World Orphan Drug Congress USA 2016
community download

[Whitepaper] Cure PSP: No Patients. No clinical trials. No treatment or cure.

The paradox of recruitment and retention


Clinical studies into rare diseases have not risen significantly since the inception of the Orphan Drug Act in 1983 despite the many incentives offered to Pharma. Recently Pharma has expanded into the rare disease space motivated by the possibility that discoveries into rare disease can translate into discoveries in more prevalent diseases.


The population in the US as of January 2016 was approximately 323 million people. The estimated number of people world-wide with rare diseases is 350 million. If everyone in the US had a rare disease that would end in suffering and death, what would be different? The answer is everything.


While it’s difficult to believe that there are upwards of 7,000 rare diseases (RD) currently identified, it is more difficult to imagine that with advances in scientific discovery and technology 95% of these diseases do not have any FDA approved drug treatments.


Download this whitepaper now for:

  • PSP
  • Getting to know patients with rare diseases
  • The need for rare disease research
  • Retention is critical to success
  • Partnering with Advocacy Organizations
  • Overcoming the challenges of rare disease clinical trials

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