Challenges in Rare Diseases

There are approximately 7,000 rare diseases. Such rare diseases generally have a genetic basis, frequently affecting patients in childhood, and are often progressive and life threatening in nature. These characteristics can have highly destructive psychological impact on children suffering from these diseases. The major challenge for R&D is to look after the impact of unique characteristics of the rare disease patients on clinical development process and the interface between clinical development and the regulatory process. A fundamental challenge in drug development for rare diseases is that there is relatively little known about the pathophysiology of disease. In addition to these challenges, for most of the rare diseases there are no available therapies.

 

  • Patient attrition
  • Interface between clinical development and the regulatory process
  • Less knowledge on pathophysiology of disease

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