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Real-world data from insurance claims could unlock a faster, cheaper way to assess whether drugs work for more peopel-and for conditions doctors haven’t even considered yet. That’s the promise of a growing field, and it’s already yielding surprising insights about popular medications.
Untapped Data Could Expand Drug Use Beyond Clinical Trials
Researchers are finding ways to leverage existing patient data to answer critical medical questions that traditional trials can’t-or won’t-address.
- Randomized clinical trials (RCTs) are the gold standard, but they’re expensive and frequently enough exclude certain populations.
- Researchers are using “real-world evidence” (RWE) from insurance claims to fill knowledge gaps.
- This approach has already revealed potential new benefits for drugs like semaglutide and tirzepatide.
- The goal is to accelerate medical progress and improve access to effective treatments.
For decades, randomized clinical trials have been the bedrock of medical approval in the U.S. But thes studies, while rigorous, aren’t always practical. They demand large patient pools,extensive data collection,and long-term follow-up-a costly and time-consuming process. The result? Fewer investigations into treatments that might be clinically valuable but lack the potential for substantial profit,leaving critical questions unanswered.
Q: Can insurance data really tell us if a drug works?
A: Yes. Researchers are developing methods to analyze real-world evidence-like insurance claims-to determine if a medicine is effective beyond its originally approved use, possibly accelerating the discovery of new benefits and expanding access to care.
“There’s little incentive for pharmaceutical companies to test combinations of treatments, for example, because a manufacturer typically aims to market its own product rather than evaluate it alongside another,” said Sebastian Schneeweiss, professor of medicine and epidemiology at Harvard Medical School. He added that certain patient groups-like pregnant women-are frequently enough excluded from trials for ethical reasons, creating further gaps in medical knowledge.
“There are very tangible use cases where we would love to have trials, but we’ll never have them, and we need to recognize this,” Schneeweiss, who also serves as chief of the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital, explained.”And at the same time, we’re sitting on this huge pile of longitudinal and patient-level data, of which only a very small proportion is used today to turn into evidence.”
This untapped data holds the potential to answer crucial questions-such as whether a diabetes drug could also prevent heart attacks, or if a treatment tested on middle-aged men is equally effective for pregnant women-far more quickly than a traditional RCT could manage.
For nearly a decade, Shirley Wang, associate professor of medicine at Harvard Medical School, and Schneeweiss have been working to transform these findings into actionable evidence. As co-directors of RCT-DUPLICATE (Randomized controlled Trials Duplicated using Prospective Longitudinal insurance Claims: Applying Techniques of epidemiology), they’ve pioneered methods that leverage real-world evidence (RWE)-including claims data from Medicare and Medicaid-to assess a medicine’s effectiveness beyond its initial approval.
Sebastian Schneeweiss (from left), nils Krüger, and Shirley Wang.
“We love RCTs,” Wang said,”but they can’t a