Edited by: Shuu-Jiun Wang and Michel Lanteri-Minet
Randomized controlled trials (RCTs) top the hierarchy of evidence for the assessment of new health technologies due to their high internal validity and reduction of confounding. However, RCTs have limited external validity with respect to the generalizability of results to a real-world setting. A particular limitation is the weak representativeness of a RCT population related to inclusion/exclusion criteria, restricted sample size and non-consideration of special populations (children and adolescents, elderly, pregnant and breastfeeding females, patients with comorbidities, patients belonging to particular ethnic groups). Other elements limit the use of RCTs data in daily practice, such as the duration of RCTs, which is often too short, and the lack of identification of predictive markers for therapeutic response. To improve the external validity of RCTs, it is recommended a supplementation with Real-World Evidence (RWE).
RWE is derived from analysis of Real-World data (RWD) collected from a variety of sources (registries, collections of electronic health records, administrative and medical claims databases…). RWD sources can be used for data collection but also to develop RWE randomized trials (large simple trials, pragmatic clinical trials) and observational studies (prospective and retrospective).
RWE providing clinical evidence plays an increasing role in health care decisions but, to be relevant and useful, it is essential that RWE be of high quality. In this perspective, many initiatives have been developed. In 2016, after the promulgation of the Cures Act designed to accelerate medical product development and bring new innovations and advances faster and more efficiently to the patients who need them in United States, the Food and Drug Administration (FDA) developed a framework for evaluating the potential use of RWE. More recently, European Medicines Agency (EMA) initiated the Data Analysis and Real Word Interrogation Network (DARWIN EU®) project with the vision to give EMA and national competent authorities in European Union Members States access to valid and trustworthy RWE.
In this context, The Journal of Headache and Pain has started a thematic series on Real-World Evidence of Emerging Therapies in Headache with the aim to collect high quality RWD dedicated to headache field.
In this collection, both review articles and original contributions are welcome.