BACKGROUND: The United States (US) and Europe vary in their adoption of real-world evidence (RWE) to drive healthcare policy. While efforts in the US were encouraged by the passage of the 21^st Century Cures Act in 2016, the European Medicines Agency (EMA) has been conservative in its approach, fearing biases in data that might impact the quality and credibility of evidence generation.

OBJECTIVES: The objectives of this paper are to evaluate the current role of RWE in US and European stakeholder decision-making and to provide considerations for RWE harmonization.

METHODS: We evaluated research papers as well as review articles from 2015 onwards to ensure relevance to policy decision making. Articles that referred to the use of RWE for regulatory, payer, market access, and policy decisions were selected.

RESULTS: Malone and colleagues indicate that while US payers are interested in RWE, they perceive several practical challenges to utilizing RWE while making policy decisions. These include the lack of relevant endpoints, the timeliness of the research conducted, and the transparency of the methodology used to conduct the research. Facey et al. explored the challenges associated with data harmonization across Europe often caused by legal, political, economic, and digital barriers. While France, Sweden, and Norway maintain robust electronic medical and governmental records with unique patient identifiers, they lack data on prescription drug use. Conversely, Italy and Portugal track utilization of prescriptions, but using bespoke registries and outcomes-based managed entry agreements. In the U.K., the National Institute of Clinical Excellence is working to incorporate comparative individual patient data earlier into the technology appraisal process to better estimate treatment effectiveness.

CONCLUSIONS: We recommend the development of a North Atlantic Healthcare Data Alliance to serve as the starting point towards harmonizing RWE protocols and data generation to provide rich and timely insights for policy decisions.