Single-Arm Trials in Oncology Registrational Studies

This document is a technical report that discusses the use of single-arm trials as registrational studies in oncology. It outlines the characteristics of single-arm trials, including their design, regulatory considerations, and the context in which they may be appropriate. The report details the advantages of single-arm trials, such as shorter completion times and smaller sample sizes, while also addressing their limitations, including potential biases and challenges in endpoint interpretation. It presents data on the approval rates of oncology drugs based on single-arm trials by the FDA and EMA, highlighting the importance of defining the target population and unmet medical needs. Additionally, the report emphasizes the necessity of careful planning and regulatory strategy when considering single-arm trials for drug approval, including endpoint selection and methods to control bias. Overall, the report serves as a comprehensive guide for understanding the role of single-arm trials in the regulatory landscape of oncology.