In oncology, over 90 percent of patient treatment experiences occur outside of clinical trials, according to Jeff Allen, Ph.D., President and CEO of Friends of Cancer Research (Friends). As sources of real-world oncology data evolve, what will it take to facilitate broader access to that data and realize its full potential?

We recently convened a group of 50 health care leaders to discuss such questions—about the role of real-world evidence (RWE) and its potential impact on patient care—during “Evidence at the Apex," Aetion’s inaugural customer summit.

Our first discussion, moderated by Jeremy Rassen, Sc.D., Aetion’s Co-founder, President, and Chief Science Officer, focused on the opportunity to use RWE to shape cancer treatment, and brought together three oncology leaders: Dr. Allen; Deborah Schrag, M.D., M.P.H., Chief of the Division of Population Sciences at Dana-Farber Cancer Institute; and Steven D. Pearson, M.D., M.Sc., Founder and President of the Institute for Clinical and Economic Review (ICER).

The discussion highlighted a number of opportunities to maximize the potential of real-world data (RWD) in oncology. The following three stood out as especially critical. 

1. Achieve alignment across the industry on core oncology outcome measure definitions and how these should be applied in clinical practice.

Lack of measure standardization today means that outcome measures differ by data set, limiting the ability to aggregate data sets easily—and therefore limiting their broader use and value.

Further deepening disparities among real-world data sets is the fact that patient circumstances may drive very different assessments by their providers. If a provider will discontinue treating a patient, for example, they may assess them differently than they would a patient they do plan to continue treating. 

To enable interoperability across cancer data sets, the field of oncology needs standards, says Dr. Schrag. 

Because standards vary across cancer centers and other facilities, definitions around progression and censoring are not consistent. For critical endpoints like progression and progression-free survival, standards are essential, including in the use case of label expansion.

As the industry awaits regulatory guidance, organizations like Friends are helping the FDA explore and establish standards through projects like Pilot 2.0, for which Aetion is a collaborator. 

“I think the FDA is caught in the crosshairs of having to make decisions [about standards and outcomes],” Dr. Allen said. “From Friends’ standpoint, in our collaborative work in the real-world evidence space, we hope to lay a framework to begin to identify the variables that FDA and others that are analyzing real-world evidence grapple with.”

2. Facilitate patients’ data sharing through transparent communication about the potential applications and in-place protections for their data.

Building trust with patients is the foundation from which most real-world or clinical trial data emerges, and neglecting to invest in open, transparent dialogue and community building around patient data could have a negative impact on future oncology research. 

As Dr. Schrag said: “We need to do a much better job ensuring that patients understand how important their data is to curing cancer. And we have to quickly get better at making our case, letting people know that good data saves lives, well beyond participating in a Phase I clinical trial, for example.”

In addition to communicating to patients why their data is important, it is equally important to share how their data may be used, and the safeguards in place to protect these data.

3. Reframe our notions of what “good evidence” looks like to enable more exploration and identification of novel insights and opportunities within RWD.

To advance cancer care using RWD, we must think beyond traditional study designs and endpoints. Data vendors have the opportunity to work closely with health systems to incorporate non-traditional endpoints that are reflective of real-world practice, and which may enable additional context and value for a patient’s outcomes.

Dr. Pearson explained: “Most of us were trained in a generation of clinical epidemiology in which good evidence doesn't look like real-world evidence. And we have the biases attendant with that. Because some of the terms used with real-world evidence are foreign to those who assess evidence, we need to educate decision makers on real-world evidence and its potential.”

As Dr. Pearson commented during the summit, RWE will need to work its way even further upstream to impact cancer care. Given that the FDA is beginning to use RWE beyond safety, it will be critical to access outcomes through electronic medical records and other data-gathering systems. However, this requires all stakeholders to cooperate and be incentivized to work together to solve the problem of interoperability.