The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space

Strategy, Management and Health Policy Large pharmaceutical companies have traditionally focused on the development of blockbuster drugs that target disease states with large patient populations. However, with large‐scale patent expirations and competition from generics and biosimilars, anemic pipelines, escalating clinical trial costs, and global health‐care reform, the blockbuster model has become less viable. Orphan drug initiatives and the...