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In the EU a rare disease is defined as one with a prevalence of 1 in 2,000 people, and in the US, as affecting fewer than 200,000 people (equivalent to 1 in 1,600 people as of 2013 population figures).

In this report, we will focus on rare diseases defined as per above. By these definitions, there are between 6,000 and 8,000 recognised rare diseases, most of them arising from genetic origin and being chronic or life-threatening.

Highlights in this white paper include:

With the ‘blockbuster bubble’ seemingly burst and patent protection giving way to huge rises in generic competition, the pharma industry appears to be focusing on high value drugs for rare diseases of low prevalence for which there is a potential for rich return on investment.
Using Citeline data, this paper analyzes the landscape for rare diseases and the density of development by phase, developing company and disease.


  • The number and prevalence of rare diseases in the US and EU
  • The number of drugs in active development by phase and the
    companies developing them
  • Areas of therapeutic focus for rare disease development
  • Drugs and vaccines in development for infectious diseases 
  • The oncology landscape for rare diseases, and which drugs
    are exhibiting the most promise

Will the Blockbuster Model be Replaced

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