Data & Quality
Real-World Evidence (RWE)
Real-World Evidence (RWE) is clinical evidence about the use, safety, and effectiveness of a medical product derived from analysis of Real-World Data (RWD); EHRs, claims, registries, wearables; collected outside the controlled environment of a randomized clinical trial.
Under the 21st Century Cures Act, FDA established a formal RWE program. Acceptable uses include label expansions, post-marketing safety surveillance, external control arms for single-arm trials, and supplementing pivotal evidence in rare diseases.
Methodological challenges (confounding, missing data, measurement variability) must be addressed through pre-specified protocols, target trial emulation frameworks, and sensitivity analyses; FDA's 2023 RWE guidance series sets the evidentiary bar.
References
Related terms
Real-World Data (RWD) and Real-World Evidence (RWE)
Real-world data (RWD) is healthcare data collected outside the context of a controlled clinical trial; real-world evidence (RWE) is the clinical evidence about an intervention derived from analyzing RWD.
External Control Arm
An external control arm is a comparator group constructed from data outside the current clinical trial; historical trial data, registry data, EHR data, or natural-history studies; used to contextualize results from a single-arm trial when randomization is infeasible.
Accelerated Approval
Accelerated Approval is an FDA pathway that allows earlier approval of drugs treating serious conditions and filling unmet medical need on the basis of a surrogate endpoint that is reasonably likely to predict clinical benefit, conditional on confirmatory post-marketing trials.
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