Challenges in Rare Diseases

Making a pharmaceutical care development program for the treatment of a uncommon disease can, however, prove to be a monumental task. Poor understanding of the characteristic history of the proposed indication due to few observational studies considering disease progression, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, regulatory uncertainties, and lack of prior clinical studies to establish a format for study execution, can all prove challenging. While individually rare, orphan diseases are really collectively common, with an estimated 350 million sufferers around the world.

  • Patient attrition
  • Interface between clinical development and the regulatory process
  • Less knowledge on pathophysiology of disease
  • Lack of awareness
  • Diagnosis
  • Developing and using Orphan medicinal products

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