WS23.1 – Small Populations, Big Challenges: Clinical Trial Supply for Rare Diseases

One in 17 people lives with a rare disease — comparable to the prevalence of cancer — yet clinical trials for these conditions often rely on only a very small number of geographically dispersed patients, making supply planning inherently complex. In this intermediate-level workshop, participants will explore the unique challenges of orphan indications and rare disease trials. Through interactive discussion, attendees will examine critical supply requirements, including small patient populations, limited and highly targeted supply volumes, global distribution considerations, and the resulting impact on study timelines.