Real World Evidence

RWE for Control Arm, Post-approval Requirements and Disease Natural History

Real-world evidence (RWE) is increasingly important in clinical trial design and drug approval. RWE is clinical evidence related to patient health status and treatment safety and efficacy generated in clinical practice. The FDA developed a framework to use RWE, which may combine with orphan, fast track, breakthrough therapy designation, and accelerated approval to support the development of drugs and biologics.

RWE can be especially important for orphan disease therapy that targets severe illnesses with unmet needs. Where single-arm trials, on the other hand, are often conducted due to the lack of effective treatments and the low feasibility of a parallel-arm controlled trial.

RWE can accelerate approval timelines and reduce drug development costs as long as sponsor companies carefully uphold established evidence and engage in early dialogue with the FDA.

Case Study

The BBCR team adopted RWE from a retrospective off-label use of study treatment to benchmark and contextualize the design of a pivotal single-arm trial in ultra-orphan indications. The FDA agreed with the proposed study design at pre-IND meetings. As a result, the biotech that obtained clinical plan approval from the FDA gained market interest and was bought a few months later.

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