Hardly any of the myriad drugs and devices developed for the multibillion-dollar market for cardiovascular disease are designed with kids in mind. Children with heart disease represent too small a segment of that market to justify companies’ investing the time and resources needed to develop specialized products. Litigation worries over products intended for children—and the challenge of conducting clinical trials for treatments often administered to newborns—are other impediments.
It all adds up to what Jim Lock, chief of cardiology at Children’s Hospital Boston, calls a “profitability gap” between the market for children and the much-larger business of treating adults with heart disease.
Matthew Emmerling’s doctors had to use an adult-size kidney balloon to open his aortic valve when he was a few days old.
The result is that in treating some of medicine’s sickest patients, physicians often must rely on instinct, back-of-the-envelope calculations and anecdotal case reports swapped at medical meetings, instead of the more rigorous clinical evidence that underpins much adult heart treatment.
In a study presented last March at a meeting of the American College of Cardiology, Dr. Beekman and his colleagues found that 63% of 473 patients treated at Cincinnati Children’s Hospital between 2005 and 2008 got devices that hadn’t received regulatory approval for the purposes for which doctors used them. Doctors at Children’s Hospital Boston say that of some 1,000 tubular devices—known as stents—that doctors put in vessels in children’s hearts in recent years, not a single case involved a use that had been approved or evaluated by the Food and Drug Administration.
