White Paper:

Rare Diseases: What They Are, How They’re Unique, & Why They Need to be Approached Very Differently

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
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