Keywords: Rare diseases, complex endpoints
There will be short presentations from three speakers followed by a panel discussion. The speakers include Dr. Dragos Roman, a division director for many of the inherited disorders that fall under an orphan designation and the challenges he faces. Dr. George Kordxakhia will present some considereations for multiple endpoints since many of the rare diseases involve a complex set of symptoms. Dr. LJ Wei of Harvard will present a novel approach in the context of these trials allowing one to consider multiple symptoms. They will be on the panel and Dr. Fan-Fan Yu from Statistics collaborative will join them. Dr. Yu was involved in the first gene therapy ever licensed and for which a novel endpoint was developed. Dr. Michael Hale of Takeda will moderate the session.