An unsettling study recently published in the journal Pediatrics shows that most medical devices that the FDA has approved to treat children weren’t tested on them first.
Mostly, such devices were tested in clinical trials on patients at least 18 years old.
One of the study’s authors, Thomas J. Hwang, a research fellow at Harvard Medical School, told Reuters.com, "Children are not simply 'small adults,' and a device found to be safe and effective in adults may have a very different safety and effectiveness profile when used in a pediatric population."
"Without this data, it is difficult for clinicians and parents to make informed treatment decisions that weigh the risks and benefits of a particular treatment," he said.
The practice of ignoring how kids might respond differently to treatments given to adults is hardly new — last year we wrote about how pharmaceutical companies often don’t conduct clinical trials with children for drugs tested on and approved for adults.
The Pediatrics study reviewed testing that had been done on medical devices meant for kids since an act of Congress helped spur their development seven years ago. Researchers analyzed data from the main clinical trial used for the 25 medical devices approved by the FDA for use in patients 21 and younger between 2008 and 2011. Nearly half — 11 — weren’t tested on any patients younger than 21, and only four of the devices had been tested on patients younger than 18.
Three of the devices included in the study were approved specifically for people younger than 18, whom the FDA considers pediatric; the rest were approved for 18- to 21-year-olds.
Researchers said, however, that devices approved only for older adolescents probably are used "off-label" in younger children because, often, there are no alternatives.
Doctors may prescribe a treatment for something other than what it was approved for (“off-label), but it’s illegal for manufacturers to promote such use.
Most of the devices in the Pediatrics study had been approved with a stipulation that their manufacturers conduct post-market trials after they reached the commercial market. The idea was to ensure they were safe and effective. But only three required that youngsters be included in the trials, and none of those was complete.
"Designing devices for children is more complex because they are still growing and may need to have the device for many decades, in contrast to older adults," Dr. Katherine Bates, a cardiology fellow at The Children's Hospital of Philadelphia, told Reuters.
There are ethical and practical issues about using children with rare diseases in clinical trials, even though without them you can’t determine how a device will perform in the long term. And government incentives for conducting clinical trials with children are available for drug manufacturers, but not for device makers.
Some of the devices in the Pediatrics study were approved from observational study data, not the more rigorous results of randomized, controlled trials. The FDA requires at least one clinical trial demonstrating that a device is safe and effective before it approves it for general patient use. But these trials don't necessarily have to include the same type of patients who will get it once it’s marketed.
Just as women don’t necessarily respond to a drug or device as men will, or the elderly versus middle-aged people, children might not either.
Cardiologist Rita F. Redberg, editor of JAMA Internal Medicine, told Reuters “Common sense would dictate that devices would be studied in the appropriate populations before they are used on the market.”
As Reuters notes, approval requirements exempt “high-risk” devices, or those that support or sustain life for patients with rare diseases (fewer than 4,000 people in the U.S. each year); such devices must demonstrate only that they are safe and probably beneficial. But they can’t be sold for profit.
The 2007 act of Congress eliminated the nonprofit provision for devices intended for children, and established grants for nonprofit pediatric device developers.
Three of the 25 devices included in the Pediatrics study invoked the rare disease exemption.
If your pediatrician, or any other doctor, recommends a certain device to treat your child, ask if it has been tested on children, or how much evidence there is that it will be effective and safe. And consider enrolling your child in a trial in order to enlarge the small body of current knowledge about how devices work in children.