Toward a better understanding about real-world evidence.

Background There has been an interest in real-world evidence (RWE) in recent years. RWE is usually generated from data derived from routine healthcare, such as electronic healthcare records and disease registries. While RWE has many advantages, it is often open to various biases, which may distort results. Appropriate understanding and interpretation are critical to the best use of RWE in healthcare decisions. Methods On the basis of a literature review and empirical research experience, we summarised the concept and methodological framework of RWE, and discussed in detail methodological issues specific to routinely collected healthcare data and observational studies using such data. Results RWE is derived from a spectrum of data generated from the real-world setting, using two broad study designs including observational studies and pragmatic clinical trials. Real-world data may usually be collected through routine practice or sometimes actively collected with a research purpose. Observational studies using routinely collected data (RCD) are the most common type of RWE, although they are prone to biases. When planning and implementing RWE studies, coherent working steps are warranted, including definition of a clear and answerable research question, development of a research team, selection of a fit-for-purpose data source, choice of state-of-the-art study design, establishing a database with transparent data processing, performing multiple statistical analysis to control bias, and reporting results in accordance with established guidelines. Conclusions RWE has been mounting over the years. The appropriate interpretation and use of such evidence often warrant adequate understanding about methodology. Researchers and policymakers should be aware of the methodological pitfalls when generating and interpreting RWE.