09/15/2014
https://www.congress.gov...
"That is why Senator Specter and I introduced Nino's Act, S. 406, which will allow children to transition out of successful treatment in NIH studies without a gap in treatment."
"How many of these are rare diseases? Is there any effort underway to expand the number of diseases we screen for? Why or why not?"
"One challenge that many of the witnesses today spoke of is the need to incentivize the device and drug/biotech industry to develop therapeutics for rare, pediatric diseases."
"If the treatment is a drug that has not been approved by the Food and Drug Administration or has not been specifically approved for a child's particular disease, then insurance companies typically wil..."