RWE: Non-Interventional Studies Must Be Able To Distinguish A True Treatment Effect, US FDA Says

21 Mar 2024

Agency describes a host of issues sponsors should consider and address before pursuing observational or case-control studies to support regulatory decision-making on drug efficacy or safety; new draft guidance was issued under the FDA’s Real-World Evidence program.

Binoculars — Observational and other types of non-interventional studies are the focus of FDA's latest real-world evidence guidance. • Source: Shutterstock

Sponsors should consider how likely it is that a non-interventional study will be able to distinguish a true treatment effect from other influences before they rely on this approach to demonstrate efficacy and safety for regulatory decision-making, the US Food and Drug Administration said.

In a new draft guidance, “Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products,” the agency discusses...