Rare Diseases and Orphan Products
- First video: Why is investing in cures for rare and orphan diseases important? What can we learn from these diseases to apply to more common health problems?
- Second video: 7000 rare diseases affect 30 million Americans; 350 new therapies so far; 1/3 of new approvals lately are orphan products. What are some examples? What is the history of rare disease efforts at FDA? Note that the speaker, former FDA head of Orphan Products Dr. Timothy Cote has left the agency to set up a consulting practice according to his LinkedIn page and other sources.