A - Abstract
Rigorously testing the growing medication pipeline for rare conditions continues to be an
industry challenge. The low prevalence rates for rare diseases run contrary to the phased drug
development processes that are the standard and poses significant obstacles for developing,
testing, and approval of promising treatments. The intent of this position paper is to bring together
into a practical framework three elements of clinical research (N-of-1, adaptive design techniques,
and multiple baseline) for testing small and variable samples of patients undergoing treatment. The
objective is to bring continuity and best practices to the evaluation of pharmaceutical products for
treating rare disease. Specifically, the paper discusses general principles for when and how they
should be applied and what limitations should be considered when developing methods for testing
treatments for rare diseases.