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.