All Times EDT
Keywords: rare disease, missing data, small sample size
Nowadays, with the development of registries, EHR, post-marketing studies, more and more reliable, and relevant external data are available for medical research and development. These Real-World Data are utilized to serve as external control, augment the sample size in single arm trial etc. through various statistical methods and approaches. Common issues such as "comparability of the external data" has been extensively discussed. However, missing data issue and challenges are less addressed, particularly, when the size of external data is moderate or small. For example, in rare disease areas and pediatric patient population. When the data size is moderate or small, missing data could be more noticeable. It's also possible the external control had clinically relevant covariate missing. Therefore, in this talk, we will discuss missing data issues when utilization moderate or small size external data and how we could make a totality of the evidence through different sensitivity analyses.