10/04/2011
https://www.congress.gov...
"I fear it will discourage research and have an impact on the pipeline of drugs."
"More than half of the 400 million people in the world who are affected by rare diseases are children, and about one-third of those children will sadly die before the age of five."
"Drug development is hamstrung by the fact that we simply do not have well characterized the endpoints for most rare diseases."
"Dr. Esham accelerated approval is a critical regulatory pathway that provides timely review of treatments for patients with serious and life threatening conditions for which there is an unmet need."