Rare Diseases: Incentives That Work and Backfire

Logosofia no Dia a Dia · Apr 25, 2026 · 27m

The U.S. Orphan Drug Act transformed rare disease treatment from 34 drugs to nearly 800 approved therapies. But this success story has a dark side: the "orphan paradox," where drugs for tiny patient populations become billion-dollar blockbusters, and new policies may actually discourage companies from developing treatments for additional rare diseases. This episode examines which incentives—tax credits, exclusivity, priority review vouchers, grants—actually moved the needle, and how the Inflation Reduction Act's orphan drug exemption is creating unintended consequences that could leave rare…