Orphan Diseases: Expansive Opportunity and Unique Challenges

Orphan drug development is attractive to drug developers for many compelling reasons: tax advantages, extended market exclusivity, accelerated approval timelines, historical­ly favorable access at premium prices, potential for lower cost clinical trials, and recent commercial success stories such as Soliris and Kalydeco. However, orphan diseases do not necessarily offer an easy win. Manufacturers must be prepared for the unique chal­lenges associated with orphan disease drug discovery, clinical trials, and market devel­opment, as well as the eventual likelihood that U.S. payers will actively manage pricing and market access for orphan products. Ultimately, considerable opportunity remains for drug developers in orphan diseases, but success will require companies to develop an orphan disease mindset for strategic decision-making and invest in capabilities com­mensurate with the anticipated nuances and challenges.

In this white paper, we lay out the clear areas of opportunity and the unique challenges associated with orphan disease drug development to help companies be best prepared to succeed in this exciting, but nuanced, space.