Use of real-world evidence (RWE) in the pharmaceutical industry is becoming increasingly common. It aids the clinical development process and provides important data for pricing discussions once medicines have been approved. With that in mind, PharmaPhorum’s Richard Staines spoke with Aetion President and Chief Science Officer Jeremy Rassen about how data obtained from outside the traditional randomized controlled trial setting is transforming drug research.
Rassen defines real-world data as data that represents the actual experience that patients have with the medications that they have been taking. This data can be generated in any ways, from electronic health records and registries to patient-reported outcomes and data collected from devices such as Fitbits or smartphones.
“Healthcare today is facing enormous challenges around figuring out how to choose the best treatments. What treatments work best, for whom, and under what circumstances?” Rassen said. “Real-world data gives us the opportunity to bring the safest, most effective treatments to market and to ensure that the patients who can benefit the most have access to and are receiving those treatments.”
But, Rassen said, there is a difference between data and evidence. “We talk a lot about real-world data, but ultimately decisions are based on real-world evidence. Real-world evidence is the application of sound scientific principles to real-world data, conducted in a way that’s recognized and supported by industry, by advisory groups, and ultimately regulators. Applying those techniques to real-world data yields real-world evidence from which scientifically valid decisions can be made.”
Rassen noted that RWE is already being used “routinely” in clinical development to inform clinical trial design—for example, to set expectations or thresholds for performance. It allows companies to assess the market size for early-stage drugs or to determine the precise incidence and prevalence of different diseases. It is also frequently used to respond to regulatory questions. But mostly, RWE is being used to augment RCTs rather than replace them.
“It’s not an either-or situation,” Rassen said. “There are certain RCTs that could never be replaced in any way by real-world data, and there are certain RCTs where RWE can be used uniquely well to accomplish the goals of the trial in much less time and at a much lower expense.”
The FDA is definitely interested. “The FDA, in their strategic framework, has made it clear that they are very much invested in the use of real-world data and evidence for regulatory decision making. But questions remain about how precisely to approach that,” Rassen said.
In an attempt to answer these questions, the FDA launched the RCT DUPLICATE demonstration project, which is being conducted by Brigham and Women’s Hospital/Harvard Medical School in collaboration with Aetion.
“The researchers are using real-world data to attempt to replicate the results of 30 randomized trials that the FDA has used over the last several years in regulatory decision-making,” Rassen said. The FDA will use the results of the study to think about how the agency can use real-world data and real-world evidence in its decision-making and plans to release specific guidelines by 2021.
Rassen said he is “thrilled” about the future use of real-world evidence, noting that Aetion is not the only company working in the field. “I see this as a worldwide phenomenon,” he said. “There are similar discussions happening in the EMA and PDMA in Japan.”
Read the full interview here.