Real-World Evidence in Pharmacoepidemiology: Methodological Rigor and Practical Applications

Dear Colleagues,

This Special Issue focuses on advancing real-world comparative evaluation of medications to generate robust evidence that supports clinical and regulatory decision-making. The value of real-world data (RWD) is increasingly recognized in both clinical research and regulatory contexts. In 2024, the US Food and Drug Administration released updated guidance on the use of RWD, including administrative healthcare claims and electronic health records, to support regulatory decision-making for drugs and biological products. Real-world evidence (RWE), defined as clinical evidence regarding the use and potential benefits or risks of a medical product derived from RWD, has already contributed to approximately one-third of recent FDA drug approvals for safety or effectiveness across varied therapeutic areas.

As healthcare systems increasingly rely on RWE, there is a growing need for rigorous approaches to evaluate medication safety, effectiveness, and utilization in routine clinical practice. The scope of this Special Issue includes pharmacoepidemiologic studies of comparative effectiveness and safety, as well as research on medication selection and utilization. Contributions may incorporate a range of analytical approaches, including causal inference methods and machine learning, to address challenges inherent in RWD and enhance the robustness of findings.

The purpose of this Special Issue is to highlight the value of real-world pharmacoepidemiologic research in complementing clinical trials and informing evidence-based care. This issue will supplement the existing literature by emphasizing applied, decision-relevant research using RWD, bridging methodological rigor with practical application, and demonstrating how well-designed observational studies can generate credible and actionable evidence. Ultimately, this Special Issue aims to support more informed clinical decision-making and contributes to improved patient care in real-world settings.

Prof. Dr. Rajender R. Aparasu
Dr. Jieni Li
Guest Editors

Manuscript Submission Information

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