Leveraging Real-world Evidence for Antihypertensive Fixed-dose Combinations: A Multidimensional Narrative Review From European Industry Perspective.

Purpose: Real-world data (RWD) and real-world evidence (RWE) offer significant potential for clinical development and clinical evidence generation. This review addresses the complexity surrounding the use of RWD and RWE in drug repurposing, such as antihypertensive fixed-dose combinations of known compounds from the 5 major pharmacological classes.

Methods: We performed a narrative review examining RWD and RWE use in the development and registration of antihypertensive fixed-dose combinations, covering their role as examples of value-added medicines, regulatory policies, scientific perceptions, application in hypertension research, regulatory use cases, and economic factors.

Findings: Well-designed RWE studies can yield clinical performance data comparable with findings of randomized controlled trials while addressing certain limitations. However, current European Union (EU) legislation regarding the usability of RWD and RWE is conservative and needs to be updated. Divergent opinions among EU member states pose uncertainty and risk for industry in marketing authorization applications involving RWD and RWE.

Implications: Real-world data and RWE offer a transformative opportunity through data sourced beyond traditional clinical trials. This approach can expedite regulatory decisions, reduce development timelines and costs, and accelerate the delivery of valuable therapies to patients. Discussions at the centralized EU regulatory level are needed to recognize and accept RWE as valid for demonstrating clinical efficacy and safety.