Real-world data (RWD) and real-world evidence (RWE) both are increasingly important tools in healthcare and research, but there are important differences. The US Food and Drug Administration (FDA) defines RWD as “the data relating to patient health status,” and RWE as "the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD.”
Jeff Elton, CEO of health data technology specialists ConcertAI, points out that while both real-world data (RWD) and real-world evidence (RWE) are useful in clinical research, the latter goes further.
“Real-world data is just that—it’s the data elements,” Elton explained. “Real-world evidence isn’t just a data element—it goes beyond that. I’ve been able to pull data together and link it to an outcome, or interest that can be part of a regulatory or reimbursement decision. It’s a lot more meaningful.”
Elton said that while use and understanding of RWE had been marching forward before the arrival of the COVID-19 virus on the global landscape, the pandemic certainly had a significant impact on healthcare and research.
“Clinical studies and the actual number of patients over the course of 2020 went down—clinics closed, patients postponed treatment,” he said. “People with slow-progressing cancer may have postponed treatment; there were fewer visits, reduced contact with clinics to reduce exposure. There were many negative consequences—about six to eight months of depressed involvement, with fewer patients, fewer trials starting up, and researchers not progressing new medicines.”
One of the ways that trial teams have sought to help research bounce back, Elton explained, is increased use of RWE in research.
“Researchers have been using RWE to help inform study design, and to develop solutions to conduct studies in a different way,” Elton said. “As horrific as the pandemic has been in many ways, it also has helped to force more innovations out of the system.”
As researchers work toward more advanced, thoughtful trial operation in the midst and aftermath of the pandemic, Elton advised, intelligent application of RWE can be helpful as trial teams ask themselves important questions (including how clinical settings and visits work, how treatments might be impacted, which patients are the most vulnerable, proper standard of care, and more) to rethink how a study is designed.
“There’s an increasing number of things in our armory with which to conduct clinical research,” Elton said.
Patient population, and factors that impact segments or entire groups of potential recruits, also can help inform study design. Elton said while many companies speak of taking a “patient-centric” approach to their services, that can have a different outcome than a “patient-first” tack.
“I define ‘patient-centric’ as meaning that technologies, workflows, etc. are optimized around the patient’s interests, burden, etc.,” he explained. “‘Patient first’ is a way of prioritizing trade-offs—it is less of a ‘design consideration’ but rather the specific health outcomes, and perhaps holistic life requirements, of the patient that guides all decisions and all trade-offs."
And while patients must be a prioritized part of the equation, life-sciences professionals cannot fail to also focus on the disease.
“Clinical studies that are pre-approval are about establishing safety and efficacy, so it is about the disease response to a new therapeutic and about the physiological response of the patient to the therapeutic outside of the disease,” Elton said.
Also, Elton advised, patient considerations should permeate the entire trial planning and execution process.
“This is about study design; where the study is run; what study participation requires from the patient as differentiated from or comparable to the standard of care; and the prospective benefit from participation—stated in more simple terms, it is about administrative, technical, and clinical aspects,” he said.
Additionally, Elton said, trial professionals, need to take the patient burden into consideration, and work to design for their convenience.
“I need to design and run for low burden – it requires no more testing, or invasive procedures than standard of care. The data should be captured just through the process of receiving the care – zero or low-load capturing of data; all of this enhances the likelihood of moving a patient appropriately to a clinical study as an option, with higher assurance of completion,” he said.