The feasibility of using real world data as external control arms in oncology trials
Lishi Lin, Merel J J Lucassen, Vincent van der Noort, Toine C. G. Egberts, J. H. Beijnen, Alwin D. R. Huitema
Abstract
• Single-arm clinical trials are performed more often within the field of oncology. • Real world data (RWD) can be used as external control arm (ECA) in these trials. • Suitable RWD sources and methodological analyses should be used. • The use of ECAs is suitable in cancer types driven by rare molecular alterations. Before real world data (RWD)-derived external control arms (ECAs) can be applied in clinical trials within the field of oncology, it must be determined whether these ECAs can function as appropriate and reliable control arms. This review provides an overview of studies in which RWD-derived ECAs were constructed and compared to a randomized clinical trial (RCT) arm. RWD-derived ECAs had similar survival outcomes as the RCT arm of comparison in six of the eight included studies. Therefore, when performing RCTs is not feasible, RWD-derived ECAs can act as control arms in single-arm clinical trials, provided that suitable RWD sources are used and suitable methodological decisions are made.