What is real-world evidence (RWE), and why does it matter?
RWE is derived from real-world data (RWD) – which can come from sources like electronic health records (EHRs), medical claims, billing data, product or disease registries, or mobile devices. The FDA defines RWE as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials.”
The promise of RWE is a fuller picture of the risks and benefits associated with therapies than we can get from traditional randomized clinical trials (RCT) alone. We can learn more about a broader population of patients, over a longer period of time, in the actual situations patients experience outside the clinic.
I attended a recent conference on the importance of RWE: the 8th Annual Blueprint for Breakthrough Forum, titled “Validating Real-World Endpoints for an Evolving Regulatory Landscape.” The biggest achievement of this meeting (organized by the nonprofit Friends of Cancer Research) was its success in convening diverse stakeholders (including representatives from the FDA, ASCO, pharma, CROs, tech, payers and, very importantly, patients) to discuss how to better use RWD and RWE. These were the meeting’s highlights for me:
Results were presented of a pilot 2.0 study in advanced non-small cell lung cancer (aNSCLC) and discussed the trade-offs of using RWE in oncology. The study demonstrated that it was feasible to use real-world endpoints to assess front-line treatment regimens using EHR and claims data. We discussed the challenges of RWD, why it behaves differently from RCT, and what information is still missing. Hearing a voice of a cancer patient in that discussion was particularly illuminating.
Christie Mangir, a breast cancer survivor, pinpointed limitations of RCTs. Diagnosed with a rare, aggressive type of breast cancer, Christie had a difficult choice when selecting treatment because there was no data relevant for her tumor type. After beginning therapy, she was bedridden, had multiple ER visits, and could not exercise. Traditional endpoints used for drug approvals (like response rate or progression-free survival) offer no insight into quality of life (QOL) issues, which could be critical information to help patients and physicians make treatment choices.
Jacqueline Corrigan-Curay, Director, Office of Medical Policy, Center for Drug Evaluation and Research (CDER), FDA, pointed out that integration of mobile technologies into RWD can provide an opportunity to fill the gap. Wearable sensors and electronic patient reported outcomes (ePRO) make it possible to collect QOL information as people go about their daily life. It can be done in both RCT and real-world settings.
This message was echoed by Janet Woodcock, Director for CDER at the FDA, who emphasized the importance of “beefing up RWE with data provided by patients”.
The FDA created a Framework for Real-World Evidence last year with their latest thinking and recommendations, in response to RCT’s limitations and rising costs. The industry widely recognizes the need to move beyond traditional trials to help patients. Recently, Koneksa presented data collected with Montefiore Cancer Center demonstrating that step count data (derived from fitness trackers) can serve as a strong predictor of short-term hospitalization risk. We need more real-world patient-generated data like this to learn more about how treatment actually affects patients, and better guide care.