Evaluating the Medical and Financial Potential of New Rare Disease Indications

Student: Tristan Cates, 2018-2019

Sponsor: Horizon Pharma, Inc.

Orphan diseases individually afflict less than 200,000 people in the US, but collectively impact 25 million. The scarcity of costumers afflicted by orphan diseases made it nearly impossible for pharmaceutical companies develop new treatments. Year 1983 brought around the Orphan Drug Act (ODA), providing financial incentives for companies that can develop effective treatment for those effected by rare diseases. Since then the orphan disease drug market has expanded producing new opportunities to utilize biomedical technology to treat those in need. Horizon Pharma was founded in 2008 to capitalize on this opportunity and create treatment for underrepresented patients in the orphan disease space. My project with Horizon Pharma involves analyzing candidate orphan diseases, and if they are theoretically treatable with current technological assets, have a significant amount of unmet need, and are financially realistic to pursue in the future.