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Data challenges for externally controlled trials: a viewpoint
Russanthy Ruthiran Velummailum;
Chelsea McKibbon;
Darren R Brenner;
Elizabeth Ann Stringer;
Leeland Ekstrom;
Louis Dron
ABSTRACT
When the preferred evidence of a large, randomized controlled trial is difficult to run in scenarios such as rare conditions or clinical subgroups with high unmet need, evidence from external sources including real world data is increasingly being considered by decision-makers. Identifying suitable real-world data that can be used to contextualize a single-arm trial, as an external control arm, presents with several challenges. In this viewpoint, we provide an overview of the common issues raised by regulatory and health reimbursement agencies when evaluating comparative efficacy and present some solutions.