In response to the 21st Century Cures Act, which aims to accelerate medical product development and innovation, the United States Food and Drug Administration (FDA) has now released final guidance about using real-world evidence (RWE) to support regulatory decision-making that includes considerations for both pre- and postapproval studies. This final release represents the FDA’s current thinking and recommendations on this topic. Although not binding or required, the guidelines provide a reference for researchers and sponsors designing studies using RWE that are intended to be submitted to the FDA.
In this guidance, "real-world data" (RWD) is defined by the FDA as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” and RWE as “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.”
The FDA clarifies that noninterventional studies are not considered clinical investigations under 21 Code of Federal Regulations (CFR) Part 312 and do not require an Investigational New Drug (IND) application. They also focus on considerations for noninterventional studies that use RWD and are intended for regulatory submission.
The guidance emphasizes the importance of transparency and documentation (including feasibility assessments, all versions of the study protocol, statistical analysis plan, etc.), the importance of maintaining data integrity and selecting data that are fit for use, and the importance of providing FDA access to individual-level data. It also highlights the importance of early engagement with the FDA at the study design stage. Some additional key recommendations are as follows:
- Sponsors should post study protocols publicly to ensure transparent data collection and analysis.
- Because study monitoring, data access, and safety reporting are essential responsibilities, monitoring of noninterventional studies should focus on data accuracy, adherence to the study plan, and prompt evaluation and remediation of deviations.
RTI Health Solutions has decades of experience in the design and implementation of noninterventional studies using RWD and RWE in regulatory submissions. Contact us to talk about how we can help with your RWD and RWE research needs.
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