About this special issue
According to the FDA, real-world evidence (RWE) is “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data. Real-world data are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources”. RWE can be generated by different study designs or analyses, including but not limited to, randomised trials, large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective). Due to their ability to collect data on groups that are routinely excluded from randomised controlled trials, RWE studies have become recognised as a useful supplement during clinical development.
The wider context of RWE incorporates the discipline of medical affairs, which has been defined as the “medical speciality that protects patients’ interests by regulating pharmaceutical company activities and leads medicine adoption through the design, implementation and communication of real-world evidence targeted to the needs of regulators, payors, prescribers and patients”. The main purpose of medical affairs is to ensure that patients’ interests are safeguarded and not overshadowed by commercial need in the context of the pharmaceutical industry.
RWE exists within a specific context and has specific purposes for the multiple stakeholders involved in medicine adoption: regulators, payors, prescribers, and patients. However, RWE publications are currently dispersed throughout the body of knowledge, with no dedicated space where stakeholders can find the evidence they need for crucial decision-making. Additionally, since this is a time of great advancement in the generation and use of RWE during clinical development, there is a need for dedicated forums for engaging in academic debate