Nature: Why Children Have to Wait Years for New Drugs

Nature recently interviewed pediatric gastroenterologist David Ziring, MD, associate director of the Pediatric Inflammatory Bowel Disease Program at Cedars-Sinai Guerin Children's, about how the scarcity of clinical trials for children further delays an already lengthy approval process for new pediatric medications.

Ziring, an associate professor of Pediatrics at Cedars-Sinai, told Nature that some physicians cope with the lack of new medications for children by prescribing drugs for their young patients that are only approved for adults. But the approach is problematic, he said.

“The drugs act on children’s bodies very differently,” Ziring told Nature, adding that without clinical trial data to guide physicians, “we’re left using either drugs that are 30 to 40 years old and less effective, or trying to use the most recently approved drugs and trying to justify to insurance companies that, despite its lack of an FDA [U.S. Food and Drug Administration] label for pediatrics, we feel that it would be safe and effective.”

Ziring told Nature that he has advocated for faster authorization of adult drugs for pediatric use, but it still usually takes at least seven years.

“We’ve made very little progress,” he said. “The community of pediatric sub-specialists that I belong to has become very frustrated.”

Ziring added that pharmaceutical companies historically haven’t been incentivized financially to move quickly on pediatric clinical trials, which are costlier and lengthier than adult ones because participants are scarce. Hesitant parents concerned about potential long-term side effects of experimental drugs pose an additional ethical challenge to researchers, who can struggle to even start a pediatric trial.

But Ziring told Nature that he remains optimistic that the timeline can improve.

“If there’s a commitment from pharma and they work closely with the FDA, that is very doable,” he said.

Click here to read the complete article from Nature.