NORD has submitted the following Letter to the Editor of the Journal of the American Medical Association (JAMA):
The recent study evaluating the evidence supporting FDA approval of novel therapeutic agents has generated debate about FDA exercise of flexibility and judgment in approving new drugs. FDA Commissioner Margaret Hamburg, MD, wrote a blog explaining why FDA supports a flexible approach to drug development. read more >
The Food and Drug Administration’s Office of Orphan Products Development (OOPD), in collaboration with the FDA Center for Drug Evaluation and Research (CDER), has launched a web-based educational tool for rare disease patients, advocacy groups, researchers and industry on various FDA-related topics. read more >
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 >
Posted at December 1, 2013 8:09 pm by Paul Melmeyer
Over the past year, Congress has taken several actions (or inactions) that seemingly nobody wanted; indiscriminately slashing budgets as part of the Sequester and shutting down the government for 16 days. read more >