Devices for children face slow approval

One-year-old Robbie Moore's legs spring with joy when his mother enters his hospital room. Two pumps rest between his jittery thighs and push blood through his sick heart.

Robbie has lived more than a quarter of his life attached to a Berlin Heart at Medical University of South Carolina's Children's Hospital.

Thick blood-filled tubes snake in and out of his tiny chest, keeping him alive until a donor heart becomes available.

The German-made ventricular- assist device is one of many lifesaving pediatric devices not yet approved by the U.S. Food and Drug Administration.

A number of forces - a cautious approval system and a small number of patients on whom to test and market the devices - frustrate desperate families.

Brittany Moore, 22, Robbie's mother, was surprised to learn the Berlin Heart, widely used in Europe, was not approved here. "We're supposed to be the best," she said.

Doctors and staff at MUSC filled out the laborious paperwork to receive a compassionate waiver. "You're just fighting one battle after another," Moore said.

On April 16, Robbie became the second child to receive a Berlin Heart in South Carolina. In a tragic blindside, about two weeks later, Robbie's father, Nicholas Moore, drowned in a canoe accident in Lake Murray.

"You just keep going," she said of the motivation that compels her between Charleston and her home in Augusta, where she works full-time at a hotel.

Dr. Andy Atz, director of the Pediatric Cardiac Intensive Care Unit at MUSC's Children's Hospital, said he sees both sides to the approval process.

"It is a delicate balance to make sure devices are really safe, and to make sure lifesaving devices and medicines are available when no other option is," Atz said.

As technology advances at a quicker pace, more patients are bumping up against the approval process, he said. Also, manufacturers know that it's easier to get approval in Europe, so they do trials abroad first, he said.

The first Berlin Heart used in the U.S. was eight years ago in Arizona, also under the condition of a special waiver.

Angela Greenwood, mother of the first child in South Carolina to receive a Berlin Heart, said, "We live in America, and we think this is the greatest country in the world."

During the process of seeking a compassionate waiver, her son Joseph Greenwood nearly died, she said. Joseph suffered from cardiomyopathy, a disease of the heart muscle, which was triggered by a viral infection.

He was put on a heart-lung device called ECMO, or Extracorporeal Membrane Oxygenation, an FDA-approved device that is designed to bridge the time from heart failure to transplant.

But ECMO can be used only for short periods, about two weeks, and the patient must be in a medically induced coma. Fluid began to build up in Joseph's lungs when he was on ECMO, Greenwood said.

On April 4, 2007, the 6-year-old boy from Florence received the 89th compassionate waiver granted in North America for a Berlin Heart. The device made him healthy enough to receive his new heart, which came nine days later.

That summer, the Greenwoods traveled to Washington to lobby for more funding for children's hospitals and met with a representative from the FDA.

"It's like this red tape that goes back and forth," she said. A company may not seek approval until a device gets used enough in the U.S., she said.

Now 7 years old, Joseph is about to begin second grade, and he recently led his family along hiking trails in the North Carolina mountains.

Hurdles also affect medicine for children. "The reality is in the U.S. 80 percent of drugs we use in pediatric patients are approved, but not for a pediatric age group," Atz said.

Using medicine "off label" means the drug is approved for other uses, such as in adults.

Scott Bradley, a pediatric cardiothoracic surgeon at MUSC's Children's Hospital, said, "It does not mean it's not safe, or it shouldn't be used that way. It's just physically impossible to test in children with so few cases."

Children tend to be healthy, and severe illnesses are rare, making the pools for testing and marketing small. "It's been difficult to get any company to sink enough money into research and development," Bradley said.

Robbie became sick with respiratory syncytial virus, or RSV, in August 2007. Eventually diagnosed with cardiomyopathy, he was hospitalized in January and again in March, when his heart failed.

"He flat-lined and they shocked him back," Moore said. "The doctor said 99 out of 100 don't come back."

Robbie was airlifted from Augusta to MUSC for specialized care.

The longest a patient has stayed on a Berlin Heart has been just more than a year, Bradley said.

Moore tells herself that "Everything happens for a reason," and "Take it one day at a time." But her true source of comfort is clear when she crawls into her son's hospital bed and smooths his curly hair.

Reach Jill Coley at 937-5719 or jcoley@postandcourier.com.