PP09-63: Registry-Based Randomized Clinical Trial (R-RCT) – The Future of RWE Registration Trial
Jingyu (Julia) Luan
Global Regulatory Affairs Director, BioPharmaceuticals R&D, CVRM
AstraZeneca United States
To present the history, background, basic design elements, advantages, challenges, and regulatory acceptance of Registry-based Randomized Clinical Trial
Literature review and real and/or mock case studies will be utilized to introduce the background of R-RCT, general design elements, comparison to traditional RCT, and discuss the regulatory acceptance from a global perspective, e.g., US, EU, Japan, Korea, and China.
In recent years, Real World Evidence (RWE) is increasingly becoming important for regulatory decision-making. Regulators around the world encourage the industry to explore different ways to enhance the use of RWE in regulatory submissions. Among the many possibilities of incorporating RWE in regulatory decision-making process, adopting pragmatism into regulatory RCT is considered as a highly promising way of utilizing RWE in regulatory decision making by the regulators and, furthermore, R-RCT is deemed as a very acceptable design of adopting pragmatism into RCT.
R-RCT is defined as a prospective randomized clinical trial that uses a clinical registry for one or several major functions for trial conduct and outcomes reporting. It is a hybrid of traditional randomized clinical trial (RCT) and pragmatic clinical trial (PCT). Compared to conventional RCT, R-RCT increases external validity and generalizability due to real-world setting of clinical trials, improves efficiency due to design streamlining and usage of existing healthcare infrastructure, reduces investigator and patient burden due to integration of clinical trial modules into existing healthcare practice, and decreases cost allowing more studies to be conducted to advance clinical care and drug development. However, R-RCT also faces some challenges, including registry data quality, ethical issues, and methodological challenges.
We will conduct thorough literature review in R-RCT and utilize real and/or mock case studies to introduce general design elements and comparison to traditional RCT. Furthermore, we will provide an overview of global regulations in RWE registration trials e.g., US, EU, Japan, and China, and discuss/analyze the regulatory acceptance of R-RCT as RWE registration trial.
In summary, among many possibilities of incorporating RWE in regulatory decision-making process, up to date, R-RCT emerges as one of the most promising ways, if not the most promising way, for RWE registration trial. The concept of R-RCT is of high interest to the scientific, medical, and regulatory communities. R-RCT design enables conducting more clinical trials with fewer resources and lower cost within shorter period and presents the bright future of RWE registration trial.