Terminkalender FDA/CMS Summit.
06.12.2017, 1:00 PM
Rare Diseases: Trends, Developments, and PRVs
With more generics on the horizon that will increase competition and drive down costs in the big drug
market, the industry is fast rediscovering the commercial opportunity in orphan drug development, helped
along by expedited approvals and the potential for expanded exclusivity periods. This opportunity is not only
good for new revenue streams but more importantly, offers hope to those patient populations living with rare
diseases by shining a new light on orphan drug development. At the intercept of capitalism and altruism, what
will be the end result? This session will discuss the implication of recent trends and developments in orphan
drugs, priority review vouchers, and the renewed interest in this market. What are the implications for these
patient populations?
Moderator: InVentiv Health
Panelists:
Ron Cooper, President and CEO, Albireo Pharma
Michael Spector, President and CEO, Caelum BioSciences
Jonathan Goldsmith MD, Associate Director Rare Disease Program, Center for Drug Evaluation and Research
(CDER) Food and Drug Administration (FDA)