Michael F. Murphy, M.D., Ph.D


Rare Diseases: Meeting the Unique Challenges of Orphan Drug Development

January 21, 2016

Poor understanding of the natural history of the proposed indication due to few observational studies studying disease progression, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, regulatory uncertainties, and lack of prior clinical studies to establish a template for study execution, can all prove challenging in creating a pharmaceutical development program for the treatment of a rare disease.