FDA: With better definition, real-world data poised to inform study design, drug approvals

Officials with the Food and Drug Administration see tremendous potential in using data buried in EHRs, insurance claims and smart devices to advance drug and device approvals using real-world evidence. But the agency also faces distinct challenges in defining those data elements. 

Pulling together evidence on drug effectiveness and safety requires a pragmatic approach to research and multidimensional data sources, three FDA officials wrote in an article in the Journal of the American Medical Association (JAMA). This means that both random clinical trials and single-intervention trials are key to effectively using data pulled from medical records and even social media to generate valuable evidence.

“There is substantial enthusiasm for the use of real-world data sources to generate so-called real-world evidence (RWE), but confusion remains about what RWE means,” they wrote. “Generation of RWE therefore is not limited to observational studies but also includes randomized trials conducted in clinical settings.”

There are also complications with the continuity of real-world data since it is collected for patient care rather than research. 

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The FDA has been eying the impact of real-world data for some time. Last week, President Donald Trump signed a bill reauthorizing the FDA’s user fee agreements for drugs and devices. Those agreements included provisions to establish a new digital health unit and a National Evaluation System for health Technology (NEST) which plans to leverage data in EHRs and health registries. 

Real-world data has other regulatory uses beyond generating evidence that can be used in drug approvals, according to the JAMA article. Researchers can use that data to guide more effective study design; for instance, it could help determine which study sites have the most useful participant candidates and provide a strong basis for calculations.

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During a study, greater reliance on real-world data can cut down on duplicate information, like medical histories, and better inform end points, FDA officials wrote. 

The FDA has said in the past that using real-world data can help keep pace with medical device innovation and development moving. For the agency specifically, it means faster approval of drugs and devices, ensuring quicker access for patients.