Real World Evidence (RWE) is finally arriving as a valued complement to clinical trials.
RWE is generated outside of the clinical trial environment, through observational data gleaned from electronic medical records (EMR), pharmacy claims, and patient reported information. It provides valuable information about the real world impact of diseases and treatment journeys.
Many stakeholders have been hesitant to use RWE due to the limited control they have over the data collection process and the inability to conduct audits of third-party data. But, as the industry hears more success stories about the benefits of RWE as external comparators for new treatments, and the use of RWE in regulatory approvals increases, stakeholders are becoming more open to using Real World Data (RWD) in their research plans.
The challenge they face now is how to capture and use RWE in a way that is transparent, reliable, and acceptable to regulators.
Earlier this year, I spoke with Dr. Amy Abernethy, then principal deputy commissioner and chief information officer at the FDA, about the changing regulatory landscape for using RWE, and how sponsors can create a robust RWE methodology.
She noted that the best source of insights into what regulators are thinking is FDA’s 2018 Framework For Real-World Evidence Program. The document offers an overarching rubric for evaluating the potential use of RWE in the submission process. It provides the context for RWE use, and can help sponsors begin to define their RWE approach and methodology.
Organizations need to determine what data they are going to collect, where it will come from, and how they will analyze it. “In a landscape where data sources are constantly changing, we want analytic methods that are as near-real time as possible and able to accommodate many use cases,” Dr. Abernethy said.
The biggest challenge for sponsors is figuring out whether the data and analytics methods are good enough for the given use case, and whether they will be acceptable to regulators.
Without clear guidance, creating an acceptable RWE strategy is complicated. But Dr. Abernethy suggested that sponsors address the following five questions to pressure test their study designs and methods, and avoid making choices that weaken the validity of their RWE approach.
Regulators’ increasing attention and acceptance of RWE offers an opportunity to create more robust submission packages that demonstrate the benefits and value of their treatments in real world settings. Those organizations that invest the time now to create a thoughtful RWE strategy that can be vetted for quality and reliability, and who are willing to talk with regulators in advance of executing any such plans, are likely to produce more compelling cases for market approval, while also quantifying clinical benefits that translate readily to real-world settings. Now is the time to embrace the use of real world evidence to enhance your regulatory submissions.