CHICAGO (Reuters) - Every year, Chicago-based cardiologist Ziyad Hijazi accompanies two or three children and their families to his native Jordan for heart operations using medical devices that are not approved in the United States.
In one such case, Hijazi implanted a device to close a hole between the lower chambers of the heart in a child from Massachusetts. The device, called an amplatzer muscular VSD, manufactured by Minneapolis-based AGA Medical, was available for 9 years in Jordan before it was approved in the United States in 2007.
According to Hijazi, who is chief of pediatric cardiology at Rush University Medical Center, and other doctors, children are getting worse treatment in the United States, and have even died, because pediatric medical devices are not approved.
Hijazi said that more than 90 percent of the medical devices he uses on children are “off-label,” meaning that they are approved by the U.S. Food and Drug Administration for other uses, for example for use in adults.
“We take responsibility as physicians for using unapproved devices on kids,” he said.
From 1989 to 2000, only one stent — a tubular device that props open vessel walls — was appropriate to use in children, said Thomas Forbes, director of cardiac catheterization at Children’s Hospital of Michigan in Detroit.
Stents are delivered through catheters, usually entering the body through the large vessel in the groin, and then threaded through the body to the destination.
“In the ‘90s, we lost lives in the cath lab. Patients have died on the table because we were using stents that were made for adults and weren’t flexible enough,” Forbes said.
Making do with adult-sized devices rather than using ones designed for children can be the difference between “potentially dangerous care and very good care,” he said.
There are currently three stents that can be modified for pediatric use, but none of them is approved for use in children in the United States, Forbes said.
While much of the focus is on cardiology, pediatric doctors from other specialties, like orthopedics, echo these concerns, saying they either use devices approved for adults or hand-make their own devices.
One factor is that companies that make medical devices focus on adults because the market is bigger. Heart diseases in children, for example, are more likely to be congenital, and rare, while in adults they are more likely to be progressive, and common.
A law signed late last year provides financial incentives to companies for making devices for children, but also requires those companies to track patients at their own expense.
“It’s a paperwork nightmare. They have to commit resources and follow these patients forever,” Forbes said. “If I’m a J&J stockholder, I’m saying, ‘I love kids, I’d love to help them out, but move on.’”
Tara Federici of AdvaMed, the trade association representing medical device makers, said the industry is looking to a meeting sponsored by the National Institutes of Health in Washington on July 23 for guidance.
“First, we need to understand what the needs are and I’m hopeful that NIH will help catalogue the needs,” said Federici, who noted that part of the problem was that physicians do not communicate effectively with industry.
Beyond that, financial, ethical and regulatory hurdles abound.
One solution might be if the government offered a significant tax credit for research and development in this area, but that is not being discussed, Federici said.
Richard Ringel, a pediatric cardiologist at Johns Hopkins Hospital, said he is awaiting the next innovation in cardiovascular medicine: stents that dissolve inside the body
“We’re all anxious for the dissolvable stent...but do you think Johnson & Johnson is going to be interested in doing this for babies? No, they want to do it for (adult) coronary patients because the numbers are so much larger,” Ringel said.
Privately held NuMed Inc has filled some of the gaps in pediatric cardiovascular medicine, but the company is small and limited in its resources.
Edwards Lifesciences, the world’s biggest maker of heart valves, is one example of a public company that has developed a product for children — a blood-oxygen monitor — but the company acknowledges the limitations.
“We have not cracked the code on this,” said Edwards Chairman and Chief Executive Michael Mussallem, who is president of AdvaMed.
“Development (of pediatric devices) is time-consuming, there are regulatory hurdles and how do you recoup your investment? The math generally does not work,” he said.
Sales of Edwards’ first pediatric device generated sales of just a couple million since its launch last year. The company said the potential of the market is $30 million to $35 million, a fifth to a quarter of the adult market.
Moreover, ethical issues associated with conducting clinical trials on children and legal liability are two other big obstacles, Mussallem said.
“There’s not a climate for innovation. A great percentage of devices are used off-label to accommodate children’s needs. Physicians are just used to getting by. It all comes back to the economics.” Mussallem said. “There is a hole there and I don’t know how to bridge it. We need public or private funding.”
Bram Zuckerman, Director of the Division of Cardiovascular Devices at the U.S. Food and Drug Administration, which regulates and approves medical devices, said part of the challenge is to get physicians to communicate better with both industry and regulators.
“The agency is very sensitive right now to the need to foster and develop pediatric devices and we’re looking to develop many different pathways,” Zuckerman said.
He said FDA will be more flexible in the way it evaluates data on devices.
For example, he said FDA recognizes that randomized controlled clinical trials — the gold standard in which participants receive the device while others receive standard treatment — are not always possible.
“That doesn’t mean we want to skimp on other key features of clinical trial designs,” he said.
Daniel Schultz, director of FDA’s Center for Devices and Radiological Health, added, “Our frustrations are the same as everyone else’s. We’d like to work with companies, but we don’t have the kinds of comprehensive studies that we’d like. No one has ever done a broad study of gaps and needs and that would be really helpful to direct our efforts.
“Surgeons tend to be inventive in terms of being able to adapt things that may not be perfect. I think we all recognize that it would be better if we had devices developed for specific situations,” said Schultz.
Reporting by Debra Sherman; Editing by Eddie Evans