Earning Their Trust: Challenges And Best Practices In Rare Disease Patient Recruitment

Recruiting for rare disease clinical trials requires a fundamental shift to recruitment models built on community partnership and co-design. While interest in clinical research among rare disease patients remains high, a significant gap exists between their willingness to participate and awareness of available opportunities. This disconnect is often exacerbated by the long, arduous journey to a correct diagnosis. To bridge this gap, study teams must engage with patient advocacy groups as strategic partners who provide essential insights into the daily realities of living with a specific condition.

True patient-centricity involves incorporating patient feedback during the protocol development phase to identify and mitigate potential barriers to participation. While decentralized trial elements are often viewed as a solution for accessibility, they are not a universal fix; for many in the rare disease community, the loss of direct connection with specialist investigators can be a deterrent. Flexibility in trial design—balancing remote monitoring with meaningful in-person care—is critical for long-term retention. By prioritizing transparency and reducing the physical and emotional burden on participants, sponsors can move beyond transactional recruitment to earn the long-term trust of these specialized communities. Download the full white paper to explore best practices for patient-led study design.