This comprehensive white paper:
- Outlines how key regulatory authorities define rare / orphan diseases
- Establishes a “rarity spectrum” from “Orphan” to “Hyper-Orphan,” sharing examples of diseases within each category on the spectrum
- Explains how rare diseases differ from the more common “specialty” diseases and rare cancers, and explains the commercial implications for biopharma companies
- Introduces the concept of a “neglected” rare disease and shows how they differ from other rare diseases.
- Uses actual product examples to show how the commercial challenges vary between “neglected” and “non-neglected” rare diseases
