Natural History and Real‐World Data in Rare Diseases: Applications, Limitations, and Future Perspectives - Liu, Jing; Barrett, Jeffrey S.; Leonardi, Efthimia T.; Lee, Lucy; Roychoudhury, Satrajit; Chen, Yong; Trifillis, Panayiota (Journal of clinical pharmacology, 2022)
Keywords: Artificial Intelligence; Disease Progression; disease progression modeling; Drug Development; Humans; natural history; rare diseases; Rare Diseases - drug therapy; Rare Diseases - genetics; real‐world data; real‐world evidence; Research Design; rare diseases; real-world data; real-world evidence; natural history; disease progression modeling