CERSI Summit: Wider use of real-world evidence continues to face hurdles

Regulatory NewsRegulatory News | 11 January 2023 |  By 

Jeff Shuren (top) and Mathai Mammen (bottom)

The widespread use of real-world evidence (RWE) in regulatory decisions continues to face challenges, including problems with data quality and methodologies, and a disconnect between the endpoints used by regulators and the information collected in clinical practice, according to panelists at the 2023 Innovations in Regulatory Science Summit sponsored by the UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI).
“I wish we could get rid of the term real-world data and real-world evidence,” Jeff Shuren, MD, director of the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration (FDA), said at the CERSI Summit. “We were an early adopter of that at the FDA and it’s become a four-letter word. At the end of the day, we’re talking about clinical evidence.”
Having endpoints that both meet regulatory standards and can be collected in routine clinical practice is a significant barrier, but one that is hard to tackle one sponsor at a time, Shuren said. He suggested that an “all-of-government effort,” that included the National Institutes of Health, could move this issue along.
“No individual company is on their own seeing the value in doing it. You really need to pull those resources together to make it work. I think there’s value if government were to lead that,” he said.
The discrepancy between clinical endpoints and trial endpoints are often “a matter of choice,” said Mathai Mammen, who is transitioning out of his role as executive vice president of pharmaceuticals, research and development at Johnson & Johnson.
“Regulatory bodies have grown accustomed to certain endpoints as valid, but in this world of new diseases and rare diseases and revisiting old diseases, putting different boundaries around existing diseases, there’s an opportunity to draw more measurements in a clinical development program [and] try to match them with measurements that are actually made in the real world,” Mammen said.
Another key issue is data quality. While registries have been a key source of high-quality real-world data, Shuren said a lot of time and resources are spent cleaning up that data to make it usable. “How can we make the data good at the outset? And quite frankly, that goes back to clinical workflows. This about industrial redesign,” he said.
Shuren suggested that groups like CERSI could undertake efforts to assess clinical workflows to find ways to produce consistent, reliable data that could be used as part of real-world studies. Reducing the cost of curating that data at the front end of the process could dramatically increase the value and efficiency of real-world data that is used to generate real-world evidence, he said.
If real-world evidence methodologies can be made more rigorous and the endpoints aligned with routinely collected clinical data, the result could be more reliable and more easily conducted comparative effectiveness studies and better data about a drug’s effectiveness and safety in subpopulations, Mammen said. “There’s a lot to do here, but I’m very optimistic that with both rigorous methodologies being demanded and endpoints being evolved, we can get to a much better place for patients,” he said.  


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