Ann: MSB Files for FDA Designations in Congenital Heart Disease, page-3

The research and development into rare pediatric diseases have garnered more spotlight attention in recent years thanks to a combination of greater public awareness and newly created laws and legislation. Out of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, Section 529 was added to the FD&C Act — establishing the Rare Pediatric Disease Designation (RPDD) program. The implementation of this program was done with the goal in mind of incentivizing the research and development of new drugs and therapies for rare childhood conditions.


…. the FDA does not require the same level of supportive data showing the effectiveness of a pediatric therapy compared to those therapies seeking Orphan Drug Designation.

“In vitro data supporting the mechanism of action of the drug in the disease or in a related disease may suffice for rare pediatric disease designation, whereas that level of data would not generally suffice for orphan-drug designation.”