Cholestatic Pruritus in Patients with Primary Sclerosing Cholangitis (PSC)
Abstract: Recruiting and retaining patients in rare disease clinical trials remains a persistent challenge, particularly when eligibility criteria are restrictive and patient populations are geographically dispersed. In this Phase 2 study for cholestatic pruritus in patients with Primary Sclerosing Cholangitis (PSC), enrollment lagged nearly a year behind schedule due to limited eligible patients, high dropout rates at traditional sites, and reluctance among patients to travel. To address these barriers, a Direct-to-Patient (DTP) clinical trial site was added to complement brick-and-mortar locations.
This case study demonstrates how Science 37’s FDA-inspected Direct-to-Patient Site, supported by Catalent’s centralized storage and home-delivery logistics, helped revitalize enrollment and retention in a rare disease setting. Through in-home visits, targeted patient outreach, and reliable direct-to-patient investigational product distribution, the DTP model expanded access beyond traditional sites and reduced patient burden. As a result, the DTP site became the top enrolling site globally, illustrating how decentralized approaches can accelerate recruitment and rescue timelines in complex rare disease trials.