Use of External Data in Oncology and Rare Disease: A Scoping Review of EMA submissions (2015–2025)

This research is a scoping review examining the use of external data in European regulatory submissions for oncology and rare disease therapies between 2015 and 2025. Guided by the EMA’s draft concept paper on external data, the review aims to identify patterns in submissions, assess the application of methodological elements recommended by the EMA, and translate findings into best practices to support regulatory evidence generation. Using EMA databases and European Public Assessment Reports (EPARs), submissions referencing external or historical controls were systematically identified, and key information, including trial design, data sources, and methodological approaches, was extracted and analyzed. This work provides a comprehensive overview of how external data are utilized in regulatory decision-making, with a focus on enhancing guidance for future drug development.