Integrative Approaches to Treatment Evaluation Leveraging Randomized Clinical Trials and Real-World Data

Music City Center 

The 21st Century Cures Act, enacted in 2016, empowers the FDA to accelerate the development of new treatments by utilizing real-world data (RWD) and evidence. As a result, parallel randomized clinical trials (RCTs) and RWD are becoming increasingly available for evaluating treatment outcomes. Integrating heterogeneous data sources presents a unique opportunity to address clinical questions that cannot be answered by any single data source alone. This talk will explore various objectives and methodologies for conducting integrative analyses of data from RCTs and RWD. By combining the strengths of both RCTs and RWD, researchers can improve the generalizability of RCT findings using the broader representativeness of RWD, increase the efficiency and statistical power of treatment effect evaluations by incorporating comparable RWD, and assess long-term safety and efficacy by utilizing extended real-world follow-up data. Specifically, we will discuss newly developed strategies to mitigate biases and optimize treatment evaluation in hybrid clinical trials with external controls, including approaches such as test-then-pool, selective borrowing, and conformal prediction.

Real-world data

Real-world evidence

Hybrid trial designs