Posted at January 22, 2014 7:11 pm by Leleah Robinson
Leleah Robinson, NORD’s Special Assistant to the Vice President of Public Policy, recently attended the FDA public workshops. This blog provides her insight into that event.
In July 2012, President Obama signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) which requires the FDA to hold at least one public meeting to “encourage and accelerate the development of new therapies for pediatric rare diseases”. read more >
Obstacles in rare disease research and orphan drug development continue to include low patient numbers, difficult recruitment, and insufficient scientific and medical knowledge such as lack of natural history data and known, relevant endpoints. Until now, collection of such information has resided primarily with clinical experts and drug development companies. But the recognition of patient registries as key determinants in accelerating research and drug development progress has propelled significant interest from patient organizations over the last decade. Today, read more >