Case Study

A Mid-Sized Sponsor Increased Site Count By 50% In A Rare Disease Clinical Trial

Source: Medidata AI
GettyImages-1302453801 trial design ps


As they embarked on an upcoming rare disease clinical trial, a mid-sized biopharmaceutical company was struggling with patient recruitment and site selection. The company needed to identify sites with the greatest access to patients and highest enrollment performance but did not have deep enough data to make confident decisions.

While there are approximately 7,000 rare diseases, 95% of these conditions do not have an FDA-approved treatment.1 This figure is impacted by many factors, including small patient populations and changing trial conditions. Between 2016 and 2020, over a quarter of rare disease trials were terminated due to low patient accrual rates.2 In such a challenging indication, it was critical for this mid-sized sponsor to choose high-performing sites based on reliable data to set their trial up for success.

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