TuberOus SClerosis Registry to Increase Disease Awareness: A Review on Alignment of Its Planning, Execution, and Publications With European Medicines Agency Guidelines

Patient registries offer a powerful and practical means of real-world data collection system for rare diseases. Many guidelines have been released to standardize patient registries, although most of them do not address issues specific to rare disease patient registries. In November 2018, the European Medicines Agency (EMA) released a draft discussion paper on methodological and operational aspects of disease registries and made proposals on good...