ICON Clinical Trial Experts Discuss the Intricacies of Rare Disease Studies

Two clinical trial experts at ICON shared their thoughts on clinical trial design, including important considerations and strategies for rare disease studies. Successfully incorporating the patient perspective, utilizing real world data (RWD), and picking appropriate endpoints are major sticking points in rare disease research.

“Consider the selection of endpoints – are they satisfactory to regulators and patients? Consideration of unmet medical needs and patient motivation to participate in a clinical trial. Ensure that disease variability amongst patients doesn’t result in selection bias.” Read more here.

(Source: Jenni Spinner, Outsourcing-Pharma, 9/20/21)