14 February 2020 - The New England Journal of Medicine published an article earlier this week raising questions about the use of observational real world evidence, and how part of this drive toward using non-randomised studies to assess the effects of treatments is due to the expensive and complex way that randomised clinical trials are being conducted.

The timing of the article coincides with the US FDA Commissioner Stephen Hahn recently pointing to the importance of RWE, saying, “I believe there is great promise in the effective use and integration of patient-level data or real-world evidence such as electronic health records, clinical trials, medical studies, and patient registries.”

And FDA now has guidance on both the submission of real world evidence (RWE) on the drugs and biologics side, as well as the use of RWE on the device side, and released an RWE framework in December 2018, part of the work of the 21st Century Cures Act from 2016. And although the agency has only used RWE in limited amounts so far (at least eight new indications have been approved for previously approved treatments), the promise of RWE is clearly at the forefront of FDA’s and drug developers' agendas.

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