Orphan and Rare Diseases

Closing the Innovation Gap in Rare Diseases: Where are we Headed?

4:30 PM–5:30 PM Jun 4, 2019 (US - Eastern)

118A, Level 100


Orphan Drug development has escalated at a rapid pace in recent years with over 500 products on the market to treat rare conditions. Despite the progress in rare disease innovation only 5% of rare diseases have any form of treatment. What does this mean for the state of the orphan drug industry and where are we headed in seeking to reduce unmet needs? How can productivity of clinical development for orphan drugs be improved? What reaction to rising expenditure on treatments for rare diseases can be expected from payers? How do all the components of the health system need to evolve to ensure the innovation gap is closed? Join an industry thought leader armed with evidence to frame the current state of rare disease treatment, the outlook for closing the innovation gap, and the implications for patients and other stakeholders over the next five years.

Session ID: 545631