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Rare Disease Facts

  • There are more than 7000+ Rare Diseases known/reported world-wide
    • The RARE GENES project maintains a comprehensive directory of all RDs
  • Over 350 Million people world-wide are affected ~30 Million in USA, ~30 Million in EU, ~70 Million in India
  • 1 in 20 Indians is affected by one of these RDs
  • About 80% of RDs are genetic in origin many of them being monogenic
  • 50% of RDs are onset at birth and the rest are late onset
  • RDs include inherited cancers, autoimmune disorders, congenital malformations, and infectious diseases amongst others include Hemangiomas, Hirschsprung disease, Gaucher disease, cystic fibrosis, muscular dystrophies and Lysosomal Storage Disorders.
  • USA is became the first country to enact Orphan Drug Act in 1983. The law is designed to facilitate the development and commercialization of drugs to treat rare diseases, termed orphan drugs. The salient features of the act are as follows:
    • Incentives to Pharma companies investing in Orphan Drugs R&D
    • Faster/Priority clearance of Orphan Drugs Application through FDA
    • Market exclusivity for 7 years for Pharma companies to recover costs
  • Global Orphan Drugs market exceeded 50 Bil $ in 2011
  • Only ~500 approved orphan drugs exist in the market and over one hundred in clinical trials.
  • Majority of the diseases have no treatment and when they exist, are mostly unaffordable
  • Early diagnosis is a critical challenge in RD management (Avg time 5-7 yrs)
  • Affordability: Genzyme and more recently Shire have charitable access program for LSD patients in India

 

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