Rare disease development is complex, characterized by distinctive challenges due to small patient populations spread across many geographies, limited disease data, and narrow site pools. These hurdles compound the universal difficulties inherent to recruiting and retaining eligible participants for clinical trials. For sponsors, successful studies involve striking a balance among cost, quality, and innovating approaches that improve trial efficiency and patient engagement.
In this checklist, we explore 9 best practices for optimizing the efficiency and quality of rare disease clinical trials, with a focus on strategies that embrace innovation and promote collaboration among key stakeholders.