RCT-DUPLICATE Findings Demonstrate Capability of Real-World Evidence Studies to Reach Conclusions Similar to Randomized Clinical Trials

Real-world evidence (RWE) technology and analytics company Aetion has announced the culmination of the RCT-DUPLICATE demonstration project, with complete findings published in The Journal of the American Medical Association (JAMA). This three-year initiative tested whether researchers asking clinical questions in real-world data (RWD)—data from patients’ day-to-day interactions with the healthcare system—would obtain similar results to findings from randomized clinical trials (RCTs). In cases where clinical trial designs aligned with real patient care processes, the RWE studies and RCTs came to largely similar conclusions.

As part of the RCT-DUPLICATE initiative, researchers created and published protocols for observational, real-world analogs to 30 completed and two ongoing RCTs, emulating each trial’s design as closely as possible. They then implemented the studies principal using the Aetion Evidence Platform (AEP), which analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on the safety, effectiveness, and value of medical treatments.

The results of Aetion’s work show that in about half of the cases where researchers were able to closely mimic the design of the corresponding RCT using RWD, the RWE study came to a similar conclusion as the analogous RCT. In many cases where RWE and RCTs did not come to a similar conclusion, the RCT design itself did not align with real-world clinical practice, creating a challenge for emulation of the trial using RWD. In these instances, RWE and RCTs may both be reaching meaningful conclusions, but to subtly different research questions.

“The RCT-DUPLICATE initiative further demonstrates that RWE has the potential to augment findings from RCTs and guides us to cases where RWE and RCTs may be expected to reach similar conclusions,” said Aetion Chief Science Officer Nicolle Gatto, Ph.D., MPH. “This work is important in promoting and understanding the value of RWE for decision-making as we continue to assess RWE’s uses, benefits, and limitations.”

The Aetion publication, alongside ongoing research like the Coalition for the Advancement of RWE through Randomized Controlled Trial Emulation (CARE) Initiative and other demonstration projects, adds to a body of evidence that demonstrates how RWE can be relied on to broaden our understanding of the safety, effectiveness, and value of medications—complementing or extending what can be demonstrated by RCTs.

Read the original press release here.

Read the JAMA paper here.