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A Patient-centric approach to rare disease drug development
Advancing oprhan drug research with the patient in mind

Q&A with Laurie Witherwax, Operations Head for Rare Disease in Project Leadership, Quintiles
According to Global Genes, more than 350 million people worldwide suffer from rare diseases. Unfortunately, according to the FDA's Every Life Foundation, only around 5% of rare diseases have a FDA-approved treatment. Quintiles recognizes the growing need for research and new treatments for these uncommon conditions. Here are some of the most frequently asked questions we hear from biopharmaceutical customers:

  • What are the challenges associated with conducting clinical trials of rare disease therapies?
  • What about the difficulties associated with patients being fewer in number and further apart?
  • Why is post-marketing surveillance of patients more common for rare disease therapies?
  • Is enough research being done into rare diseases and what is Quintiles' approach?

In the May 2016 issue of Nature InsideView, Laurie Witherwax addresses these questions - and more - as well as outlines the patient-centric approach that Quintiles deploys to help pharmaceutical and biotechnology companies get new therapies to patients.

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Quintiles, the world?s largest provider of biopharmaceutical development and commercial
outsourcing services, has worked on more than 230 rare disease studies since 2010.



Learn more about our perspectives on Rare Disease
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