Signal Boost: Real-World Evidence Fits US FDA’s Safety Ambitions More Easily Than Efficacy

RWE is already transforming the US FDA’s active surveillance systems and could catalyze change in pregnancy postmarketing studies, but is advancing more slowly for use as efficacy evidence.

Using real-world evidence requires identification of signals in a universe of noisy data. (Shutterstock)
Key Takeaways
  • Real-world evidence still is rarely central to FDA approval decisions, but the agency noted real-world data’s contribution to a new indication for Genentech’s Actemra (tocilizumab) and a new pediatric dosing regimen for UCB’s Vimpat.
  • The FDA Sentinel RWE Data Enterprise’s linked electronic health records and claims data are helping identify outcomes and variables in structured and unstructured data for postmarketing safety surveillance.
  • RWE can improve postmarketing pregnancy safety studies, potentially helping with traditionally slow enrollment, as well as signal detection.

 

Real-world evidence is firmly entrenched in the regulatory and drug development toolbox, but grand visions of approvals with RWE in a starring role are dimming in favor of emerging supporting...

In drug development, RWE and real-world data should not be seen as an alternative to traditional randomized controlled trials, but as tools to improve studies, John Concato, outgoing associate director...

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