Orphan & Rare Diseases

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

10:45 AM–12:00 PM Jun 20, 2017 (America - Los Angeles)

Room 3, Upper Level

Orphan-drug development has escalated at a rapid in recent years, and 449 unique drugs have now received approval for one or more orphan indications. Drugs for rare diseases represented more than 40% of new drug approvals in 2014 to 2016. What does this mean for the state of the orphan drug industry and where we are headed in seeking to reduce unmet needs? How can the 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: 25183