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Real World Evidence Shown to Emulate Some Clinical Trials

Data pointing finger

(Vitaly Vlasov, Pexels)

26 Apr. 2023. A study of insurance claims records matching the design of randomized clinical trials shows claims data can in some cases resemble clinical trial results to make causal inferences. The analysis of real-world evidence matching clinical trial data appears in the 25 Apr. 2023 issue of the Journal of the American Medical Association or JAMA.

The JAMA paper reports on work by the RCT-Duplicate project, an initiative of researchers at Brigham and Women’s Hospital in Boston, a teaching and research hospital affiliated with Harvard Medical School. RCT-Duplicate — RCT standards for randomized clinical trials — seeks to discover if data stored in large-scale medical databases, such as insurance claims or electronic health records, can be mined to answer questions about the efficacy or safety of therapies, without conducting full randomized clinical trials. While randomized trials are considered the gold standard for decision-making on these questions, they’re often expensive and long-term undertakings, thus the need for alternative, yet still reliable, methods for generating useful data.

The RCT-Duplicate project receives financial support from the Food and Drug Administration, for the agency to better understand if real-world medical evidence can help conduct its regulatory functions. The 21st Century Cures Act, signed into law in Dec. 2016, calls for FDA to evaluate real-world evidence as a regulatory tool. In Apr. 2019, Science & Enterprise reported on FDA’s role in the project testing real-world evidence for post-marketing assessments of drugs already approved by FDA, using software made by Aetion Inc., a medical technology company in New York.

Claims records matched to clinical trial design

The JAMA paper reports on de-identified data drawn from health insurance claims databases maintained by Optum and IBM MarketScan, as well as Medicare claims, correlated with data from 30 completed and two ongoing drug-assessment clinical trials. The authors — Shirley Wang and Sebastian Schneeweiss, pharmaceutical epidemiologists and economists at Brigham and Women’s Hospital and co-directors of the project — selected the 32 trials in a range of health conditions for their propensity to emulate insurance claims characteristics. The researchers matched insurance claims records to the design of clinical trials, particularly their populations, interventions, comparator variables, outcome measures, and time.

The study team used the Aetion Evidence Platform algorithms to emulate randomized clinical trials with insurance claims records. In their analysis, the authors found where insurance claims records closely match key clinical trial properties, in 16 of 32 or half of the trials, data from the insurance claims files are strongly correlated to clinical trials findings. In the other 16 trials, where some but not all clinical trial characteristics match up to insurance claims, correlations are weaker. The authors note the divergence between clinical trial and insurance claims properties, and in some cases pure chance, impairs the ability to draw clear conclusions from the claims data.

Where clinical trial properties can be emulated in insurance claims — not always an easy task say the authors — real-world evidence or RWE can complement clinical trials to better understand how medications work in clinical practice. “The RCT-Duplicate initiative further demonstrates,” says Aetion chief scientist Nicolle Gatto in a company statement, “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.”

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Disclosure: The author owns shares in IBM.

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