Patient and Stakeholder Engagement
Register Now: Patient Advocacy Groups and Their Role in Orphan Drug Development
There are more than 7,000 known rare diseases, and these conditions afflict an estimated 30 million Americans (almost one in 10). Worldwide, some 350 million are living with a rare disease. But for 95 percent of these conditions, there are no FDA-approved treatments — not one. Conventional wisdom holds that innovation follows need: that if...