Pharmaceutical companies would have to spend a lot of money on pediatric anticoagulation studies for diseases that are rare among these patients, explained Michael A. Portman, MD, FAHA, of Seattle Children's Hospital.
Pharmaceutical companies would have to spend a lot of money on pediatric anticoagulation studies, for diseases that are rare among these patients, and the statistical methods differ as well for adult studies, explained Michael A. Portman, MD, FAHA, director, Pediatric Cardiovascular Research, Center for Integrative Brain Research, and professor of pediatrics at Seattle Children's Hospital and the University of Washington in Seattle.
Why have there been few studies on the use of pediatric anticoagulation?
I think it’s most due to the interest [from] the pharmaceutical companies and spending the money. They would have to spend a lot of money on relatively few patients who would take their medications. So for this study, we were really fortunate in that Daiichi Sankyo sponsored the study. We enrolled quite a few patients for a pediatric study; we had, I believe, 168 patients enrolled—so that's pretty large for a pediatric study. In contrast, for an adult study, you would have thousands of patients. So there are difficulties even when the drug company sponsors [a study]; there is a rarity of the patients, because these are rare diseases. And most of the companies and actually the FDA demands certain statistical methods. And you really can't use those statistical methods in children because of the rarity of the diseases. So that's another barrier, the type of information that the FDA requires for approval of those drugs, although I think they're becoming more lenient, understanding that we can't use adult type trial design in children.