Researchers Find 15% of Trials Could be Replicated Using Real World Data

Regulatory NewsRegulatory News | 10 October 2019 |  By 

A study published this week in JAMA Network Open finds that currently available real-world data (RWD) sources can only be used to feasibly replicate 15% of clinical trials.
The aim of the study was to determine whether RWD could be used to power observational studies that answer the same clinical questions as traditional clinical trials.
Randomized controlled trials (RCTs) are considered the gold standard for clinical evidence to support the safety and efficacy medical products due to high levels of internal consistency and reduced bias.
However, as the authors of the study write, “Compared with RCTs, RWE [real-world evidence] better reflects the actual clinical environments in which medical interventions are used, including patient demographics, comorbidities, adherence, and concurrent treatments,” noting that RCTs are costly and time intensive compared to observational studies.
To conduct the study, the authors reviewed 220 clinical trials conducted in the US that were published in the top seven medical journals in 2017 and determined whether RWD obtained from insurance claims and electronic health records (EHRs) contained the information necessary to replicate the studies.
Of those trials, the authors were only able to identify 86 (39%) that “had an intervention that could be ascertained from insurance claims and/or EHR data.” From there, the authors narrowed the trials further to identify ones with an indication and inclusion/exclusion criteria that could be extracted from RWD.
From there the authors were able to identify just 33 (15%) trials with one or more primary endpoints that could be ascertained from available RWD sources.
“This finding suggests the potential for real-world evidence to complement clinical trials, both by examining the concordance between randomized experiments and observational studies and by comparing the generalizability of the trial population with the real-world population of interest,” the authors write.
However, the authors caution that for new products, RWE is unlikely to serve as a replacement for RCTs and point out that many of the trials they looked at could not be replicated because the data necessary to do so “are unlikely to appear in an EHR in structured form if at all.”
But the authors stress that RWE could be used to provide “critical insights” into product safety and efficacy in the postapproval setting and could allow the US Food and Drug Administration (FDA) to identify and act on safety issues more quickly.


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