EXCOR®, the first ventricular assist device (“VAD”) designed for use in children. Berlin Heart, a closely-held German company, designed the mechanical heart device, which is already approved for use in Europe and Canada. FDA designated the device as a Humanitarian Use Device, which means it is used to treat or diagnose a disease affecting less than 4,000 people in the United States each year.
FDA approved EXCOR® under the Humanitarian Device Exemption (“HDE”), which allows for approval without efficacy studies, and only requires a demonstration that the probable benefit of the device outweighs the risks from its use and that the device does not pose an unreasonable or significant risk of illness or injury. The applicant must also show, for example, that there is no comparable device available to treat the disease.
EXCOR® is a mechanical cardiac support system designed specifically for children with congenital or other heart defects that prevent their hearts from pumping enough blood into their bodies. EXCOR® consists of one or two external pumps connected to tubes which are implanted into the ventricles and major arteries, and a separate driving unit. Each external pump controls one ventricle. The device comes in different sizes to accommodate newborn babies through teenagers, and functions to bridge patients while they await a heart transplant. According to FDA, “[I]n infants, the median waiting time for a donor heart is 119 days [and] . . . 12-17 percent of children and 23 percent of infants die while on the wait list for a heart transplant.”