Clinical Research: Phase 1 - Phase 4

Clinical Trials in Rare Diseases: Every Patient Counts

In many rare and ultra-rare disease studies, sponsors do not have the luxury of choosing the countries or sites where the study will be conducted. Instead, they must use the sites where those patients are already being treated, regardless of geography. Additional protocol criteria – such as treatment-naïve patients or general prevalence of a disease by region – may limit site selection even further, requiring the sponsor to open sites in countries that normally would not be considered for clinical trials. In this white paper, we explore the many aspects of clinical trial design and management that sponsors must consider to maximize the likelihood of study success.