U.S. doctors push for medical devices for children
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.
REUTERS |