June 10th was an emotional – but very educational – day for everyone who attended the Food and Drug Administration’s public meeting on Patient-Focused Drug Development for Inborn Errors of Metabolism. (more…)
Biologic therapies have been lifesaving and life changing for the patients we represent – more than 30 million Americans with more than 7,000 rare diseases. Literally, hundreds of thousands of rare disease patients have found their lives bettered by biologics and we are collectively grateful to the innovators and companies… 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
FDA Commissioner Margaret Hamburg, MD, today announced a new public-private partnership to develop regulatory science that will speed patient access to new medical device technologies. This is the first public-private partnership specifically focusing on this need. (more…)
