Tag: ECA

Regulatory Insights from FDA Review of the External Control Arm in a Pediatric Rare Disease Trial

The approval of eflornithine (brand name Iwilfin) for pediatric high-risk neuroblastoma offers an interesting example of how the FDA evaluates single-arm trials supported by external control arms (ECAs). External controls are increasingly common in oncology and rare disease drug development, but they remain a source of careful regulatory scrutiny. In this case, the FDA’s assessment… Continue reading Regulatory Insights from FDA Review of the External Control Arm in a Pediatric Rare Disease Trial

External Control Arms in Clinical Trials: Case Studies of Health Authority Submissions

In recent years, the landscape of clinical trials has been evolving, with innovative methodologies gaining traction. One of such advancement is the use of External Control Arms (ECAs) in clinical trials. ECAs utilize existing patient data external to the planned clinical trial as a comparison group.   An ECA can be constructed using data from… Continue reading External Control Arms in Clinical Trials: Case Studies of Health Authority Submissions