1.Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases
Huanhuan CUI ; Ling TANG ; Can CUI ; Zhuxing YAO ; Zhimin YANG ; Haixue WANG
JOURNAL OF RARE DISEASES 2024;3(2):175-180
Clinical trials of drugs for rare diseases face special challenges such as a limited number of patients, difficult recruitment, long trial period, and frequent video interviews during the trial. Therefore, in the clinical operation of rare diseases, a decentralized clinical trials (DCT) model based on the " patient-centred" research and development concept is implemented. With the help of decentralized elements and digital health technology, the barriers of geographical restrictions can be overcome and subjects do not have to be limited to traditional clinical trial sites (hospitals/research centers), which can significantly reduce the burden on subjects, increase their representation, and obtain a wider range of scientific research data. To guide the industry′s scientific and standardized application of DCT in the research and development of drugs for rare diseases, the Center for Drug Evaluation of the National Medical Products Administration (NMPA) organized the stake holders to draft the