Doctors and experts are focusing on those side effects, which include developmental delay, growth problems, infertility, hearing loss, kidney and heart problems and the possibility of a second cancer developing, Katzenstein said.
"Children are not just small adults," he said. "They're different. They handle drugs differently. They have different side effects."
"One of the key things we hope for is that Congress acts to put incentives in place that will allow industry to bring their expertise to the table," Adamson said. "There's not going to be increased money. ... [Just] incentives that help level the playing field."
"There are too many children who are in a similar situation to Mollie, where the outcome isn't what it needs to be and we're at a point where we're going to have tremendous discoveries," he said. "We need to turn those discoveries into better treatments."