04/28/2014
https://www.congress.gov...
"the NIH spends a substantial amount on research on various rare diseases, including those affecting children, but with over 6,000 known rare diseases, not every one has been fully addressed."
"without a statement of policy on rare diseases and orphan products, it is not possible [for the FDA] to ensure consistency in that process."
"the Agency strongly urges Congress to ensure that, if a change is made, the population limit is clearly defined, not variable."
"increasing understanding of the role of genetics in rare diseases will unquestionably have a considerable impact on their diagnosis, prevention, and treatment."