The Center for Drug Evaluation (CDE) has released the “Guiding Principles for Natural History Research of Rare Diseases in Drug Development,” addressing the limited availability and reliability of natural history information for rare diseases in China. This move follows the US Food and Drug Administration’s 2019 release of the “Rare Diseases: Natural History Studies for Drug Development,” which did not have a direct counterpart in China until now.<\/p>\n\n\n\n
Understanding Natural History Research<\/strong>
Natural history research of disease refers to observational studies designed to enhance understanding of a disease, including its development and prognosis. The aim is to identify relationships between demographics, genetics, environmental factors, and other elements such as treatment methods and medication, and how these influence the disease’s development and outcome. In these studies, patients may receive current standard treatment and\/or treatment for complications. Any interventions that could alter clinical manifestations or disease progression should be included as research elements.<\/p>\n\n\n\n