11 September 2019 - A new study in JAMA Network Open finds that drugs and medical devices approved by the US FDA based solely on non-randomised controlled trials exhibited larger effect sizes compared to products that required further study in randomised trials. 

But the authors called for greater FDA transparency to instil confidence that decisions about drug and device approvals based on observational data are appropriate.

The goal of the study, conducted by researchers including Stanford Professor John Ioannidis, Cochrane Founder Lian Chalmers and City of Hope Hematologist Benjamin Djulbegovic, was to identify how large treatment effects must be for regulators to consider data from non-RCTs sufficient for approval, as RCTs are considered the “gold standard” for generating clinical evidence for medical interventions.

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