White Paper:

Success in Rare Diseases, Part II: Optimizing Clinical Trial Design for the Rare Disease Ecosystem

This paper outlines why it’s especially challenging to design clinical trials for rare disease therapies.  It describes key considerations regarding:

  1. Defining the patient population for a clinical program
  2. Selecting endpoints
  3. Selecting comparator(s)
  4. Defining cohort sizes

This paper is part 2 in an ongoing series.  Part 1 can be downloaded here.

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