RWE to Supplement RCTs: FDA Projects Continue

Regulatory NewsRegulatory News | 15 October 2019 |  By 

The US Food and Drug Administration (FDA) has been working with several different data and research companies to further its understanding of how real-world data (RWD) and real-world evidence (RWE) can help to inform its regulatory decision making.

One such company that has signed an RWE research collaboration with FDA is COTA, Inc., which is working on a project related to replicating a breast cancer study.

Andrew Norden, chief medical officer of COTA, told Focus in a phone interview that breast cancer is an area where RWE can prove valuable, as breast cancer clinical trials can take a long time to complete and as endpoints like survival or even progression can also take a significant amount of time to read out.

But Norden also cautioned that RWE should not be seen as a replacement for randomized clinical trials (RCTs). In fact, a recent study in JAMA Open Network found that RWD could only be used to feasibly replicate 15% of high-profile clinical trials conducted in the US in 2017.

“I don’t think the optimal scenario is to obviate the need for clinical trials,” Norden said, noting there “may be a small subset” of trials for which that’s possible, but, “Where this gets more rational and exciting is in the scenarios unfolding in oncology with a novel drug coming out that targets a novel mutation or an unmet need and patients don’t want to be randomized to standard of care or placebo… the question is can we use RWD to see if we can do better with the data from these single-arm trials.”

Or if a real-world control arm can help a sponsor make a better decision on if an investigational drug can be moved from Phase 2 to Phase 3, he added.

Mike Doyle, CEO of COTA, added that he thinks FDA is pushing forward on RWE at Congress’s request, under the 21st Century Cures Act, because of the opportunity to approve additional indications more quickly. But he also said FDA’s work in the RWE space is not necessarily contingent on how these collaborative agreements play out.

Both Doyle and Norden noted the potential for “enormous savings” from the use of RWD and RWE. “The goal here isn’t to replace a trial, it’s to separate existing trials and complement existing trials,” Norden added.

And though Norden discussed the potential for bias and other issues, he noted that it’s “highly unrealistic” to think RWE is going to replace clinical trials.

But can RWE accelerate the pace of clinical development? “The answer is yes and that’s moving forward,” he added, offering the example of the expanded indication for Pfizer Ibrance (palbociclib) thanks to RWD from electronic health records (EHRs).

Meanwhile, Aetion announced on Tuesday that it’s working with McKesson’s oncology EHR system to help researchers at Brigham and Women’s Hospital, who are leading the FDA demonstration project known as RCT DUPLICATE.


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Tags: cancer, RCTs, RWD, RWE, trials

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