Combining RCT and RWD – applications in rare diseases and practical recommendations

In this special keynote episode, I’m excited to share the recording of Professor Tim Friede’s thought-provoking presentation from The Effective Statistician Conference 2024. Tim, a leading expert in biostatistics and clinical trial design, dives deep into the combination of randomized controlled trials (RCTs) and real-world data (RWD)—especially in the context of rare diseases. Drawing from his work at the University Medical Center Göttingen and numerous European research initiatives, Tim presents a compelling case for integrating RWD to support small or underpowered RCTs using advanced statistical models. He shares real-world examples (including CJD and Alport syndrome), simulation insights, and practical recommendations that can change how we approach evidence generation in low-prevalence populations. What You’ll Learn: