2025 Orphan Drugs Report:
Are Orphans That Different?
For more than a decade, orphan drugs’ growth has outpaced that of non-orphan prescription drugs. Niche products’ share of worldwide prescription drug sales by value has doubled over the last decade, from less than 10% in 2014 to almost a fifth...
Top Drugs and Companies: Darzalex Dominates
Top Orphans Mirror Mainstream Drugs
Blood Disorder and CNS Drugs in Cancer’s Shadow
Orphan Pipeline: CAR-Ts and Gene-Edits
Orphan Shares at J&J, Merck, Roche Rise; Fall Back at Vertex
Uncertain Policy Environment May Shift Forecasts
HOW WE HELP