Earning Their Trust: Challenges and Best Practices in Rare Disease Patient Recruitment

Key Takeaways

• Rare disease trials face structural enrollment headwinds from limited prevalence, geographic dispersion, and delayed diagnosis that narrows the reachable, eligible population.
• Stringent inclusion/exclusion criteria can further contract already small pools, making feasibility planning and protocol design pivotal to accrual success.
• Proactive trial awareness efforts help connect diagnosed patients to opportunities, reducing missed enrollment windows and improving the probability of timely screening.
• Patient partnership in trial design and recruitment messaging strengthens credibility and trust, increasing engagement and willingness to participate.