Top 5 Challenges In Finding Rare Disease Patients

ISR asked respondents with involvement in patient recruitment and retention activities for Phase 2/3 rare disease studies about their pain points regarding patient recruitment. By far, the most challenging issue reported by respondents in finding patients for their recent rare disease studies was multiple sites with access to only a few patients (42%). The second most challenging issue was multiple entities pursuing same small pool of patients (19%), followed by rigid inclusion/exclusion criteria leading to high screen-out rate (15%).