By Fineline Cube 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.
Understanding Natural History Research
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.
Initiating Natural History Studies
The study of natural history can be initiated by drug development enterprises, researchers, or patient organizations. It is important to note that drug development should not be delayed waiting for the results of these studies, as they often face a series of unknown factors. However, the value of natural history research in the development of rare disease drugs is significant, and data from these studies can serve as a useful supplement to drug registration research.
Protecting Patient Privacy and Data Confidentiality
Research on the natural history of diseases should have corresponding measures in place to fully protect patient privacy and ensure the confidentiality of data. For the application purpose, scope of use, relevant parties using the data, and the rights of the research object, the research object or family members (guardians) should be fully informed, with informed consent or general informed consent or exemption (such as retrospective studies). The research plan should be approved by the ethics committee, and the collection and use of corresponding data should comply with the requirements of relevant laws and regulations.-Fineline Info & Tech