Combining RCT and RWD – applications in rare diseases and practical recommendations
A Talk by Prof. Dr. Tim Friede (Head of the Department of Medical Statistics, University Medical Center Göttingen)
About this Talk
Randomized controlled trials (RCTs) are the gold standard for evaluating interventions. However, they are often considered to be difficult to conduct and may therefore suffer from small sample sizes. Here we demonstrate how RCTs can benefit from the inclusion of real world data (RWD). More specifically, hierarchical models for evidence synthesis can be utilized to combine RWD and RCT data to increase the precision of the RCT effect estimate. In the comprehensive cohort study design, the RCT and the cohort study are carried out in parallel. It allows to assess the external avlidity of an RCT and can also be very efficient when the RCT and registry are evaluated jointly. In conclusion, there are various promising ways of linking data from RCTs and RWD. Therefore, a joint consideration of RCT and RWD data appears desirable, in particular in rare diseases and paediatrics.
Moderators
Strategic Advisor, Verisian
Unleashing the Power of Statistics and Data Analytics to Drive the Future of Clinical Development | Experienced Transformative Leader in Biostatistics & Data Science | Entrepreneur | Advisor | Speaker