Editorial: The use of real world data for regulatory purposes in the rare diseases setting
Viviana Giannuzzi, Violeta Stoyanova‐Beninska, Virginie Hivert
Abstract
The use of real world data for regulatory purposes in the rare diseases setting Today, Real World Data (RWD) have been recognized as an important source of information not only for public health purposes but also for scientific-health research (EU Digital Strategy eHealth, 2022; Food and Drug Administration, 2018). As an alternative or complementary to the traditional clinical research setting, such as clinical trials, evidence generated in the real-world contributes to better understanding of diseases and life-cycle of medicines. This has been acknowledged not only by public authorities (European Commission, 2018), clinicians and researchers (Polak et al.), but also by patients, who claim the need to consolidate knowledge on the economic, social, and quality of life impacts of rare diseases (Delaye et al.).