Tags: 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 that are 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 therapies that target severe illnesses with unmet needs, where single-arm trials are often conducted due to the lack of effective therapies and low feasibility of a parallel-arm controlled trial.
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 by the FDA gained interest in the marketplace and was bought a few months later.
RWE has the potential to both accelerate approval timelines and reduce the costs of drug development, as long as sponsor companies carefully uphold established evidence and engage in early dialogue with the FDA.
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Specializing in rare disease, Boston Biotech Clinical Research works with biotech, pharmaceutical, device companies and investors to streamline the clinical trial process. Our experienced team helps each client reach their specific goals by customizing a clinical and regulatory road map of simplified programs and streamlined protocols to meet our clients’ requirements.