The U.S. Food and Drug Administration today 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,” said
Susan Cummins, M.D., M.P.H, chief pediatric medical officer in the FDA’s
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.”
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 (ECMO) 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. Rather than having to show a reasonable assurance of
effectiveness, devices submitted under the HDE marketing route need to
prove that the probable benefit from use of the device outweighs the
probable risk of illness or injury from its use to obtain the FDA’s
approval.
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.
The FDA’s Orphan Products
Grant Program supported the U.S. clinical trials for the EXCOR Pediatric
System with grants of $400,000 per year for three years.
For more information:
The
FDA, an agency within the U.S. Department of Health and Human Services,
protects the public health by assuring the safety, effectiveness, and
security of human and veterinary drugs, vaccines and other biological
products for human use, and medical devices. The agency also is
responsible for the safety and security of our nation’s food supply,
cosmetics, dietary supplements, products that give off electronic
radiation, and for regulating tobacco products.
