Conference Program

Leveraging Real-World Data to Accelerate Regulatory Clinical Studies: Hybrid Study Design (303724)

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Wei-chen Chen, FDA/CDRH 
Heng Li, FDA/CDRH 
Nelson Lu, FDA/CDRH 
Changhong Song, FDA/CDRH 
Ram Tiwari, Bristol Myers Squibb 
Chenguang Wang, Johns Hopkins University 
Yunling Xu, FDA/CDRH 
*Lilly Yue, US Food and Drug Administration 

Keywords: Real-world data, hybrid study design, Propensity scores

High-quality real-world data may be utilized to generate real-world evidence for regulatory clinical studies. This presentation will focus on leveraging real-world data (RWD) via hybrid study designs in regulatory clinical studies: 1) A non-randomized comparative study in which RWD is used as a comparator group for a traditional clinical study; 2) A traditional single arm, non-comparative study consisting of a pre-specified number of prospectively enrolled patients augmented by patients from RWD; and 3) A traditional randomized controlled trial (RCT) consisting of prospectively enrolled patients augmented by patients from RWD to approximate a traditional RCT of a larger sample size. Innovative propensity score-based methods will be discussed, which are used to design such clinical studies and deal with potential confounding bias introduced by leveraging RWD. Examples based on pre-market regulatory review experience are provided to illustrate the implementation of these proposed methods.