Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases

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 Technical Guideline for the Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases. This guideline provides scientific recommendations for the development and implementation of DCT for rare disease drugs. It aims to solve the difficult and key problems during rare disease drug research and development, improve the efficiency and optimize patient experience. This article, combining the research and development concepts in the guideline, explains the current research and development thinking on the application of DCT in the research and development of rare disease drugs, with a view of providing reference for the industry.