Real-world evidence (RWE) is increasingly vital in regulatory submissions, supported by the FDA’s evolving guidance since the 21st Century Cures Act. Best practices include early and ongoing FDA engagement, clear justification of data sources, prespecified protocols and SAPs, and patient-level data in compliant formats. Sponsors must ensure internal validity by mitigating biases, validating variables, and aligning endpoints with regulatory expectations. Case studies show success when RWE is fit-for-purpose, well-documented, and addresses unmet needs, as in Lumakras, Vijoice, and Prograf, while failures like Omblastys stress the risks of poor endpoint choice and inadequate alignment.

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