Keywords: regulatory submission, time to event, integrated summary of efficacy (ISE), integrated summary of safety (ISS)
It is well-known that each NDA/BLA submission has its own challenges and interesting questions. More specifically, integrated summary and analysis have some unique statistical challenges. Since CDISC and eCTD are implemented in the last few years, the regulatory submissions have been more streamlined. However, the statistical challenges and questions are still existing for NDA/BLA submissions, especially for integrated summary/analysis components. This roundtable is designed to discuss the statistical challenges and interesting questions associated with the integrated summary/analysis in NDA/BLA submissions. The goal would be for industry statisticians to compare notes and share their experiences in these integrated summary/analysis, as well as, get the regulatory perspective on such integrated summary and analysis from participating FDA statisticians.
Some key questions/talking points for discussions: 1. How do we handle the patients who enrolled multiple studies (especially pivotal studies) in one ISS/ISE? 2. How do we handle the situation when time to event definitions are different among the pivotal studies but need to combine them in one ISE? 3. How do we handle the different assessment schedules among the multiple pivotal studies so those safety and efficacy assessments can be analyzed and summarized in one ISS/ISE?