Orphan Does Not Always Mean Alone—A Case Study of Eosinophilic Esophagitis, an Orphan Disease

Johanna Rahnasto


Eosinophilic esophagitis (EoE) is a rare immunoinflammatory disease that has recently attracted significant drug development activity. Two EoE drugs have been approved, and clinical trials with several products are ongoing. This Article uses EoE as an example to discuss orphan drug regulation in the EU and United States and its interplay with medicine, profitability, competition, and patient needs. The Article concludes that the development of several competing therapies for an orphan disease like EoE increases uncertainty for patients and pharmaceutical companies compared to non-orphan drugs, even while orphan incentives are necessary to ensure a financially lucrative environment.