China’s Center for Drug Evaluation (CDE) has released draft “Technical Guidelines for the Application of Decentralized Clinical Trials in Rare Disease Drug Clinical Research and Development,” open for public feedback for one month. The guidelines have been formulated by a research team that extensively reviewed the guidance and suggestions from foreign regulatory agencies such as the FDA and EMA on the implementation of decentralized clinical trials (DCTs). The team engaged in discussions with clinical trial institutions and industry stakeholders to assess the necessity, feasibility, and rationality of implementing DCTs in China, particularly in the context of rare disease research and development.
Given that the comprehensive implementation of DCT in China is still maturing, and the urgency is highest in the field of rare disease research and development, these guidelines are proposed to address key issues such as limited and widely dispersed patient populations, the need for high follow-up frequency in rare disease drug development, and considerations for patient experience and protection.
Decentralization of clinical research on rare diseases has been under discussion since August 2022. DCTs shift clinical trials from offline to online, reducing barriers for patient participation. Patients can interact remotely with clinical doctors and submit information without face-to-face contact, leading to a more convenient experience and improving patient access. However, the practice of DCT in China is still limited, and issues such as patient privacy protection remain to be addressed.
The document highlights that when applying for DCT/DHT within a clinical research program for rare disease drugs, sponsors should pay attention to clarifying the responsibilities and communication channels of DCT parties, developing an effective risk management plan, and considering new methods and models that involve subject personal privacy protection, data security, and other compliance issues. Special consideration should be given to the DCT process on the basis of traditional clinical trial implementation. During the implementation of DCT, sponsors need to ensure that generated data is properly recorded and traceable. Applicants/sponsors planning to include DCT elements and DHT in the clinical development plan should communicate and exchange ideas with the CDE in a timely manner and maintain close communications with the CDE throughout the overall implementation process.
The protocol of DCT and the application of DHT provide a new digital model for drug clinical research and development. Based on the application scenarios of DCT, the document suggests considering remote recruitment outside the research center and collection of electronic source data within the research center, doctor-patient interaction/education, electronic informed consent, remote clinical trial visits, and Direct to Patient (DTP) and remote drug recovery as convenient and beneficial drug management methods for rare disease patients.
